Intense. Sharp. Continuous. Unrelenting. Pain. 

For Tim, a sickle cell pain crisis, medically known as a vaso-occlusive crisis (VOC), isn't just discomfort. It's pain so intense it can bring life to a halt. 

Other times, he said, the pain feels like "somebody really big is sitting on your chest," making it difficult to breathe. 

Tim gets a visit from Carter Jensen of the Kansas City Royals. 

Tim has lived with sickle cell disease since he was 1 month old. The inherited blood disorder has shaped nearly every chapter of his life, bringing frequent hospitalizations, missed school days, surgeries and complications that most young adults never have to consider. In 2025 alone, Tim was admitted to the hospital approximately 11 times because of severe pain crises. After a lifetime of battling the disease, he is now making history as the first Children’s Mercy patient to receive gene therapy for sickle cell disease, marking a milestone both for him and for the future of care at Children’s Mercy.  

A lifelong battle 

Diagnosed shortly after birth, Tim became a familiar face at Children’s Mercy. Hospital admissions, blood transfusions and pain management became part of his routine. Despite the challenges, he developed a determination that impressed his care team. Over the years, he learned how to advocate for himself, take ownership of his health and navigate the realities of a chronic disease that follows patients everywhere, from classrooms and family vacations to major life milestones. 

"I missed so much school," Tim recalled. Yet he refused to let sickle cell disease define him. Even when complications from the disease led to hip surgery shortly before prom, he worked relentlessly to regain his mobility so he could attend. "I wasn't sure I was going to be able to make it," he said. "I was still having trouble standing on my own two feet." Ultimately, he made it to prom and graduated from high school in 2024. Looking back, Tim says one thing has always remained true: "I never let sickle cell keep me down."  

Sickle cell disease is an inherited blood disorder caused by a change in hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. Instead of remaining round and flexible, red blood cells become rigid and sickle-shaped. Those cells can block blood vessels, reducing oxygen flow and causing severe pain as well as serious complications affecting organs throughout the body. 

Shabnam Arsiwala, MD, director of the Sickle Cell Disease Program at Children’s Mercy, explains that sickle cell disease affects much more than blood. "Because blood flows everywhere, it can affect every organ of the body," she said. "It can cause pain crises, stroke, vision loss, acute chest syndrome, kidney disease and many other complications." Sickling occurs continuously, but certain triggers such as illness, infections, weather changes or physical stress can dramatically increase its severity and cause patients to enter crisis.  

While many patients live full lives with medication and supportive care, Tim's disease remained particularly severe. According to Dr. Arsiwala, his 11 hospital admissions in a single year placed him among the most medically complex patients in Children’s Mercy's sickle cell population. 

"We saw that his disease was very complex," Dr. Arsiwala said. "He was getting admitted for pain crises quite a lot, and that was limiting his potential and what he could do for himself and for his growth as a person." At the same time, she witnessed something equally important: Tim's readiness. She described him as responsible, accountable and increasingly independent in managing his health care. Those qualities helped make him an ideal candidate when the conversation turned to a groundbreaking new treatment option.  

A new possibility 

That conversation began in 2025. 

Tim poses with Dr. Keith August at the Hyundai Hope on Wheels event where he and other patients got to leave their handprints on a Hyundai vehicle. 

Members of Tim's care team approached him and his parents, Tasha and Tim Sr., to discuss gene therapy, a treatment designed to fundamentally transform how sickle cell disease behaves in the body. They reviewed the risks, benefits and lengthy treatment process. Tim and his family took time to learn about the therapy and ultimately decided to move forward.  

Considering this a phenomenal opportunity for their son’s future, Tasha said, "For our son to even have a chance at a regular life, it's mended the little patches of broken heart we've had throughout the years." 

Tim recalled his excitement: "I said yes, I would love to. I'd be very interested."  

For Tim, saying yes launched a journey that has been underway for nearly a year and continues today. 

Unlike traditional treatments that manage symptoms, the gene therapy Tim is receiving, called CASGEVY®, works by using a patient's own blood stem cells. Those cells are collected, sent away for specialized gene editing and then returned to the patient after chemotherapy preparation. The process increases production of fetal hemoglobin, a powerful form of hemoglobin present before birth that can help prevent red blood cells from sickling.  

The science behind the treatment is complex. The hope behind it is simple. 

"The thought is that he won't need to come into the hospital for vaso-occlusive crises anymore," said Jean Freudenthal, PharmD, Advanced Therapeutics Program Director at Children’s Mercy. "This is about improving quality of life and allowing someone to get back to normal things."  

Building a gene therapy program 

For Dr. Freudenthal, Tim's treatment represents far more than a single patient milestone. She was hired to help build Children’s Mercy's Advanced Therapeutics Program and establish the infrastructure necessary to bring cutting-edge cell and gene therapies to patients across multiple specialties. The work required years of planning, coordination and partnership across disciplines. 

"This is a huge milestone," Dr. Freudenthal said when discussing Tim becoming the hospital's first sickle cell gene therapy patient. "Tim is our first patient receiving gene therapy for sickle cell disease, and hopefully he is only the beginning."  

Preparing Tim for treatment has required a highly coordinated effort involving specialists in hematology, bone marrow transplant, nursing, pharmacy, psychology, social work, rehabilitation medicine, pain management and numerous other team members. After consenting to treatment, Tim began months of preparation that included stopping his hydroxyurea therapy, receiving red blood cell exchanges, and undergoing stem cell collection so his cells could be edited and manufactured for infusion. Tim needed only one cell collection cycle as opposed to an average of two to six cycles in clinic trials, moving Tim’s case faster than many patients experience. His cells were then sent for manufacturing before being returned to Children’s Mercy for the next phase of treatment.  

For Tim, the process has been worth it, even when it has been difficult. 

"The hardest part for me has been the blood transfusions," he said. Years of medical procedures have made finding veins increasingly difficult, often turning routine visits into hours-long appointments. Yet he has remained focused on what comes next.  

A transformative future 

The outcome physicians hope to achieve with this treatment is often described as transformative. 

At Children’s Mercy discoveries become new possibilities for patients and families.

Rather than turning off the gene responsible for sickle cell disease, the gene-editing process reactivates fetal hemoglobin production. The result is a dramatic decrease in the impact of the disease and the possibility of eliminating future pain crises and many complications associated with sickle cell disease. 

"We call it transformative therapy," Dr. Arsiwala said. "The disease burden is not there anymore because fetal hemoglobin becomes so powerful." While previous organ damage caused by sickle cell disease cannot be reversed, new complications may be prevented, and many patients experience dramatic improvements in quality of life.  

Clinical studies have produced encouraging results. Among participants ages 12 to 35 who received CASGEVY® and had sufficient follow-up, 93.5% experienced no severe vaso-occlusive crises for at least 12 consecutive months after treatment. Researchers have also reported dramatic reductions in hospitalizations related to sickle cell disease.  

Looking beyond sickle cell 

Those statistics are significant to physicians and researchers. 

For Tim, they mean something much more personal. 

He dreams about earning his CNA certification and nursing license. He hopes to help other patients living with sickle cell disease, drawing on his own experiences as someone who has spent nearly his entire life around hospitals and nurses. Maybe most importantly, he wants the chance to do ordinary things without worrying whether they will trigger a medical crisis.  

One memory stands out. 

Several years ago, Tim finally saw the ocean for the first time during a trip to Florida. Excited to experience it up close, he rolled up his pant legs and stepped into the water. The sudden temperature change triggered one of the worst pain crises of his life. 

After treatment, he hopes for a different outcome. 

"I want to go back to the ocean," he said.  

For most people, walking along a beach is an ordinary experience. For Tim, it represents something much bigger: freedom from a disease that has shaped every chapter of his life. 

As the first Children’s Mercy patient to begin gene therapy for sickle cell disease, Tim is helping open a new chapter not only for himself, but also for the patients and families who will follow. And for the first time in a long time, the future he imagines is defined less by hospital admissions and pain crises and more by possibility. 

Stop by to connect with our team, learn more about our nationally recognized pediatric programs, and discover how we’re advancing care, research and education to improve the health of children. While you're there, be sure to check out our sponsorship of the popular Puppy Play Pen, where attendees can enjoy some much-needed puppy cuddles and a fun break between sessions. We look forward to meeting pediatric providers, trainees and colleagues from across the country in San Diego.

View full details and agenda here.

There’s a special sense of community at Children’s Mercy Kansas and the College Boulevard Clinics, where children and families are cared for like neighbors.

Expert pediatric care comes with familiar faces, helping hands and meaningful moments. That blend of skill, compassion and connection makes our Kansas campus a special place to receive care and to work.

A bit about the location and services

As Kansas’ only freestanding pediatric hospital, Children’s Mercy Kansas connects families with nationally recognized services right here in their community. Employees support patients and their loved ones through the inpatient unit, 24/7 Tom Watson Emergency Department, outpatient surgery, more than 30 specialty clinics, laboratories and medical imaging services. Their knowledge and compassion help each child feel safe and supported.

That work strengthens the entire health system. By serving children with lower-acuity needs, staff expand access to timely pediatric treatment in Kansas while preserving capacity at Adele Hall for those who need the highest levels of specialized care. It’s a vital contribution that takes every role working together.

Children’s Mercy Kansas Fast Facts

• Home to 553 employees at CMK and 108 at College Blvd.
• Kansas’ only freestanding pediatric hospital.
• Home to an inpatient unit and the 24/7 Tom Watson Emergency Department.
• Offers outpatient surgery, more than 30 specialty clinics, laboratory services and medical imaging.
• Helps expand access to timely pediatric treatment while preserving capacity at Adele Hall for highly specialized care.
• Growing through a $152 million, four-year expansion that includes a new Medical Office Building, expanded programs, renovated spaces and improved access.
• Known for a close-knit, collaborative culture where employees support and celebrate one another.

That reach is set to grow through a $152 million, four-year expansion. A new Medical Office Building will bring outpatient clinics together and add an Orthopedic Rehabilitation Center, Sleep Clinic, Infusion Center and more. Renovated spaces, new clinical programs, a parking deck and accessibility improvements will support collaboration and create an even better experience for those we serve.

The Kansas culture

As the campus grows, its “Better Together” spirit remains at the heart of the work. Each day, employees bring their talents, dedication and compassion to make a meaningful difference for children, families and one another.

That connection feels a lot like a small town. When a challenge arises, colleagues walk down the hall, pull up a chair and solve it together. Strong relationships help everyone respond quickly and do what’s right for those they serve.

That support extends to everyday appreciation. Whether through a shout-out during a campus huddle or a note left on a colleague’s desk, going the extra mile is noticed. At Children’s Mercy Kansas and College Boulevard, people make the difference and celebrating them is simply part of who we are.

What others say about Children’s Mercy Kansas

• We loved all the care [team members.] They were so good with [child] and had a lot of patience while conversing with her.”
• “The physicians here have wonderful bedside manner! Polite, helpful and thorough.”
• “All the nurses were very kind and patient! Thankful for everything they did while we were there to make us feel comfortable.”
• “Everything was explained very well, and all our questions were answered.”
• “Would definitely recommend this hospital if any of my friends are in need of help for their kids.”
• “Such a clean, comfortable and well-kept location. And close.”
• “Love that they have a [facility] dog on staff.”
• “Always a warm welcome there! The volunteers are super helpful and love that the playrooms have goodies.”
• “All was taken care of. Overall great experience with all the dedicated staff and with all the love and care they rendered toward my child.”

Learn more about Children's Mercy Kansas.

Named in honor of  Girish Shirali, MD, a pioneering pediatric cardiologist and beloved mentor, the symposium continues a tradition of fostering learning, collaboration and innovation among healthcare professionals dedicated to improving outcomes for children with heart disease.

Attendees will have the opportunity to hear from leading experts from across the country, engage in discussions on emerging clinical practices and research and connect with colleagues from a variety of disciplines involved in cardiovascular care. The program is designed to provide valuable insights for physicians, advanced practice providers, nurses, trainees and other healthcare professionals who care for patients with congenital and acquired heart conditions.

In addition to a robust educational agenda, the symposium offers opportunities for networking and collaboration with peers who share a commitment to advancing pediatric cardiovascular medicine.

"We're excited to once again welcome experts from across the nation to Children's Mercy for this signature event," said the Heart Center planning team. "The symposium continues to be an important opportunity to learn from one another, share new ideas and strengthen connections that ultimately benefit the patients and families we serve."

Event Details:

Dates: October 19-20, 2026

Location: Big Slick Auditorium, CMRI

Find more information on the Children's Mercy CloudCME website.

Hotel accomodations:

Sheraton Kansas City Hotel at Crown Center (0.4 miles)
2345 McGee Street, Kansas City, MO.
Phone: (816) 841-1000

Room block is available until block is filled.

If you choose, other nearby accommodations include:

The Westin Kansas City at Crown Center (1 mile)
Hotel Phillips (2 miles) 

Transportation:

Distance from Kansas City International Airport: 22 miles

Continuing Education Credit:

This activity has been approved for:

Children’s Mercy Hospital is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians. This activity has been approved for AMA PRA Category 1 Credit™

Register today and join colleagues from across the region and beyond for one of pediatric cardiology's premier educational events. For questions, please contact Rachel Detwiler, MHA, Senior Department Administrator.

The milestone patient was a 4-year-old child diagnosed with congenital atresia of the left main coronary artery and severe aortic valve stenosis. Following a cardiac arrest during diagnostic evaluation, she required prolonged extracorporeal membrane oxygenation (ECMO) support and later transitioned to a Berlin Heart ventricular assist device while awaiting transplantation. She was listed for transplant on June 15 and successfully underwent heart transplantation on July 22. 

This case highlights the Heart Transplant Program's expertise in managing critically ill children through the full spectrum of advanced heart failure therapies, including mechanical circulatory support and transplantation. The multidisciplinary team provides comprehensive medical, surgical, rehabilitation and psychosocial support tailored to each patient's unique clinical needs. 

Since performing its first heart transplant in 2015, Children's Mercy has continued to expand its pediatric transplant capabilities. Program milestones include the first combined heart-liver transplant in 2022, the 50th heart transplant in 2023 and now the 75th heart transplant in 2026. The program has completed eight heart transplants so far this year and continues to grow with the addition of two physicians, further enhancing access to advanced transplant care for children across the region. 

The Children's Mercy Heart Transplant Program offers evaluation and management of patients with end-stage heart failure, congenital heart disease requiring transplantation, and those who may benefit from advanced heart failure therapies, including ventricular assist devices and heart transplantation. Through close collaboration with community providers, the program strives to ensure timely referral, seamless care coordination and long-term follow-up for patients and families throughout the Midwest and beyond.

A recent patient at the Children's Mercy Partial Hospitalization Program (PHP) illustrates how a multidisciplinary, individualized approach can help children with complex neurobehavioral needs develop effective coping skills and achieve meaningful progress.

The patient, an elementary school-aged child diagnosed with ADHD and Disruptive Mood Dysregulation Disorder (DMDD), experienced intermittent but severe behavioral escalations that affected functioning at home and school. Despite receiving outpatient therapy, case management, medication management and educational supports, symptoms continued to escalate, ultimately leading to an emergency department visit. A referral to the PHP followed.

The child was admitted to the program's Complex Neurobehavioral Unit (CNBU), which provides specialized treatment for patients whose behaviors place themselves or others at risk. The program is designed for children who require a higher level of support than traditional outpatient services can provide. Treatment plans are individualized and delivered by a multidisciplinary team that may include psychiatrists, pediatricians, psychologists, therapists, board-certified behavior analysts and behavioral health technicians.

Upon admission, the patient demonstrated difficulty tolerating frustration, particularly during academic tasks, limited ability to wait for reinforcement or attention, and challenges with emotional regulation. The treatment team focused on reducing unsafe behaviors while building adaptive coping strategies, communication skills and replacement behaviors. Frequent behavioral monitoring and short feedback cycles helped the patient develop greater self-awareness, self-control and tolerance of frustration.

The team incorporated the patient's interests into treatment to increase engagement and motivation. Through consistent skill-building and individualized supports, the patient learned techniques such as deep breathing, self-monitoring, taking breaks and verbalizing emotions and needs. Over time, he demonstrated improved emotional regulation, greater independence and increased ability to identify triggers and evaluate his own behavior.

A key component of care was close collaboration with the patient's family. Daily review of behavioral data and ongoing caregiver involvement helped reinforce treatment strategies across settings. Prior to discharge, the PHP team coordinated with the patient's school to support a successful transition and maintain consistency in behavioral expectations and interventions.

Following discharge, the patient continued outpatient follow-up with the PHP team, transitioning from weekly to less frequent visits as progress was maintained. According to the care team, the skills developed during treatment have translated into improved functioning at home, at school and in the community. The patient's family also reported increased confidence in managing transitions and advocating for needed supports.

For providers caring for children with significant emotional and behavioral challenges, the PHP offers an option for patients who require intensive, structured intervention but do not need inpatient psychiatric hospitalization. The program aims to help patients build practical coping skills, improve emotional regulation and support long-term success across environments.

For more than a decade, Stephanie Kukora, MD, neonatologist and Director of Scholarship in the Bioethics Center, has partnered with colleagues at St. Paul’s Hospital Millennium Medical College (SPHMMC) in Addis Ababa to help expand neonatal training opportunities and quality improvement efforts. Through collaborations with the Vermont Oxford Network and nonprofit partners, the team supported the development of a neonatal intensive care fellowship program that is helping strengthen specialized neonatal care capacity across Ethiopia. 

More recently, Anik Patel, MD, FAAP, Pediatric Emergency Medicine physician and Global Health Associate Program Director, has contributed to the development of Ethiopia’s first Pediatric Emergency Medicine-Pediatric Intensive Care (PEM-PICU) fellowship at SPHMMC. The program has already graduated its first cohort of fellows, representing a significant milestone in pediatric critical care training within the country.

The partnership also includes participation by Ethiopian physicians in Children’s Mercy’s Pediatric Bioethics Certificate Program. During a recent visit to Addis Ababa, Drs. Kukora and Patel collaborated with local physicians through educational sessions, bioethics seminars and simulation-based training. The visit provided opportunities to strengthen existing relationships, support trainees and exchange clinical expertise with colleagues working in resource-constrained environments. 

Clinicians from both institutions report that the collaboration has become a valuable source of shared learning. Ethiopian physicians bring extensive experience caring for critically ill children despite equipment and supply limitations, while Children’s Mercy faculty contribute expertise in neonatal medicine, emergency care, critical care education and bioethics. These exchanges have helped identify innovative approaches to clinical practice, quality improvement and resource stewardship. 

As the partnership continues to grow, Children’s Mercy faculty are exploring additional opportunities for multidisciplinary engagement, including expanded educational exchanges involving nursing, respiratory therapy and other clinical specialties. Ongoing collaborative work in neonatal medicine, pediatric critical care and bioethics is expected to continue through virtual teaching, trainee participation and joint scholarly activities. 

This international collaboration reflects Children’s Mercy’s commitment to advancing pediatric specialty care through education, mentorship and global partnerships while supporting the development of sustainable training programs that improve outcomes for children worldwide.

Karli, who was diagnosed with cerebral palsy in early childhood, presented with significant lower-extremity muscle tightness, spasticity and toe walking that affected her mobility. After undergoing multiple interventions over the years, her family sought evaluation at Children's Mercy's multidisciplinary spasticity clinic, where specialists from pediatric neurology, physical medicine and rehabilitation, and pediatric neurosurgery conducted a comprehensive assessment to determine the most appropriate next steps.

The clinic's collaborative model allows specialists to evaluate patients together in a single visit, facilitating shared clinical decision-making and development of individualized treatment plans. Karli's care team included Rose Gelineau-Morel, MD, pediatric neurologist; Sathya Vadivelu, DO, pediatric rehabilitation medicine physician; and Paige Haynes, MD, pediatric neurosurgeon. 

Following multidisciplinary evaluation, the team determined Karli was an appropriate candidate for selective dorsal rhizotomy, despite being older than the typical age range for the procedure. Providers cited her motivation, maturity and ability to participate fully in the rigorous postoperative rehabilitation process as key factors supporting candidacy. SDR is a neurosurgical procedure that reduces lower-extremity spasticity by selectively sectioning sensory nerve rootlets that contribute to abnormal muscle tone. 

The procedure was performed by pediatric neurosurgery in partnership with rehabilitation medicine specialists, who provided intraoperative monitoring to help identify appropriate nerve rootlets for treatment. Following surgery, Karli transitioned directly into Children's Mercy's inpatient rehabilitation program, allowing for early initiation of intensive therapy without delays in care. 

Postoperative rehabilitation included several weeks of inpatient therapy followed by a coordinated transition to outpatient rehabilitation services closer to home. This integrated approach supports continuity of care while enabling patients and families to remain connected to local support systems during recovery. 

Karli demonstrated strong engagement throughout rehabilitation and experienced meaningful functional gains following treatment. Within months of completing inpatient rehabilitation, she was participating in recreational activities including swimming, basketball and soccer and entered high school with improved walking ability and confidence in her mobility. 

For patients with cerebral palsy and persistent lower-extremity spasticity, comprehensive evaluation by a multidisciplinary movement disorders and spasticity team can help identify candidates who may benefit from advanced interventions such as SDR. Careful patient selection, coordinated surgical care and intensive rehabilitation remain critical to achieving optimal outcomes.

During her stay on the Liver and Renal Unit, Jenna formed a connection with Liv Golden, BSN, RN, who was serving as a nurse technician at the time and is now a registered nurse on the same unit. Through clear communication, emotional support and thoughtful efforts to help normalize the hospital experience, Liv helped Jenna and her family navigate an understandably stressful period. 

Jenna’s family described how Liv took time to explain procedures in age-appropriate ways, engage Jenna in conversations about shared interests and support not only the patient, but also her parents. Small acts of compassion, from helping make a hospitalization feel less intimidating to checking on caregivers’ well-being, left a lasting impression. 

Nearly a year after Jenna’s hospitalization and recovery, her family surprised Liv during her nursing pinning ceremony at Pittsburg State University to thank her for the care she provided. The moment highlighted the enduring relationships that can develop when clinical excellence is paired with empathy and family-centered support. 

At Children’s Mercy, multidisciplinary teams recognize that outcomes extend beyond diagnosis and treatment alone. Meaningful communication, caregiver support and compassionate nursing care are critical components of the patient and family experience, helping children and families feel seen, supported and empowered throughout their care journey.

Throughout his care, Connor developed strong relationships with the clinical teams who supported him through treatment, hospitalizations and recovery. While his medical journey included radiation therapy and ongoing oncology care, Connor credits the compassion and dedication of Children's Mercy nurses and care teams as some of the most meaningful aspects of his experience. 

Connor's story also underscores the value of integrated supportive services. Working with Children's Mercy Art Therapist Katie Brewer, MS, LPC, ATR-BC, he transformed a radiation treatment mask into a Spider-Man-inspired work of art using his own 3D printing skills. The project provided an opportunity for creative expression, helping him process his experiences while reinforcing identity, resilience and self-efficacy beyond his cancer diagnosis.

Art therapy is one of many supportive care resources available to patients at Children's Mercy. These services can help children and adolescents navigate the emotional and psychosocial challenges of serious illness, reduce anxiety and foster coping strategies throughout treatment. Connor's experience demonstrates how comprehensive pediatric cancer care extends beyond treating disease to supporting the whole child and helping patients thrive throughout their health care journey and beyond. 

As he moves into adult care, Connor continues to embody the resilience that has defined his journey, offering encouragement to other patients facing health challenges: “Never, never give up. Always believe in yourself.”

In welcoming attendees, symposium leader David Sutcliffe, MD, emphasized the value of bringing together a diverse group of transplant specialists to share knowledge, spark new ideas and strengthen partnerships that ultimately benefit transplant patients and families. 

The symposium featured a robust educational agenda focused on the evolving landscape of transplantation and the importance of multidisciplinary collaboration. Steven Webber, MBChB, MRCP, delivered the keynote presentation before joining a distinguished lineup of experts from Children’s Mercy and beyond.

A highlight of the morning was the opening plenary session, How Did We Get Here, Where Will We Go?, featuring David Sutcliffe, MD, Will Gibson, MD, Ryan Fischer, MD, Amy Gallo, MD, and Judith VanSickle, MD. The discussion explored the progress made in transplantation and opportunities for future innovation and growth. 

Attendees also participated in a multidisciplinary panel discussion, Navigating Life After Transplant: Building Seamless Transitions Together, which brought together nurses, social workers, physicians and a transplant patient to discuss the critical role of coordinated care as patients transition from pediatric to adult services. The session highlighted the value of teamwork and partnership in supporting patients throughout their transplant journey. Additional presentations examined the broader needs of transplant patients and families, including palliative care involvement in solid organ transplantation, the psychological effects of transplantation and approaches to goals-of-care conversations and symptom management.

The afternoon focused on organ donation and the powerful stories that inspire the transplant community. Lori Markham presented The Changing Landscapes of Donation, while Danica Stewart shared The Family Perspective of Organ Donation and Transplantation, offering attendees a deeper understanding of the lasting impact donation has on patients, families and care teams. 

The inaugural symposium concluded with a social gathering that allowed attendees to continue conversations, build new professional relationships and strengthen existing collaborations. The Brendan Tripp Elam Transplant Center team hopes the connections made and ideas shared during the event will continue to advance transplant care and improve outcomes for patients and families across the region. 

"Being a part of the inaugural Transplant Symposium was an honor! We have been planning for this for more than two years and to see it come to fruition was great. We were able to reach multiple organ teams and multidisciplinary staff throughout the hospital. In the future, I think we would love to expand our regional reach and hope to continue to grow the symposium bigger and better each year. I am so proud of the work we did and am honored to be on the planning committee " said Katelyn Walser, RN, BSN, CPN, CCTC.

As the Brendan Tripp Elam Transplant Center and Midwest Transplant Network look ahead, the success of the inaugural event demonstrated the value of bringing together experts from across disciplines to share knowledge, learn from one another and further a shared commitment to the transplant community.

Now 2 years old, Lilly has successfully undergone the first two stages of surgical palliation for HLHS and continues to receive ongoing care through a close partnership between Children’s Mercy Kansas City and Children’s Mercy Wichita. Her care team includes pediatric cardiologist Rabah Daoud, MD, who provides longitudinal cardiology follow-up in Wichita, allowing much of her routine cardiac monitoring and advanced imaging to occur closer to home. 

The expansion of Children’s Mercy Wichita is helping improve access for regional families managing complex pediatric conditions. With enhanced specialty services, advanced cardiac imaging capabilities and coordinated care pathways, patients like Lilly can receive comprehensive follow-up care locally while still benefiting from the expertise and resources of the Ward Family Heart Center in Kansas City. 

“Lilly has successfully completed the first two stages of her surgical repair and is now awaiting her third,” Dr. Daoud said. “Thanks to the comprehensive cardiac imaging and specialized care now available here in Wichita, she has been able to receive the advanced testing and close follow-up she needs much closer to home.” The ability to identify concerns early, provide timely intervention and reduce the burden of travel has helped support Lilly’s progress and quality of life. 

Lilly’s journey highlights the value of integrated pediatric specialty care, demonstrating how collaboration between regional and tertiary care teams can improve access, continuity and outcomes for children with congenital heart disease throughout the region.

Born prematurely at 28 weeks with Down syndrome, Cate required early NICU support at Children’s Mercy and experienced ongoing medical and social instability, including re-hospitalizations and time in foster care.

Just before her first birthday, she was diagnosed with myeloid leukemia associated with Down syndrome (ML-DS)—a rare but highly treatable subtype of leukemia. Her care involved multiple cycles of inpatient chemotherapy over approximately six months, with close monitoring, nutritional support and frequent readmissions between treatments. 

While ML-DS carries a favorable prognosis—with reported five-year survival rates around 85%—Cate’s course underscored the importance of vigilant management and multidisciplinary care, particularly in children with underlying genetic conditions and complex social needs.

Shortly after completing chemotherapy, Cate developed severe sepsis, requiring escalation to the PICU with ventilatory support and dialysis due to acute renal failure. She experienced additional complications, including vascular compromise that resulted in partial foot amputation. Despite these challenges, Cate demonstrated remarkable resilience, ultimately recovering renal function and discontinuing dialysis.

Her recovery reflects the strength of an integrated care model, including:

• Timely escalation to critical care
• Close collaboration across oncology, intensive care, nephrology and rehabilitation teams
• Ongoing support from care management, social work and family-centered services

Equally important was the role of consistent, engaged caregivers. Cate’s foster family—Joel Koenig, MD, pediatric urologist at Children’s Mercy, and his wife Karen who later adopted her—worked in close partnership with her care team, helping provide continuity and advocacy throughout prolonged hospitalizations and recovery. 

Now cancer-free and thriving at age 9, Cate continues to receive specialty follow-up care to support her long-term health and development. 

Her journey also speaks to the lasting impact these experiences can have on providers. Her adoptive father, a Children’s Mercy pediatric urologist, now integrates his perspective as a parent into his leadership in care management and patient-family services—reinforcing the importance of whole-child, whole-family care.

Cate’s case is a powerful reminder that optimal outcomes in medically complex pediatric patients depend not only on clinical expertise, but on coordinated systems of care and strong partnerships with families at every step.

Today, Wyatt is a thriving, energetic child with the stamina to enjoy daily activities that once felt out of reach. His clinical course, however, reflects the complexity and long‑term management often required for children diagnosed with severe congenital urinary tract obstruction.

Wyatt’s care at Children’s Mercy began prenatally. At a routine 20‑week ultrasound, he was diagnosed with marked dilation of the kidneys, ureters and bladder, raising significant concerns for obstructive uropathy and postnatal renal function. He was referred to the Elizabeth J. Ferrell Fetal Health Center, where specialists in Fetal Health, Neonatology and Pediatric Nephrology collaborated closely for the remainder of the pregnancy.

For more than four months, the team provided coordinated counseling, anticipatory guidance and care planning, helping the family understand potential outcomes and prepare for postnatal interventions. Wyatt was delivered at 37 weeks and diagnosed with posterior urethral valves (PUV), a rare cause of bladder outlet obstruction in male infants.

He underwent surgical intervention within days of birth, followed by years of intensive medical management. Despite ongoing nephrology care, multiple hospitalizations and daily medications, his renal disease progressed. Ultimately, he developed end‑stage kidney disease requiring transplantation.

After 607 days on the transplant waitlist — during which several potential living donor options were explored but did not proceed — Wyatt received a directed deceased‑donor kidney transplant from a young donor in his community. The donation, made possible through the generosity of the donor’s family during their own loss, proved life‑changing.

In November 2025, Wyatt underwent kidney transplantation at The Brendan Tripp Elam Transplant Center at Children’s Mercy. Despite the surgical complexity, his postoperative course was smooth, allowing discharge within one week. The clinical improvements were rapid and sustained: increased energy, improved appetite, developmental gains and the ability to participate fully in age‑appropriate activities.

Today, Wyatt continues to do well, with stable graft function and a markedly improved quality of life. For his family, Children’s Mercy represents not only advanced clinical expertise, but also continuity, collaboration and compassion throughout every stage of care.

Wyatt’s case underscores the importance of early fetal diagnosis, multidisciplinary management and timely access to pediatric transplant services — and the profound difference this care can make for children with complex renal disease and their families.

The statement, “Update on Education for Families and Patients With Pediatric Heart Disease: A Focus on Technological Advancements, Procedures, and Transitions of Care,” was developed by nursing leaders and multidisciplinary complex care teams with extensive experience supporting children with congenital and acquired heart disease and their families. It synthesizes over a decade of emerging evidence on best practices for patient and family education across the care continuum, including procedural preparation, interstage monitoring, transitions of care, and the expanding role of digital health technologies.

The statement emphasizes a family-centered, trauma-informed approach to education and communication, underscoring the need to align clinical information with patient and caregiver understanding at each stage of care. It also highlights the critical role of interdisciplinary collaboration among cardiologists, nurses, psychologists, child life specialists, therapists, and social workers in supporting outcomes and long-term adjustment.

“Dr. Gross-Toalson and I have long shared a clinical focus on family-centered and trauma-informed care,” said Dr. Erickson, who chaired the writing group. “As we developed this updated statement, it was essential that these principles be explicitly incorporated into the evidence base.”

The original AHA statement on this topic was published in 2003. Work on the update began in early 2024. Following extensive peer review, the statement was approved through the AHA scientific review process in October 2025 and published online in March 2026.

Significance for Clinical Practice

AHA Scientific Statements are widely cited in peer-reviewed literature and are frequently used to inform clinical guidelines, grant applications, and health policy. They serve as expert consensus documents reflecting the current state of cardiovascular evidence.

“This paper provides a structured, evidence-based framework to guide cardiology providers in integrating education and well-being into clinical practice,” said Dr. Gross-Toalson. “Because cardiology appropriately emphasizes evidence-based care while tailoring decisions to individual patients, it was critical to apply that same rigor to education, communication, and family support.”

She added that families consistently identify communication with the care team as a key determinant of psychosocial adjustment. Standardizing and simplifying educational approaches—while remaining responsive to individual learning needs—may support improved long-term outcomes.

Notably, the authors extend beyond traditional educational recommendations, addressing how cognitive, emotional, developmental, and social factors influence how patients and families receive, process, and apply clinical information.

Additional perspective from Dr. Erickson is available in an accompanying AHA Science YouTube video.

Join us for an enriching day filled with networking opportunities and continuing education sessions, focusing on the transplant family perspective, the latest innovations, transition data, pharmaceutical updates, social inequities and much more.

As a designated Approved Provider by the American Board for Transplant Certification (ABTC), Children’s Mercy Kansas City grants up 6.0 Category I Continuing Education Points for transplant Certification (CEPTCs) for the offering. ABTC Approval Number: 197-69.

Register here. 

Hotel Accommodations:

• The Westin Kansas City at Crown Center
• Sheraton Kansas City Hotel at Crown Center

Booking deadline is June 25. 

We look forward to welcoming you to this groundbreaking event!

Children's Mercy presentations

James O’Brien, MD, FACS – “Topics in Surgical and Catheter-Based Therapeutic Interventions 2026” (Moderated); “Yes, I Am Going to Do That to the Heart: Creating Trust Between Patient and Surgeon”

Stacy Reynolds, CPNP-PC – “Tackling SSI’s: A Nurse-Led Solution,” “Managing Medications in Real Life,” “Beyond the Technology: When Worlds, Culture and Care Collide”

Bianca Cherestal, MD – “Group Discussion on Acute Care – Inpatient Cardiology Learning: Case Presentations and Scenarios”

Geetha Raghuveer, MD, MRCP, MPH, FAAP, FACC, FAHA – “How to Care for Your Coronaries Early in Life! Lifestyle and Prevention of Atherosclerotic Coronary Artery Disease”

Lisa Laddish, DNP, CPNP-AC, CPNP-PC - “Congenital Heart Disease and Post-Operative Management: A Master Class for Nurses, APPs, and Front-Line Clinicians,” “Managing Medications in Real Life," “Together in Innovation and Discovery: Showcasing Top Nursing Oral Abstracts” (Moderated), “Together at the Table: Breakfast With Cardiac Nursing Leaders”
 

Abstracts

• Daniel Forsha, MD, MHS, Physician, Heart Center
• Mahmoud Jaara, MD, Pediatric Fellow, GME Fellowship Education
• Ashley Valenza, Student Research Assistant, Clinical Pharmacology and Toxicology
• Tony Zunica, MD, Pediatric Fellow, GME Fellowship Education

Through learning, sharing and networking, the conference aims to equip the pediatric and congenital cardiovascular care community with tools to help patients achieve a normal quality and duration of life while supporting their families. 

Theranostics is a precision medicine approach to care that uses the same targeting system to both find disease in the body and treat it using radiopharmaceuticals. By delivering radiation directly to diseased cells, it treats disease more precisely, minimizes damage to healthy tissue, and results in fewer side effects.

This unique public-private collaboration will allow all four organizations to bring both theranostics research and treatments to adults and children in Kansas City and beyond.

Learn more here. 

“Dr. Burson has served the Springfield region as a lead pediatrician for Mercy for more than 25 years,” said Debbie Schwartzkopf, interim chief administrative officer of Children’s Mercy with Mercy. “His decades of work with patients and families, community health organizations and Mercy colleagues have shaped his perspective on the needs and strengths facing pediatric health care in the region. I am excited to welcome him to our team.”

As he steps into this new leadership role, Dr. Burson will continue to care for his current patients.

“I have been a member of the Springfield medical community with Mercy since I completed my training,” said Dr. Burson. “I am first and foremost a general pediatrician and I look forward to bringing that perspective as I work closely with leaders from both Children’s Mercy and Mercy to continue advancing a pediatric system designed around the needs of children and families.”

For more than a decade, Dr. Burson has helped guide the growth of Mercy’s women’s and children’s services, and his leadership has contributed to the development of numerous pediatric programs.

Dr. Burson also brings extensive statewide and academic experience. He has served on the March of Dimes Board of Directors at regional and state levels, supported the Missouri Immunization Coalition and taught future pediatric providers through faculty and preceptor roles with Missouri State University, the University of Missouri School of Medicine, and multiple medical and advanced practice training programs.

The Children’s Mercy with Mercy joint venture was officially launched on Nov. 1, 2025.

In mid-November, Children’s Mercy announced plans to build a new, dedicated pediatric outpatient facility in Springfield on the Mercy South campus. Construction is now underway, with an anticipated opening date in Dec. 2026.

About Children’s Mercy

Founded in 1897, Children’s Mercy is a nationally recognized, independent pediatric health system and a world leader in pediatric translational research committed to transforming the lives of children and families. With hospitals and specialty clinics across Missouri and Kansas, Children’s Mercy delivers world-class, family-centered care closer to home. As a nonprofit organization, Children’s Mercy reinvests every dollar donated into advancing pediatric medicine, supporting innovative research and training the next generation of pediatric specialists. With more than 800 pediatric experts, researchers and faculty we are dedicated to improving outcomes and creating a healthier future for all children. Children’s Mercy has earned repeated recognition from U.S. News & World Report as one of “America’s Best Children’s Hospitals” and is the first health system in Missouri or Kansas to receive six consecutive Magnet designations for nursing excellence. Thanks to generous philanthropic support and a strong volunteer community, Children’s Mercy provides hope, comfort and the prospect of brighter tomorrows to every child who comes through its doors. Visit Children’s Mercy and the Children’s Mercy Research Institute to learn more, and follow us on Facebook, LinkedIn, Instagram, Bluesky and YouTube for the latest news and videos.

About Mercy 

Mercy, one of the 15 largest U.S. health systems and named the top large system in the U.S. for excellent patient experience by NRC Health, serves millions annually with nationally recognized care and one of the nation’s largest and highest performing Accountable Care Organizations in quality and cost. Mercy is a highly integrated, multi-state health care system including 50 acute care and specialty (heart, children’s, orthopedic and rehab) hospitals, convenient and urgent care locations, imaging centers and pharmacies. Mercy has over 1,000 physician practice locations and outpatient facilities, more than 5,000 physicians and advanced practitioners and more than 50,000 co-workers serving patients and families across Arkansas, Illinois, Kansas, Missouri and Oklahoma. Mercy also has clinics, outpatient services and outreach ministries in Arkansas, Louisiana, Mississippi and Texas. In fiscal year 2024 alone, Mercy provided nearly half a billion dollars of free care and other community benefits, including traditional charity care and unreimbursed Medicaid.

Integrated emotional, cognitive and mental health support — alongside world‑class cardiac care

Children’s Mercy is proud to introduce the Heart Center Wellbeing Program, a comprehensive model of care that brings together specialized psychosocial support and neurodevelopmental services with traditional cardiac treatment — so families receive seamless, whole‑heart care from diagnosis through recovery.  

What’s new

The Heart Center Well-Being Program unites two established services — the Thrive Program and the Cardiac Neurodevelopmental (CND) Program — to strengthen provider collaboration and deliver cohesive, family‑centered support in both inpatient and outpatient settings.  

Why it matters

Children with heart conditions — and their families — benefit when emotional health, coping skills, learning and development are supported alongside medical care. By expanding access to these resources, the program helps families navigate treatment, build resilience and optimize developmental outcomes across the care journey.  

Who we serve

From prenatal diagnoses through transitions for adult congenital heart disease, the Heart Center Wellbeing Program connects patients and families to the right experts and resources at the right time, making it easier to get the help they need. 

Meet the team and explore services here. 

On Oct. 18, Children’s Mercy proudly hosted BARE (Biliary Atresia Research and Education) “On the Road,” a powerful half-day event dedicated to supporting families, patients, caregivers and medical professionals affected by biliary atresia (BA)—a rare and serious pediatric liver disease.

This event was part of BARE’s national initiative to bring education, resources and community-building directly to leading pediatric institutions. In collaboration with BARE, Children’s Mercy’s Child Life team and liver nurses created a joyful “kids camp” for patients and siblings, ensuring a welcoming and supportive environment for all attendees.

The day brought together a diverse and passionate group of participants. BARE, a national nonprofit founded by parents of children with BA, led the charge in driving research and education. Medical experts from across disciplines, including hepatology, surgery, psychology, nutrition, and transplant care, shared their insights. Among them was Jonathan Merola, MD, from Cincinnati Children’s, who presented “Frontiers in Pediatric Liver Transplantation.” Families living with BA and those who have undergone liver transplants came together with their caregivers to share experiences and build community. Advocates like Lauren Wilmer from COTA (Children's Organ Transplant Association) and Chloe Simmons from Make-A-Wish Missouri and Kansas offered valuable resources and support.

The event’s impact was felt deeply. Families gained practical knowledge on diagnosis, treatment, mental health and life post-transplant through expert-led talks and breakout sessions. Connections formed among attendees who understood the unique challenges of the BA journey, fostering emotional support and solidarity. Open dialogue between clinicians and families helped shape future care models and research priorities. By visiting individual centers like Children’s Mercy, BARE ensured its resources reached diverse communities. All BA families were invited free of charge, including those receiving care outside of Children’s Mercy.

For Children’s Mercy, hosting BARE “On the Road” was a meaningful affirmation of its leadership in pediatric liver care, while highlighting the hospital’s commitment to holistic support—clinical, emotional, and educational—for families navigating chronic illness.

BARE “On the Road” was more than an event, it was a celebration of resilience, knowledge, and community. By bringing together experts, advocates, and families, it created a space where healing and hope could thrive.

Children’s Mercy was the first to use 5-base HiFi LRS, the world’s most-advanced genomic sequencing system, in the clinical setting to accelerate diagnosis for even more patients and families.

“Children’s Mercy is a trailblazer in every sense of the word—we’re not just advancing medicine, we’re reshaping it,” said Ale Quiroga, MD, MBA, President and CEO, Children’s Mercy. “This research isn’t simply a breakthrough; it’s a blueprint for what pediatric care can and should be. By deepening diagnostic precision and elevating the care experience for families, we’re equipping our clinicians with sharper tools, faster answers and greater insight. And we’re doing it for the children who need us most.”

The study compared 235 children who received the new LRS test to more than 500 children who underwent traditional genetic testing. The results were striking:

• Higher diagnostic success: LRS identified the cause of a child’s condition significantly more often than standard testing increasing the diagnostic rate by 10% over all previous tests combined.
• Faster answers: On average, families received a diagnosis in less than a month—compared to the traditional methods, which took about 91 days on average.
• Fewer tests, less stress: LRS often replaced the need for multiple rounds of testing, reducing the emotional and financial burden on families.

“Every day matters when a child is sick and undiagnosed, and this research shows that we can deliver answers faster, with fewer tests and with greater clarity than ever before,” said Tomi Pastinen, MD, PhD, Vice President, Associate Chief Medical Officer for Clinical and Research Integration, Children’s Mercy, and corresponding author of the study. “Children’s Mercy is not just doing research—we’re integrating research with clinical care—we’re raising standards and transforming pediatric medicine.” 

The benefits of clinical LRS identified in the study are likely only the beginnings of what may be uncovered through expanded testing and further understanding of noncoding regions.

“What we’ve uncovered so far is powerful—but what’s ahead is even more promising,” said Isabelle Thiffault, MSc, PhD, FACMGG, Director of Translational Genomics, Children’s Mercy, and first author of the study. “As we continue to explore the noncoding regions of the genome and expand access to this technology, we’re opening doors to diagnoses that were once out of reach. This is how we change the future of pediatric medicine—by building a foundation for discoveries that will help generations of children to come.”

Children’s Mercy is leading the way in rare disease discovery with the use of LRS in its nationally recognized Genomic Answers for Kids (GA4K) program, a first-of-its-kind pediatric data repository to facilitate the search for answers and novel treatments for pediatric genetic conditions.

In just four years, GA4K has delivered more than 2,100 diagnoses—offering answers to families who have long searched for them. The program is fueled by the generosity of donors and the dedication of researchers, clinicians and families who believe in a future where no child’s condition goes undiagnosed.

The eastern Missouri teen’s trouble started in March 2023. “My back was hurting any time I did anything,” said Kyndall. “I didn’t even know how to explain it; it just started hurting one day.”  

Her local pediatrician ordered an X-ray and MRI, then referred her to an area orthopedic specialist who confirmed her spine was fractured. She wore a brace for 12 weeks and started physical therapy, but the pain continued. And continued. And continued.  

“Her back hurt all the time,” Kyndall’s mom, Destayne, said. “She went to volleyball practice, but she came home crying. She couldn’t sit at a desk. Something was wrong.”   

They kept pressing for answers but weren’t finding any solutions. By early 2025, Kyndall was beginning to experience numbness in her legs. They had more lab work and tests done — all negative, no cancer, no lupus — and were told to do more therapy and talk about pain management.  

“I said, ‘Absolutely not,’” said Destayne. “That’s when I started Googling ‘top spine doctors in the U.S.’ I came across Dr. Shaw’s name.”

From a web search to spine surgery 

After 2 years of pain and no progress, Kyndall’s mom searched for “top spine doctors” online and found Dr. Shaw. They booked an appointment and drove across the state to meet with him.

Aaron Shaw, DO, FAAOS, is a physician on the Orthopedic Surgery and Spine Care teams at Children’s Mercy. Destayne called Children’s Mercy, had an in-depth conversation with the Spine Care team and scheduled an appointment for Kyndall with Dr. Shaw. They drove 4.5 hours to get to Kansas City in late April. 

“She'd been through a lot: 2 years of chronic back pain and still trying to push through it to help her team,” said Dr. Shaw. “I think she's very selfless in that approach.” But no kid should have to suffer pain to play the sports they love. They started talking about Kyndall’s options.  

“He was super nice and told me a lot about all the steps we could take to fix it,” said Kyndall.  

“He sat with us and actually listened to us,” agreed Destayne. That same morning, the team was able to get Kyndall a CT scan, which showed what they had suspected.  

"She had stress fractures on both sides of her spine, but they hadn’t healed — and didn’t really look like they were going to heal,” said Dr. Shaw. 

Stress fractures can occur in young, active people, especially athletes who use repetitive hyperextension movements. “Certain athletes are more at risk: gymnasts, volleyball players and football players, especially the linemen,” explained Dr. Shaw. Most of these stress fractures will heal on their own with rest and therapy. But sometimes, like in Kyndall’s case, they just ... don’t.  

“We talked about all the things you could do, which is what she had already been through,” said Dr. Shaw. “If we can avoid surgery, that's always the goal. But when we're getting to the point where they’re unable to do the things that they enjoy, and it's starting to affect quality of life and mental health, we have a good team that can help take care of those patients.” 

Kyndall was a good candidate for a spine surgery technique developed by Children’s Mercy Section Chief John T. Anderson, MD, and Richard Schwend, MD, FAAP, FAOA, who recently retired from Children’s Mercy. Dr. Shaw and other spine surgeons here perform 10 to 15 of these procedures a year to help young athletes like Kyndall. They place a screw across the area to stabilize the fracture, clean out any scar tissue that has formed, then place a bone graft material to stimulate healing. 

“We’ve had really good success with this particular technique,” said Dr. Shaw.

Pain-free for the first time in 2 years 

Kyndall had spine surgery and stayed overnight at Children’s Mercy to recover. Local patients often have this surgery outpatient, but her team didn’t want her travel home — a 4+ hour car ride — right after surgery.

Kyndall had the 3- to 4-hour surgery in mid-May. KC-area patients can have this surgery outpatient and go home the same night. But Kyndall stayed overnight to be better prepared for a long car ride home. She and her family raved about the inpatient care she received, including the team members who took the time to braid her hair.  

“They were just there, and anything we needed or wanted, they got,” said Destayne. “It was truly an amazing experience.”  

Dr. Shaw checked on her repeatedly until she was cleared to go home, in a pillow-filled car with detailed instructions for pain medicines, planned stops and making the drive as comfortable as possible.  

Kyndall was blunt about her recovery: It was painful and hard for several weeks. “Then about 3 weeks after surgery, I stopped having pain,” said Kyndall. “I was confused because I've always had the pain there!”  

“It was the first time she’d been pain-free for a day in over 2 years,” said Destayne.  

It was a new experience for Kyndall, who was excited to get back to normal teen life. “I just wanted to go shopping every day,” laughed Kyndall, teasing her mom. “She wouldn’t take me.”

“Listen to your kids” 

Technically, shopping isn’t prohibited post-surgery, but intense sports are. 

“For the first 6 weeks after surgery, we don’t want patients to stress the area,” said Dr. Shaw. “We want the body to do its work, heal and grow that bone. No bending, lifting or twisting — just take it easy.”  

After 6 weeks, patients can start increasing activity as they work on strengthening their core and lower back muscles and increasing their flexibility. 

Slowly, Kyndall began to get back to the activities she loved this summer, including volleyball camp, even though she wasn’t able to participate fully. She has continued physical therapy with a local therapist and has follow-up visits with Dr. Shaw both in-person and virtually. She reports her back is feeling “amazing”! 

Kyndall’s back is now feeling “amazing,” and she’s looking forward to a school year full of volleyball, friends and sporting events.

Kyndall’s advice for other teenagers with chronic back pain? “Go see Dr. Shaw,” she laughed. “No, really, just find a good doctor and make sure you’re doing all your physical therapy. And don’t push yourself too hard!”  

“Trust your kids and their bodies and what they’re saying,” added Destayne. “Listen to your kids. Get a second opinion if you need to. She would not be where she is today without that.” 

Kyndall is playing for her 8th grade volleyball team now and hoping to play with the travel volleyball team that supported her through her spine surgery journey. She’s looking forward to a no-pain school year, including being able to do things with her friends, enjoy sporting events and maybe even run track in the spring.  

“Kyndall is an amazingly resilient young lady,” said Dr. Shaw. “I'm very proud of the recovery that she's made so far and look forward to seeing what she's going to do in the future.”

Whether your child needs routine care or highly specialized treatment, our pediatric experts are here to help. From tiny newborns to growing teens, we’re committed to improving outcomes and making every experience as comforting and supportive as possible.

Why families choose Children’s Mercy:

• Pediatric care designed just for kids.
• Specialists trained in the unique needs and development of children.
• A focus on family-centered care and emotional support.
• Innovative treatments backed by research and compassion.

These rankings are proof of our parents’ confidence in choosing Children’s Mercy. But for us, it’s not about the accolades or the numbers — it’s about making a difference in the lives of children and families across our region and beyond.

This clinic treats children with elevated pressure in the blood vessels connecting their heart and lungs. Families benefit from consulting both cardiology and pulmonology specialists simultaneously and are supported by a team of physicians, a nurse practitioner and a nurse coordinator who helps manage the child's complex needs, from medication pre-approvals to arranging in-home equipment.

“One of the greatest strengths of our program is the partnership between cardiology and pulmonology. Dr. Birnbaum from cardiology is our program director, and Dr. Singh from pulmonology is our co-director. This collaboration truly sets us apart as a PH program,” said Kate Carp, RN, PH nurse coordinator. “We conduct joint inpatient rounds and outpatient clinics, allowing both specialties to maintain ongoing communication and collaboration in providing quality, seamless care. Families appreciate the convenience of consulting with two specialties in one visit, ensuring everyone is aligned on the plan for their child.”

Brian Birnbaum, MD, PH program director, emphasized the importance of patient-centered care in seeking RCP accreditation, “Accreditation helps us provide the strongest clinical care to our patients and families. We aim to provide excellent PH care, and accreditation helps to exemplify that. It lets our patients see that we want to remain at the forefront of developments in the pediatric PH world.” Kate Carp, RN, added, “On a practical level, seeking accreditation has motivated us to improve PH-related education for staff and establish written guidelines and protocols reflecting the specialized care we provide.”

The accreditation was awarded based on a variety of factors, including the quality and depth of resources available for expert care of PH patients and the array of therapies offered by the center.

As Dr. Kyler explains, children with asthma and obesity have worse outcomes and poorer response to medications used to treat asthma symptoms, such as systemic corticosteroids (SCS), compared to lean children with asthma. Currently, the pharmacologic mechanisms underlying these differences and their associations with obesity aren’t well understood.

The goal of this study is to find out how having obesity affects how asthma medicine works in the body. To do this, Dr. Kyler and her team are comparing how the medicine moves through and affects the bodies of kids with asthma who have obesity and those with an average weight.

“It’s important to grow our understanding of obesity-related differences in inflammation and drug processing and response during asthma attacks. My hope is to improve steroid safety and response for these kids through obesity-focused dose optimization," said Dr. Kyler.

This K23 grant will support Dr. Kyler on her path to becoming an independent clinician scientist in the field of pharmacokinetic (PK) modeling and pharmacodynamic (PD) biomarker development for children with asthma and obesity.

Mentors on the study include Bridgette Jones, MD, MSCR, Clinical Pharmacology & Toxicology, and Elin Grundberg, PhD, Genomic Medicine Center.

“This work will position Dr. Kyler to become a leader in the field of precision therapeutics for children with asthma and obesity, able to link pharmacologic translational science to the bedside for performance of pediatric clinical trials,” said Dr. Jones and Dr. Grundberg.

In this new role, Dr. Wagner will lead the development of a world-class translational cardiovascular research program, building on the strengths of the Children’s Mercy Research Institute (CMRI) while exploring new opportunities for innovation and collaboration. His responsibilities include:

• Oversight and execution of all Heart Center research operations
• Mentorship and training of research staff and future cardiovascular scientists
• Building strategic partnerships with regional institutions (e.g., KUMC, Stowers Institute)
• Securing funding to ensure long-term program sustainability

 “I first came to Children’s Mercy as a third-year medical student rotating on the cardiology ‘Blue Team’ service. From that day forward, I was hooked and knew I wanted to become a Pediatric Cardiologist,” says Dr. Wagner, “Almost 20 years later, I am honored and humbled to cultivate another great program in the Heart Center that will improve the lives of our cardiac patients.”

Dr. Wagner’s vision includes establishing three distinct research tracks:

1. Exploratory and Pre-Clinical Research
2. Clinical and Diagnostic Trials
3. Cardiovascular Outcomes Research

About Dr. Wagner

Dr. Wagner is the Matson Family Endowed Professor in Cardiac Research and Professor of Pediatrics at the University of Missouri-Kansas City School of Medicine. He joined Children’s Mercy in 2013 and has served as Division Director of Clinical Pharmacology and Toxicology for more than five years. His research has significantly advanced the understanding of pharmacogenomics and precision medicine in pediatric cardiology.

He has led and contributed to numerous NIH-funded studies and national trials, including FUEL and FALD, and has received over $4.5 million in external funding. Dr. Wagner is also the principal investigator of the IMPROVE-FALD program and director of the Cardiology Pharmacogenomics Repository (CPR).

Since 2013, the Children’s Mercy Super Q Express Clinic — held at both Children’s Mercy Hospital Kansas and the Adele Hall Campus — has helped patients with 22q11.2 deletion syndrome and 22q11.2 duplication. The multidisciplinary clinic coordinates care for patients and families affected by these complex and unpredictable genetic conditions. Instead of going to multiple appointments per month, patients see all their subspecialists in one day. 

“We are really the premiere site for 22Q patients in the Midwest, serving five states (Missouri, Kansas, Nebraska, Arkansas and Oklahoma) and are generally getting one to two new referrals a month,” 22Q Clinic founder Max Feldt, DO, Endocrinology, 
said. “We’ve been very fortunate to build the necessary resources and team members.”

Gaige, now a teenager, was diagnosed with 22Q11.2 deletion as a newborn at Children’s Mercy. “With the clinic, they all get together, talk about him as a whole person and are able to coordinate his care,” said Heather, Gaige’s mom. “We 
appreciate the way Gaige is treated: as an individual. It’s an amazing group, and Gaige really enjoys seeing the doctors.” 

As an infant, Rachel was diagnosed through Children’s Mercy genetic testing and has traveled with her family from Wichita to attend the 22Q Clinic since it opened. “It has been incredible what the 22Q Clinic has done for our daughter’s care,” said Rachel’s mom, Katie. “We’re grateful for the staff and the support network and would encourage anybody who has a child with 22Q to get plugged into this clinic. It’s a different level of care. Honestly, it’s just like a family.” 

David is one of the clinic’s many patients who make the trip up from Arkansas. “With multiple body systems affected, I believed we needed an interdisciplinary clinic where doctors can talk to each other,” said David’s dad, Ningning. “Children’s 
Mercy has a clinic that coordinates everything. Without those doctors, we wouldn’t have guidance to move forward. The growth of the clinic will be very helpful for kids and allow doctors to provide a more holistic picture of the patient’s condition.”

• 100% 1-year patient survival post heart transplant
• Third best overall survival rate in the country from listing
• Regional leader in ventricular assist device (VAD) patient volume
• Successful transplantation of single-ventricle patients at all three stages of repair: Norwood, Glenn, Fontan
• Shorter average length of stay for patients: 12 days (National average: 21 days)

Heart transplant volumes

The number and types of pediatric heart transplants performed at Children’s Mercy between 2019 and 2024 (fiscal year).

Heart transplant survival rates

Patient Survival
1/1/2021 – 6/30/2023 for 90-day and 1 year
7/1/2018 – 3/12/2020 and 6/13/2020 – 12/31/2020 for 3 year

Children’s Mercy Kansas City has outstanding pediatric heart transplant patient and graft survival while caring for complex patients at all ages. The Scientific Registry of Transplant Recipient (SRTR) tracks outcome metrics including graft and patient survival. Access the SRTR outcomes. 

Heart recipient age

Scientific Registry of Transplant Recipients

The age of patients who received pediatric heart transplants at  Children’s Mercy between 2018 and 2022.

Heart transplant by diagnosis

Scientific Registry of Transplant Recipients

The diagnoses of patients who received pediatric heart transplants at Children’s Mercy between 2019 and 2024 (fiscal year).

Dr. Shannon Carpenter has accepted the role of Division Director of Hematology, Oncology and Bone Marrow Transplant, effective immediately. Dr. Carpenter earned her undergraduate degree in Biochemistry from the University of Virginia and her medical degree from Virginia Commonwealth University. Following pediatric residency at Cincinnati Children’s Hospital, she completed her pediatric hematology/oncology fellowship at Duke. Prior to joining Children’s Mercy in 2008, Dr. Carpenter was a faculty member at the University of Texas – San Antonio. Dr. Carpenter has been an Associate Division Director in Hematology/Oncology/Bone Marrow Transplant and Section Chief of Hematology since 2013. She stepped into the role of interim Division Director of Heme/Onc/BMT in September 2024.

Thank you to the members of the search committee for all their efforts on behalf of the DOP and Children’s Mercy: Committee Co-Chairs Drs. Sherwin Chan (Radiology) and Tarak Srivastava (Nephrology), and committee members Drs. Ghufran Babar (Endocrine), Todd Bradley (Genomic Medicine Center), Denise Bratcher (GME), Andrea Carver (Heme/Onc), Aileen Har (Gastroenterology), Tomoo Iwakuma (CMRI), Karen Lewing (Heme/Onc), and Doug Myers (Heme/Onc).

Children’s Mercy Kansas City is thrilled to announce a comprehensive $152 million expansion plan for Children’s Mercy Hospital Kansas (CMHK) aimed at enhancing patient care and improving access to existing services and new clinical programs. 

“As the only freestanding pediatric hospital in the state of Kansas, this expansion will allow for even more families to have access to world-class care closer to home,” said Alejandro Quiroga, MD, MBA, CEO & President, Children’s Mercy. "We're really excited about this project because it shows how dedicated we are to giving the best care possible to our community. By bringing all our outpatient services together, improving our hospital facilities, and creating a modern, connected campus, we're making sure we can meet the growing needs of patients and families in the region.” 

The first phase of the project, already underway, is the expansion of surgical services which includes the renovation of the Sterile Processing Department and the addition of new pre-op, post-op and procedural spaces. Construction is set to be complete by summer 2026. 
 
“We have teams of nationally recognized surgeons and proceduralists. The addition of new procedures rooms and expanded facilities will allow CMHK to meet the growing demand for procedural services in specialties such as ENT, Orthopedics and Gastroenterology,” said Shawn St. Peter, MD, Senior Vice President, Surgeon-in-Chief, Children’s Mercy.  
The expansion project also includes: 

• New Medical Office Building (MOB): A state-of-the-art facility to consolidate outpatient services currently spread across multiple locations, providing a seamless and connected experience for patients and families. The MOB is scheduled to open in the Summer of 2027. 
• Hospital Renovations: Upgrades to the hospital space vacated by the consolidation of outpatient services, enhancing the overall patient and staff experience. These renovations are expected to be completed by the Summer of 2029. 
• New Service Offerings: Introduction of new programs such as Inpatient Rehab, Intensive Outpatient Programs, an expanded GI Motility program and a Neuroscience Center of Excellence. 
• Parking and Exterior Updates: Modern parking structures, new entrances, and exterior improvements to facilitate better wayfinding and accessibility. (Parking and Emergency services will still be accessible throughout construction.) 

“We listened to the needs of physicians and families in this community and the expansion of our Kansas hospital strengthens our capacity to serve the growing pediatric population,” said Amy Fallon, MPH, PhD, President of Regional Operations, Children’s Mercy. “This is more than just adding rooms or advancing technology; it’s about deepening our commitment and reaffirming our dedication to delivering top-quality, accessible health care to every child that needs it.”  

The community is invited to be part of this initiative. Philanthropic gifts will advance this renovation to improve the health of all children and families, bringing expert care closer to home.
 

Taylor’s doctors thought she had placenta previa, a condition where the placenta covers the cervix. The doctors mentioned the baby might also have a hole in her spine — spina bifida — but a second opinion was needed. They recommended that Taylor see a specialist at Children’s Mercy to perform an amniocentesis — a test that checks for genetic or chromosomal conditions using a sample of amniotic fluid surrounding the baby. It was soon confirmed that baby Rose had fluid buildup in the brain (hydrocephalus) and spina bifida.

“Everything just felt like it moved so fast,” recalled Taylor. “All of it was just one big blur, but I remember that we had to move fast so that we didn’t miss the cutoff for doing a surgery in utero.”

With amniocentesis test results in hand, the Elizabeth J. Ferrell Fetal Health Center (FHC) assembled a team of experts to help support Taylor each step of the way as she weighed her options. “I sat down with surgeons, doctors, nurses, all the different departments including social services and billing — even the mental health services.”

That type of collaborative care is a hallmark of the FHC, which provides advanced care for high-risk pregnancies before, during and after delivery. Taylor and James eventually planted new roots in Kansas City thanks to the care they received here. Their time spent at CM inspired Taylor to join the Fetal Health Center Patient Family Advisory Committee (PFAC).

“I was so grateful that the CM team acted immediately,” said Taylor. “Sometimes, waiting can be too late. It’s something we’ve talked about recently in a PFAC meeting.”

While James was still commuting back and forth from Atlanta, Taylor relied on support from her mother, who lives in Kansas City. The team of reinforcements from CM helped everything come together. Before surgery, Taylor visited CM twice a week to check baby Rose’s progress by measuring her size, heartbeat and fluid to determine whether a ventriculoperitoneal (VP) shunt was needed to help move excess fluid out of her brain into her abdomen.

In the moments before fetal surgery, Taylor sat with a CM chaplain and felt comforted by the care and attention given to both her and her unborn child. “It was my first surgery — ever!” she said, recalling her nerves. “They were very understanding and very patient with me. My nurses, Sarah and Christina, were fantastic with making sure that me and baby Rose were well taken care of during and after in-utero surgery.”

For FHC nurses like Sarah Rock, BSN, RNC-OB, CNOR, C-EFM, the admiration goes both ways. “I am always humbled by our fetal health center patients,” Sarah said. “So many times, our patients are going through possibly the worst time in their lives, but they show so much grace and optimism.

“James and Taylor continually showed up with bright spirits, optimism and grace. James drew this creative drawing that showcased the different aspects of labor and delivery and the Fetal Health Center. We still proudly display his work in our unit!”

Taylor holds a special place in her heart for her surgical team as well. “I had great surgeons,” she said. “CM really helped with perfecting the surgery so that more children with Rose’s diagnosis can have the opportunity to walk.”

The in-utero surgery was successful, but Taylor still had one more to go. Her C-section was scheduled for Dec. 24. It was a day she proudly remembers, as Rose was born gift-wrapped inside her amniotic sac.

“Sarah and Christina came in on Christmas Eve when they didn’t have to and I was extremely grateful,” said Taylor. The care team reminded her: it’s ok not to be ok.

“It was very comforting, especially with delivering during COVID and not having a lot of people able to be there with me. It was very hard doing back-to-back surgeries, but I had a great speedy recovery.”

From Sarah’s perspective, coming into work an extra shift on Christmas Eve was a “no-brainer.” “I am very thankful that our leadership supports our nurse/patient relationships, making it an option for me to be a part of this very special Christmas miracle!” she recalled.

Another element of Taylor’s village of support was a mentor from Parents Offering Parent Support (POPS). Although no two kids are exactly alike, the POPS program connects CM families with others who have experience facing similar health challenges for encouragement and advice.

Today, Rose’s spine has healed and she walks without assistance. Taylor is an advocate for helping her daughter and other kids navigate and embrace their differences. She said, “Whether your differences are seen or unseen, they need to be talked about and explained to kids.”

She thanks the Kreamer Resource Center for being a helpful reference point for having those conversations with Rose’s classmates and teachers.

“Unfortunately, in a lot of communities that I’m in, I don’t know of an adult who has spina bifida or another child of color who has spina bifida,” said Taylor. “I had to force myself to stand outside of the situation and realize there is not going to be another case like Rose because she is the only “Rose” with this certain hole on her spine. There is no other Rose that has this VP shunt.”

Rose is now a bubbly 4-year-old who loves trying backflips and being a compassionate cheerleader for others. You’d never know she was on her fifth surgery at CM. She has also picked up her parents’ creative skills and loves to draw.

“She is such a giving and thoughtful person who won’t hesitate to give a stranger a hug,” Taylor explained. “I’ve had to learn to kind of let that go because she would hug people and they would immediately start crying, with uplifted emotion.”

In their journey with Rose, Taylor and James are now paying it forward. They created a non-profit called Better Outcomes Foundation that helps provide resources and care packages to at-risk expecting parents. The foundation’s parent-centered services offer nine months of comfort — with a goal of improving mental health and increasing positive outcomes for baby and mom in the healing process.

“We realized that most of the care and attention just goes to mom, but mom’s care support person also needs resources,” said Taylor. “Sometimes that support person is just as drained, afraid or scared. We need to make sure both are ok and cared for throughout this process so they can continue to support one another and baby.”

Taylor credits the entire team at CM for helping her family through what can be an overwhelming and scary process. Her advice for other families going through it: “Make sure you take the time to find a team that you feel comfortable with. Have people in the room who are very knowledgeable and patient and willing to answer. You’re trusting them with your life and your child’s life.

 “I do not know where or how Rose would be today if it wasn’t for the team at Children’s Mercy — they are amazing,” Taylor said.

Listen here.

As Children’s Mercy Research Institute (CMRI) grows its research programs, the institute has welcomed many innovative, early-career investigators to its roster of researchers. These investigators bring their novel ideas, unique talents, and diverse interests to the CMRI. The following is a profile on one of the emerging principal investigators.

Ayanda Chakawa, PhD, Developmental and Behavioral Health, is both a clinical psychologist who provides behavioral health care to youth and a researcher interested in improving access to that care.

She is particularly interested in behavioral health care access issues for youth from historically marginalized populations. This includes those from Black, Indigenous, and People of Color (BIPOC) groups, socioeconomically disadvantaged backgrounds, and those from families with limited English language proficiency.  

Dr. Chakawa leads the ACCESS (Adolescent and Child Centered Engagement to Supports and Services) Behavioral Health Lab. The lab team studies how embedding behavioral health care into primary care clinics impacts access. They also study the impact that referring patients to other Children’s Mercy behavioral health clinics has on accessing care. In 2020, Dr. Chakawa, Leslee Belzer, PhD, Developmental & Behavioral Health, and Trista Perez Crawford, PhD, published an article based on their research supporting a multi-tiered integrated behavioral health care model to meet the needs of underserved sociodemographic groups. Dr. Chakawa, Dr. Belzer, Dr. Crawford, and Hung-Wen Yeh, PhD, Health Outcomes and Services Research, also published study results that indicated racial disparities in the use of telehealth for integrated primary care visits during COVID-19. Their research helped support a return to in-person integrated primary care clinical appointments.

PATHWAY project

Through her ACCESS Lab research and clinical experience at Children’s Mercy, Dr. Chakawa observed that many families faced challenges trying to navigate the behavioral health care system; this was even more pronounced for families whose child received a referral to another clinic for care. She wanted to address these challenges. In 2021, Dr. Chakawa received the Children’s Mercy Katharine Berry Richardson (KBR) award. With the KBR award funding, Dr. Chakawa began her research project, the PATHWAY (Promoting Access To Healthy Well-Being Across Youth) Behavioral Health Program, to address barriers to care by developing a culturally-relevant behavioral health care navigation model.

In 2022, Dr. Chakawa was named the first Diversity, Equity, and Inclusion (DEI) Scholar in the Children’s Mercy DEI Scholars Program. The goal of this program is to study the lack of diversity and inclusion in health care and the impact of health disparities on patients and families. Through the DEI Scholar award, Dr. Chakawa expanded research staffing for the PATHWAY program and engaged parent consultants as research partners to inform the development and implementation of the PATHWAY program. “The DEI Scholar award has provided increased protected time for me to research and build a clinical intervention that I’m passionate about,” said Dr. Chakawa. “I get to use my professional strengths to address a DEI-related public health crisis.”

The PATHWAY study involves piloting an individualized intervention to support families with behavioral health care navigation. Study participants are parents of youth referred to behavioral health services through primary care. The participants are from predominantly BIPOC and socioeconomically disadvantaged backgrounds and speak English or Spanish. The intervention follows participants for 13 months and includes surveys and care navigation check-ins. During the check-ins, the study team conducts motivational interviews to clarify referral information, help set goals to obtain care, and assess family confidence in meeting those goals. “This study has created space for families to explain and for us to assess what the actual barriers are. Then, we can address these barriers,” said Dr. Chakawa. “Patient-family education is a huge component in this process.”

Community engagement

From development to implementation, community engagement is the foundation for this study. Dr. Chakawa partners with community members from the Children’s Mercy Mental Health and Consejo de Familias Latinas y Hispanas Parent Family Advisory Councils (PFACs). In addition to the PFACs providing consultation, four parents from the PFACs form a designated community advisory board to the PATHWAY study. These parents include Emily Hamm and Jamila Weaver from the Mental Health PFAC and Brenda Quintana and Bertha “Alicia” Aldape from the Consejo de Familias Latinas y Hispanas PFAC. “The community members involved provide feedback at every phase of the study,” said Dr. Chakawa. “Their voices are there, and that is a critical component of our study.”

The community members bring their personal experience to the study. “Dr. Chakawa’s research sheds light on something families have known for so long - behavior health system navigation is challenging and complex,” shared the PATHWAY study community advisory board. “We love the collaborative effort that has gone into the project and the opportunity to be part of the change. We hope more research is on the horizon. If the PATHWAY project is any indication of where the direction of mental health care access is going, we know families of the future will be grateful for what we are doing today.”  

Trends

Although preliminary data is limited since the PATHWAY study is still ongoing, Dr. Chakawa has noticed some trends, including issues with system-level barriers like clinic paperwork requirements or communication issues that affect the navigation process. Another data trend is that social determinants of health impact both access to care and study engagement. These determinants include economic stability, education access, health care access, neighborhood environment, and social/community environment.

Dr. Chakawa’s long-term goal is to scale up the PATHWAY intervention to include more care navigation check-in opportunities for participants and to include staff dedicated to behavioral health care navigation throughout the health care system. She is applying for external grant funding to expand the study and its intervention.

In addition to her role at Children’s Mercy, Dr. Chakawa is an assistant professor at the University of Missouri-Kansas City School of Medicine. She joined Children’s Mercy in 2017 as an intern and then completed her fellowship in psychology in 2019. She received her PhD in clinical psychology from Auburn University, an MA in developmental psychology from the University of Alberta (Canada), and her BSc in psychology (with counseling emphasis) from Oakwood University, a historically black college/university in Huntsville, Alabama.

Take a listen here.

Three Children’s Mercy Kansas City disciplines — neonatology, fetal cardiology and maternal fetal medicine — are collaborating with hospitals across North America to answer an important question: How does delayed umbilical cord clamping impact the neurodevelopment of babies with congenital heart disease (CHD)? 

Research has established that delaying cord clamping for 30 to 45 seconds or longer benefits both preterm and full-term newborns. Delayed cord clamping can: 

• Preserve the baby’s red blood cells. 
• Reduce the need for transfusions. 
• Improve iron levels, which reduces the risk of anemia and boosts brain development. 
• Decrease the risk of necrotizing enterocolitis. 
• Decrease the risk of brain bleeds. 
• Improve neurological and developmental outcomes. 

Newborns with CHD have been excluded from previous cord-clamping studies because of their complex health variables, including how blood volume affects newborns with cardiac issues.  “The reason they were excluded before is the reason we need the study now,” explained Laura Vricella, MD, FACOG, Medical Director of the Elizabeth J. Ferrell Fetal Health Center. 

“The already established benefits of delayed cord clamping may certainly be favorable to newborns with CHD,” said Maria Kiaffas, MD, PhD, Director of Fetal Cardiology, who outlined some of the hypotheses the study will be able to test.

 “Newborns with certain types of CHD (i.e. cyanotic) do benefit from a robust amount of red blood cells, and the ones that will need surgery may need less transfusions when they start with a good hematocrit. In addition, the decreased risk of necrotizing enterocolitis, a common complication in newborns with CHD, can favorably affect their morbidity and length of hospitalization.” 

“These kids are known to be at higher risk for neurological developmental issues,” said John Daniel, MD, MS, Neonatal ECMO Director. “The brain may receive less oxygen as a fetus in certain CHD types, and then newborns live in a persistently hypoxic state. They often go on cardiopulmonary bypass to have their operations. There are a lot of risk factors.” 

The new multi-site study, “CORD-CHD: Cord clamping among neonates with Congenital Heart Disease,” is led by Carl H. Backes, Jr., MD, from Nationwide Children’s, with Dr. Kiaffas and Dr. Daniel serving as site Principal Investigators at Children’s Mercy. The study is funded through the National Institutes of Health’s National Heart, Lung and Blood Institute. 

The study randomizes participating families into two groups: One where cords are cut after a delay of 30 to 60 seconds and one where cords are cut between 60 and 180 seconds following delivery. Patients will have neurodevelopmental follow-ups at 3-4 months, 9-12 months, and 22 to 26 months of age. The study will recruit participants for five years and run until 2030. With a total sample-size goal of 600, Children’s Mercy is tasked with enrolling 5 to 10 families per year. 

“We’ve sailed past our recruitment goals,” said Cheri Gauldin, BSN, CCRC, Senior Research Nurse Coordinator, Children's Mercy Research Institute. The team started recruiting participants in February and has already enrolled 11 families and completed 10 deliveries — the most of any participating site so far. 

When the team identifies a potential candidate (around 40% of the center’s deliveries have some form of CHD), they explain the study to the family at an interdisciplinary care meeting. Most families have been familiar with delayed cord clamping, either from previous births or by talking to their obstetricians, and have been eager to participate. 

“The Fetal Health Center has done an amazing job,” said Cheri. “They are being very proactive with helping us make sure everybody knows who’s enrolled.”  With the cooperation of today’s Fetal Health Center families, the innovative study will ensure future patients get the best evidence-based care possible. 

“This study wouldn’t be possible without the center,” agreed Dr. Daniel. “As our center continues to grow, we wanted to introduce research that bridges the gap between neonatology and maternal fetal medicine. This is a truly multidisciplinary study, and our Fetal Health Center is uniquely positioned to offer it to families.” 

Learn more about our Heart Center, Neonatology and Fetal Health Center at childrensmercy.org.

 

The Department of Orthopedic Surgery and Musculoskeletal Science at Children’s Mercy Kansas City performs approximately 100 surgeries to improve function for cerebral palsy patients each year, utilizing the single-event multilevel surgery (SEMLS) approach.

SEMLS incorporates several procedures into one surgical intervention. For example, bilateral proximal femoral varus derotational osteotomies are often combined with other lower extremity procedures. Doing so reduces the number of surgeries and hospitalizations for these patients, streamlining their postoperative care, reducing length of stay and helping patients advance to rehabilitation faster.

In pediatric hospitals where the team is highly skilled in SEMLS, outcomes have been very good and patient/family satisfaction superior.

Improving Pain Management for SEMLS Surgery

Epidural catheters are currently the gold standard when cerebral palsy patients undergo bilateral multilevel orthopedic surgery.

However, one of the challenges for those who receive an epidural is that it must remain in place postoperatively, usually for two days. This often requires the patient to be immobile, affecting the physiology of recovery, including lung function and mobility. A longer hospitalization also affects the family dynamic, requiring parents to take more time away from work or other caregiving responsibilities. Additionally, patients may have comorbidities that preclude epidural catheter placement.

This patient population also presents with additional medical challenges, including the fact that many are taking anti-spasmodics, may be on a baclofen pump, or may be on anti-epileptic medications.

To improve upon the patient’s experience, Kathryn Keeler, MD, Children’s Mercy Pediatric Orthopedic Surgeon, and Nichole Doyle, MD, Pediatric Anesthesiologist, collaborated to develop an Enhanced Recovery After Surgery (ERAS) protocol.

ERAS Protocol for SEMLS Patients

The ERAS protocol Dr. Doyle and Dr. Keeler developed is used for cerebral palsy patients undergoing bilateral lower extremity surgeries. 
Key elements of the protocol include:

• Use of peripheral nerve blocks instead of epidural catheters.
• Earlier mobilization of patients, with an earlier start to physical therapy.
• Discontinued use of the Foley catheter before arrival to the post-anesthesia care unit.
• Use of dexmedetomidine infusion for the first postoperative day as part of a multimodal pain management plan.

To determine the effectiveness of the protocol, they performed a pilot study of 10 consecutive cerebral palsy patients undergoing bilateral lower extremity surgery from May to July 2020, comparing them with 10 controls from 2017 to 2019. The groups were matched by Gross Motor Function Classification System (GMFCS) score, procedure performed and age. The biggest difference between the two groups was in the type of pain control used. The control group received epidural catheters.

The ERAS group received peripheral nerve blocks in the operating room with clonidine or dexamethasone added to prolong the blocks based on the surgical procedures planned.

Preoperatively patients were counseled about the protocol and what to expect. Some high-risk patients were started on gabapentin at this visit. On the day of surgery families were instructed to continue clear liquids until two hours before surgery. If gabapentin was not started at the preop visit, patients were given a dose preoperatively.

Intraoperatively, the ERAS patients received intravenous (IV) acetaminophen and ketorolac. They also had intraoperative dexmedetomidine and/or ketamine infusions. A short-acting narcotic was given as needed.

At completion of the surgery the Foley catheter was removed. Upon transfer to the floor, the patients continued on a dexmedetomidine infusion 0.1 mcg/kg/hr-0.2mcg/kg/hour and they were placed on scheduled acetaminophen, ketorolac, gabapentin and a bowel regimen. They received valium, narcotic, ondansetron and diphenhydramine as needed. If needed, a PCA/NCA was started.

Key Outcomes

This pilot study showed that following an ERAS protocol can help minimize the variation of care and enhance the patient and family experience with bilateral lower extremity surgery. Results showed a decreased length of stay of almost a full day and a reduction in postoperative narcotic consumption for the ERAS group. Side effects such as nausea, vomiting and pruritus also decreased in the ERAS patients.

Overall, performing single-shot peripheral nerve blocks at the beginning of the surgery has the potential to substantially increase the value of care delivered to this complex patient population, but further study in a larger patient population is needed to confirm results.

ERAS Pilot Study Highlights

• 10 ERAS protocol patients; 10 control group patients.
• GMFCS 4 for both groups.
• Decreased postoperative narcotic use in ERAS group to 0.12 morphine equivalents/kg per day compared to 0.19 morphine equivalents/kg in control group.
• Postoperative need for a PCA/NCA pump was zero in the ERAS group compared to four in the control group, two of which occurred after discontinuation of the epidural catheter.
• Time to postoperative discharge from physical therapy was 1.70 days in the ERAS group versus 2.44 days in the control group.
• Hospital length of stay was 2.20 days for the ERAS group versus 3.0 days in the control group.

Collaborate with us to study the ERAS protocol

Children’s Mercy is seeking to study the ERAS protocol in a larger patient population by collaborating with other pediatric institutions performing single-event multilevel surgeries for cerebral palsy patients. To work with our team, contact:

Kathryn Keeler, MD, Pediatric Orthopedic Surgeon 
kkeeler@cmh.edu

(816) 234-3696

Nichole Doyle, MD, Pediatric Anesthesiology 
nmdoyle@cmh.edu

(816) 234-3464

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

Reference:

1. Schendel DE, et al. (n.d.). Public Health Issues Related to Infection in Pregnancy and Cerebral Palsy. PubMed - NCBI. National Center for Biotechnology Information. Retrieved 9/18/2020 from: https://www.ncbi.nlm.nih.gov/pubmed/11921385.

Almost 1 in 13 American children (7.6%) have IgE-mediated food allergies (FA). The standard treatment for FA is avoiding allergenic foods, but alternative items can be hard to find and afford. Around 21% of children with FA also experience food insecurity (FI), or a household’s inability to acquire food due to lack of money or resources.

FI disproportionately affects Latinx, Native American and Black communities and has been rising during the pandemic. The impacts of FI on children include higher rates of asthma, obesity, academic trouble, behavior issues, anxiety, depression and more. The combination of FA and FI is dangerous: Children with both FI and FA are at greater risk of anaphylaxis than are their peers.

Shroba Heads AAAAI Food Insecurity Work Group

Because of the increase in FI, the American Academy of Allergy, Asthma and Immunology (AAAAI) FI Work Group surveyed allergists’ FI knowledge and practices. The survey report was published January 2022 in The Journal of Allergy and Clinical Immunology: In Practice.¹

Jodi Shroba, APRN, MSN, CPNP, is the lead author and chair of the AAAAI FI Work Group. Her FI experience includes serving on the Children’s Mercy Hunger-Free Hospital Council and the medical advisory board of the Food Equality Initiative.

Food Insecurity Knowledge and Screening

In the fall of 2020, the work group surveyed a random sample of AAAAI members. COVID-19 fatigue may have played a part in the survey’s low response rate (6.3%), but it’s possible that lack of FI knowledge kept members from responding. If so, the survey may overestimate the FI knowledge base. Most respondents were allergists, female and white and worked in urban or suburban areas. The lack of diversity in the respondent sample may also skew results, especially because FI has had a greater impact on underrepresented communities.

The survey found 71.2% of respondents didn’t know if their FA patients experienced FI within the past six months. Only 24.5% routinely screened for FI. According to recent research, this suggests that fewer allergists screen for FI than do physician practices (30%) and hospitals (40%).² Sixty-six percent of respondents were aware of general food banks in their area, but only 5.7% knew of allergen-free pantries, and 28.3% reported not being aware of any available FI resources.

Barriers to Screening

The most common barrier to FI screening was lack of knowledge (70%), followed by not having resources to give food-insecure patients (40%). It is also possible that FI assumptions keep allergists from screening: 27.8% said they didn’t think FI was an issue for their patients, and 63.9% reported their patients have never discussed struggling to afford safe foods.

Sixty percent of respondents were interested in implementing FI screenings. They requested logistical support — FI conversation tips and validated screening questionnaires — and FI resources, like pantry lists, pamphlets and digital materials.

Guidance on Implementing Screening

Responding to this interest, the work group offered the following FI screening advice:

1. Prepare: Learn about FI. In addition to its own report, the group recommends the FI toolkit from the American Academy of Pediatrics (AAP).³
2. Select a screening tool and method: Annual, universal screening will decrease provider bias and stigma. The most validated clinical screening tool is the Hunger Vital Sign (see below).
3. Gather resources: Compile FI resources and highlight FA-sensitive ones. (See the full study for a starter list.) Federal FI programs include the Supplemental Nutrition Assistance Program (SNAP), the Special Supplemental Nutrition Program for Women, Infants, and Children (WIC), and the Emergency Food Assistance Program, but none have specific provisions for families with FA.
4. Get Involved: Create or join an FI group. For example, the Children’s Mercy Hunger-Free Hospital Council (HFHC) is a multidisciplinary team that works to improve the health care system’s response to FI, which is consistently identified as a priority issue in the hospital’s community health needs assessment. Initiatives supported by the HFHC include the Kids Eat Free summer food service program, fresh produce distribution and the community garden. Representation from the FA community lends a valuable perspective to the council, helping to ensure that programs to address FI are inclusive and equitable.

Future Areas of Study

More research is needed to understand the impact of FI on FA patients. Because FI has a greater impact on Latinx, Native American and Black communities, the work group would like to see increased diversity in FI and FA study samples. Future studies could include the impact of socioeconomic status on FA, whether specific FAs could be red flags for FI, and whether adding an FA question to the current Hunger Vital Sign tool would be helpful.

Learn more about food allergies and food insecurity

Jodi Shroba, MSN, APRN, CPNP
Food Allergy Program Coordinator, Allergy, Asthma and Immunology
jashroba@cmh.edu
(816) 960-8885

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

1. Shroba J, Das R, Bilaver L, et al. Food insecurity in the food allergic population: a work group report of the AAAAI Adverse Reactions to Foods Committee. J Allergy Clin Immunol Pract. 2022;10(1):81-90. doi:10.1016/j.jaip.2021.10.058

2. Fraze TK, Brewster AL, Lewis VA, Beidler LB, Murray GF, Colla CH. Prevalence of screening for food insecurity, housing instability, utility needs, transportation needs, and interpersonal violence by US physician practices and hospitals. JAMA Netw Open. 2019;2(9):e1911514. Published 2019 Sep 4. doi:10.1001/jamanetworkopen.2019.11514

3. Screen and Intervene: A Toolkit for Pediatricians to Address Food Insecurity. Developed by the American Academy of Pediatrics (AAP) and the Food Research & Action Center (FRAC). January 2021.

In its 18^th consecutive year of funding from the National Institutes of Health (NIH), the Chronic Kidney Disease in Children (CKiD) study continues to provide groundbreaking and novel findings with an incredible impact on children with chronic kidney disease (CKD). Children’s Mercy Kansas City is one of two lead centers—and among more than 50 participating nephrology programs throughout North America—that follows children in the study  longitudinally and studies  a variety of outcomes associated with their CKD status.

Likewise, Children’s Mercy is a leader in the International Pediatric Peritoneal Dialysis Network (IPPN), SCOPE, NAPRTCS and other collaboratives advancing the understanding and treatment of pediatric kidney disease.

Bradley Warady, MD, Division Director of Pediatric Nephrology; Director, Dialysis and Kidney Transplantation and Professor of Pediatrics, University of Missouri-Kansas City School of Medicine, is Co-Principal Investigator of the CKiD and IPPN projects, and shares key clinical findings from seven collaborative research and quality initiatives involving the Children’s Mercy Pediatric Nephrology team and national/international colleagues in 2021.

1. The impact of blood pressure on CKD progression

A CKiD study analyzing longitudinal ambulatory blood pressure monitoring (ABPM) data from 679 children¹ confirmed that children who exhibited poor control of their mean arterial blood pressure also had more rapid progression of CKD, with a different pattern in those with glomerular versus non-glomerular disease. The importance of this study is that it addressed a modifiable risk factor for CKD progression. Whereas there are risk factors that cannot be altered, such as gender and genetics, blood pressure is a factor that can be controlled in most cases, and as this study revealed, can potentially exert a significant influence on the progression of CKD.

2. Socioeconomic factors disproportionately impact cardiovascular health in African American children with CKD

Another CKiD study demonstrated some cardiovascular risk factors, specifically high blood pressure and high left ventricular mass index, are influenced by socioeconomic status.² The study looked at data from more than 600 children, and the African American population was disproportionately affected by adverse socioeconomic factors such as maternal health, food insecurity, home income or insurance status. This study, which was featured as one of the best articles of the year by the American Journal of Kidney Diseases, demonstrated how social determinants of health can adversely affect both children and adults with CKD.

3. Development of a new estimating equation for kidney function

Clinicians use glomerular filtration rate (GFR) as a measure of kidney function, and over the years, there have been a variety of different equations—and a number of different variables—used to estimate it. By using data from 928 participants in CKiD, the study team published new estimating equations for individuals with CKD from age 1 through 25 years.³ The average of the creatinine and cystatin-based equations, without race coefficients, yield unbiased estimates of GFR. These updated equations are vital to closely and accurately monitor the progression of CKD in patients and are now being shared and adopted throughout the worldwide nephrology community.

4. 14 years of patient outcome data published, informing global care

This year, the IPPN published information regarding key factors that influence the morbidity and mortality of the global pediatric PD population.^4,5 In addition to reviewing important clinical management and outcome data collected over the course of the registry pertaining to nutritional status, anemia, CKD-MBD, preservation of residual kidney function and growth, it also highlighted the significant roles that infection and cardiovascular disease play in terms of patient survival. The international scope of the registry helped assess risk factors related to the geographic location of the patient and the economic status of various regions of the world and emphasized the importance of ongoing global advocacy and support to enhance the care of all children with end-stage kidney disease (ESKD) on maintenance dialysis. .  

5. Dialysis outcomes of children  with SLE

Limited information exists on the outcome of dialysis for children with lupus nephritis (LN) compared to children on dialysis with non-lupus glomerular disease.  In a retrospective NAPRTCS cohort study⁶, children with LN were found to have a higher risk of hospitalization and to be less likely to receive a kidney transplant in the first three years after dialysis initiation. Non-white race was also associated with a lower rate of kidney transplantation. The study calls attention to the need to address  risk factors for hospitalization of children with LN,  in addition to determining the actions  necessary to help achieve equitable access to transplantation.

6. Culture-negative peritonitis in children on PD

The identification of a causative organism in children with peritonitis helps optimize cure rates and avoid unnecessary antibiotic exposure. Unfortunately, culture-negative peritonitis is common. In a study conducted by the SCOPE collaborative⁷ based on 5.5 years of observation, 27% of peritonitis episodes were culture-negative. Most importantly, a survey of participating sites revealed marked variability of effluent sampling and culture techniques, a possible contributing factor to the high culture-negative rate. The findings point to an opportunity to improve standardization of PD effluent evaluation  using quality improvement methodology, as is now being carried out by SCOPE, and ideally improve organism detection.    

 7. Quality improvement initiatives can improve vein preservation over a child’s lifetime

Hospitalized children with CKD frequently have intravenous (IV) lines placed which can cause long term injury to their vasculature. For those children whose CKD worsens over time and who ultimately require the use of dialysis, damaged vasculature can hinder or prevent the development of an arteriovenous fistula (AVF) for the performance of hemodialysis, an important clinical burden during childhood and into adulthood.

An institutional quality improvement initiative and subsequent publication from Children’s Mercy featured a call to “Save the Vein.”⁸ It identified and implemented the steps necessary to prioritize IV-line placement in the dominant arm and preserve vasculature in the non-dominant arm. Whereas only 47 percent of children were having IVs placed in the preferred arm at baseline, by the end of this quality improvement project, it rose to 94 percent. The project demonstrated how dissemination of appropriate education and auditing of clinical practice can help improve the quality of care over a child’s lifetime.

Focusing on quality of life for kids with CKD

“The primary goal for these studies is to benefit and support children with CKD—to improve their outcomes and reduce their risk for additional burdens as they age,” says Dr. Warady. “Recognition of key risk factors for poorer outcomes, followed by prompt and successful targeted interventions, has the potential to lessen those risks and purposefully improve kids’ quantity and quality of life. Children’s Mercy will continue to work with colleagues in  CKiD, IPPN, SCOPE, NAPRTCS and other significant collaborative research efforts with a goal to continually improve the care, management and outcomes of children who have CKD and ESKD.”

References

1)  Dionne JM, Jiang S, Ng DK, Flynn JT, Mitsnefes MM, Furth SL, Warady BA, Samuels JA; CKiD study group. Mean Arterial Pressure and Chronic Kidney Disease Progression in the CKiD Cohort. Hypertension. 2021 Jul;78(1):65-73.

2)  Sgambat K, Roem J, Brady TM, Flynn JT, Mitsnefes M, Samuels JA, Warady BA, Furth SL, Moudgil A. Social Determinants of Cardiovascular Health in African American Children With CKD: An Analysis of the Chronic Kidney Disease in Children (CKiD) Study. American Journal of Kidney Diseases. 2021 Jul; 78(1):66-74.

3)  Pierce CB, Muñoz A, Ng DK, Warady BA, Furth SL, Schwartz GJ. Age- and sex-dependent clinical equations to estimate glomerular filtration rates in children and young adults with chronic kidney disease. Kidney International. 2021 Apr; 99(4):948-956.

4)  Borzych-Dużałka D, Schaefer F, Warady BA. Targeting optimal PD management in children: what have we learned from the IPPN registry? Pediatric Nephrology. 2021 May;36(5):1053-1063.

5)  Ploos van Amstel S, Noordzij M, Borzych-Duzalka D, Chesnaye NC, Xu H, Rees L, Ha IS, Antonio ZL, Hooman N, Wong W, Vondrak K, Yap YC, Patel H, Szczepanska M, Testa S, Galanti M, Kari JA, Samaille C, Bakkaloglu SA, Lai WM, Rojas LF, Diaz MS, Basu B, Neu A, Warady BA, Jager KJ, Schaefer F. Mortality in children treated with maintenance peritoneal dialysis: Findings from the International Pediatric Peritoneal Dialysis Network Registry. American Journal of Kidney Diseases. 2021 Sep;78(3): 380-390. 

6)  Wasik H, Chadha V, Galbiati S, Warady B, Atkinson M.  Dialysis Outcomes for Children with Lupus Nephritis Compared to Children with other Forms of Nephritis: A Retrospective Cohort Study. American Journal of Kidney Diseases. 2021 Aug; 28

7)  Davis K, Bryant K, Rodean J, Richardson T, Selvarangan R, Qin X, Neu A, Warady B.  Variability in Culture-Negative Peritonitis Rates in Pediatric Peritoneal Dialysis Programs in the United States. Clinical Journal of the American Society of Nephrology. 2021  Feb; (2): 233-240

8)   Singh NS, Grimes J, Gregg GK, Nau AE, Rivard DC, Fields M, Flaucher N, Sherman AK, Williams MU, Wiley KJ, Kerwin K, Warady BA. "Save the Vein" Initiative in Children With CKD: A Quality Improvement Study. American Journal of Kidney Diseases. 2021 Jul;78(1):96-102.

According to the Centers for Disease Control, new diabetes cases have decreased over the last decade, except in people under 20. In fact, in 2018, approximately 210,000 children and adolescents younger than age 20—or 25 per 10,000 U.S. youths—had been diagnosed with diabetes. This included 187,000 with type 1 diabetes.¹

The increasing frequency of both type 1 and type 2 diabetes in children and adolescents is a growing clinical and public health concern. Studies show long term that poor disease control at any age significantly raises the lifetime risk of serious complications, including heart and kidney disease, blindness and death.

Partners in Improving Pediatric Diabetes Care

In 2017, The Leona M. and Harry B. Helmsley Charitable Trust awarded funding to Children’s Mercy Kansas City to address these growing concerns by embarking on a grant project to achieve performance improvement in health care systems, leveraging predictive analytics technology developed by Cyft, Inc. The project’s goal was to use big data to identify high-risk youth with type 1 diabetes for more intensive intervention. The current grant extends the implementation of that work.

Mark Clements, MD, PhD, pediatric endocrinologist, Director of Endocrine/Diabetes Research, and Medical Director of the Pediatric Clinical Research Unit at Children’s Mercy Kansas City; and Leonard D’Avolio, Assistant Professor, Harvard University and Brigham and Women’s Hospital, and CEO, Cyft, Inc., are leading this project.

Also providing leadership to the current project are Sanjeev Mehta, MD, MPH, advisory committee chair, Joslin Diabetes Center; Susana Patton, PhD, CDE, director of intervention development, Nemours Children’s Health System; Ryan McDonough, DO, champion for clinical quality improvement; and several staff and faculty from the Children’s Mercy Diabetes Center and other departments across the hospital. Laurel Koester and Ben Stang from the Helmsley Charitable Trust serve as program officers who engage in ongoing conversations with the team on all aspects of the project.

Leveraging Technology to Rapidly Learn

Initially, the project used the large volume of data captured from routine care of youth with type 1 diabetes, such as the free text notes found throughout electronic health records (EHRs). By combining EHR data with multiple other data sources — such as diabetes self-management device data and patient-reported outcomes data — the researchers were able to develop robust predictive models for clinically important outcomes. They focused on two preventable outcomes: deteriorating blood glucose (A1c) control, and hospitalization for diabetic ketoacidosis. Having robust models to predict these two outcomes gave the researchers important targets for improvement.

From this foundation, Dr. Clements, Dr. D’Avolio and colleagues have now developed a nascent initiative called the Rising T1DE Alliance. The idea is to bring researchers and diabetes centers with similar goals together to learn how to scale innovations based on the immense amount of data generated during routine diabetes care, and to rapidly test innovative approaches to care that hold promise for improving outcomes for these youth.

Grant Supports Creation of the Rising T1DE Alliance and its Rapid Learning Approach

Based on the success of the initial project, Children’s Mercy recently received $9.75 million in funding from the Helmsley Charitable Trust to support the launch and scaling up of the Rising T1DE Alliance and its proactive, rapid approach over the next three years. The goals of this funding cycle — which will continue leveraging the predictive analytics technology developed by Cyft — are to 1) apply patient- and family-centered design to the development and implementation of novel behavioral, mHealth and care delivery interventions, 2) test multiple interventions for effectiveness in improving each predicted outcome, 3) predict new clinically relevant outcomes, 4) test new data types and sources for their ability to improve outcome predictions, 5) evaluate the cost-effectiveness of various approaches, and 6) disseminate findings.

Once novel interventions have been tested and validated, the team’s learnings can be shared via playbooks with other clinics. With this approach, other clinics will have access to the details necessary to implement an intervention and/or predict an outcome, thereby saving time and resources and leading to better decision-making.

Current interventions being tested include:

• Remote patient monitoring, which involves frequent sharing of diabetes data and frequent video or audio micro-visits via telehealth. The goal is to deliver timely and personalized interactions, facilitating just-in-time discussions regarding current disease control and self-management.
• Text messaging interventions to nudge behaviors and support communication between families and the health care team.
• Behavioral economic interventions that incentivize youth for positive behaviors that promote health. The researchers think this may be of interest for youth with diabetes who are predicted to have a rise in their A1c because it may help them better manage insulin at meals. This is a first-of-its-kind intervention.
• Just-in-time diabetes education based on the real-time changes in a young person’s glucose levels.
• A screening and referral program for social determinants of health.

Early Results Show Improved Outcomes

Children’s Mercy is already leveraging data science to predict outcomes and to apply quality improvement methods and implementation science to improve diabetes outcomes.

Dr. Clements has presented early results from this research at high-impact meetings, including the International Society for Pediatric and Adolescent Diabetes, and team member Emily DeWit has presented findings at the Advanced Technologies & Treatments for Diabetes conference. Upcoming data publications are also anticipated.

Highlights include:

• A validated model to predict 90-day rise in Hemoglobin A1c.
• A validated model to predict 180-day risk for hospitalization due to diabetic ketoacidosis.
• Early evidence of clinically significant risk reduction for rising HbA1c with remote patient monitoring.

Moving forward, the Rising T1DE Alliance will bring academic- and industry-based innovators together with other interested diabetes centers, benefiting patients and their families. The initiative will help researchers to better understand patient behaviors, to implement timely and appropriate interventions, to continuously monitor outcomes across large clinic populations, and to share successes that other health care professionals can implement with confidence. This, in turn, will drive better care for children and adolescents with type 1 diabetes, reducing the lifetime risk of serious complications and improving satisfaction with the overall model of diabetes care.

Learn more about Pediatric Endocrinology and Diabetes at Children’s Mercy

Mark A. Clements, MD, PhD, Rick and Cathy Baier Family Endowed Chair in Endocrinology; Director, Endocrine/Diabetes Clinical Research; Medical Director, Pediatric Clinical Research Unit

maclements@cmh.edu

(816) 960-8803

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

Reference:

1. National Diabetes Statistics Report 2020: https://www.cdc.gov/diabetes/pdfs/data/statistics/national-diabetes-statistics-report.pdf. Accessed 7/14/2020.

Approximately 2.5 million infants are born at less than 32 weeks’ gestation worldwide every year, with approximately 75,000 of them born in the U.S. Many of these preterm infants develop the severe form of lung disease bronchopulmonary dysplasia (BPD) and have poor lung function. The longer-term effects can range from prolonged oxygen therapy to increased morbidity after common lung infections.

Postnatal steroids are among the few medications shown in randomized trials to be beneficial in ameliorating BPD. But there is wide variability across the specialty of neonatology related to treating premature infants with a medication that requires practitioners to walk the fine line between safety and efficacy.

In fact, a policy statement issued by the American Academy of Pediatrics on the topic concluded, “Because available data are conflicting and inconclusive, clinicians must use their own clinical judgment to balance the adverse effects of BPD with the potential adverse effects of treatments for each individual patient. Very low birth weight infants who remain on mechanical ventilation after 1 to 2 weeks of age are at very high risk of developing BPD.”¹

To provide neonatologists with better guidance when treating these infants, researchers with the Division of Neonatology at Children’s Mercy Kansas City in the Center for Infant Pulmonary Disorders are examining several clinically important questions using a combination of internal and external funding. These questions include:

• When is the best time to treat these patients with postnatal corticosteroids?
• Which premature infants would benefit most from corticosteroids?
• How do we measure respiratory function in premature infants and determine if inhaled therapies and other therapies are working?

As an active member of the BPD Collaborative, an international consortium of 26 institutions, and the Children’s Hospital Neonatal Consortium (CHNC), the goal for these researchers collectively is to utilize sophisticated databases and testing to help answer these questions for neonatologists in the U.S. and across the globe, eventually establishing more definitive guidelines related to caring for this vulnerable patient population.

Evaluating the Timing of Postnatal Corticosteroid Treatment for BPD

In the Level IV NICU at Children’s Mercy Kansas City, there was a wide variability with regard to timing of steroid treatment among preterm infants with evolving BPD. Alain C. Cuna, MD, Neonatologist, Assistant Professor of Pediatrics, University of Missouri-Kansas City School of Medicine, noticed that some clinicians chose to treat infants who remained intubated on moderately high ventilator settings at around 2 to 3 weeks of life. Others opted to delay treatment until the baby’s lungs had more time to mature and additional efforts to wean them from mechanical ventilation had been ineffective.

To examine the association between timing of steroid treatment and outcomes in high-risk preterm infants with evolving BPD, Dr. Cuna and his colleagues conducted a retrospective cohort study of preterm infants treated with low-dose dexamethasone for BPD in the Children’s Mercy NICU.

Infants treated with dexamethasone at day of life (DOL) 14 to 28 (moderately late group) were compared to infants treated at DOL 29 to 42 (delayed group).

Fifty-five infants (25 with moderately late treatment; 30 with delayed treatment) were identified for this research. The mean age at treatment was 23 days in the moderately late group, and 35 days in the delayed group. At the time of treatment, infants treated moderately late were more likely to be on high-frequency ventilation (96% versus 47%, P < 0.0001) and had a higher fraction of inspired oxygen (71 ± 18% versus 56.2 ± 18.4%, P = 0.005) compared to infants treated later.

Despite being the sicker group, moderately late-treated infants (14 to 28 days) were discharged at an earlier corrected age compared to infants with delayed treatment (29 to 42 days). In the Children’s Mercy cohort, earlier treatment also was associated with fewer days on mechanical ventilation, and fewer days on supplemental oxygen, as well as less treatment with repeat or rescue steroids. There was also a trend toward more tracheostomy among delayed-treated infants.²

These findings led Dr. Cuna and his colleagues to conclude that among preterm infants, earlier treatment with steroids could potentially modify the disease trajectory and allow for the sicker cohort to have better outcomes compared to delayed treatment.

Validating the Children's Mercy Results Nationwide

With this first study being limited to a small cohort, Dr. Cuna and his colleagues sought to validate their results in a larger patient database. Working with the CHNC, they posed the same question regarding timing of corticosteroid treatment and outcomes, this time via a multicenter retrospective cohort study looking at data from January 2010 to December 2016 from the Children’s Hospitals Neonatal Database and Pediatric Health Information System database.

The study population included preterm infants <32 weeks’ gestation treated with systemic corticosteroids after 7 days of age and before 34 weeks’ postmenstrual age. Stepwise multivariable logistic regression was used to assess the association between timing of corticosteroid initiation and the development of Grade 2 or 3 BPD as defined by the 2019 Neonatal Research Network criteria.

Using this much larger database, Dr. Cuna and his colleagues identified 598 corticosteroid-treated infants (median gestational age 25 weeks, median birth weight 760 grams). Of these, 47% (280 of 598) were first treated at 8–21 days; 25% (148 of 598) were first treated at 22–35 days; 14% (86 of 598) were first treated at 36–49 days; and 14% (84 of 598) were first treated at >50 days. After adjusting for birth characteristics, admission characteristics, center and comorbidities, the infants first treated at 36-49 days (aOR 2.0, 95% CI 1.1–3.7) and >50 days (aOR 1.9, 95% CI 1.04–3.3) had higher independent odds of developing Grade 2 or 3 BPD when compared to infants treated at 8–21 days.

The results from this much larger database were recently published in Pediatric Pulmonology.³ The results mirrored the findings that Dr. Cuna identified in the initial retrospective study at Children’s Mercy. In short, later initiation of treatment among preterm infants with systemic corticosteroids was associated with a higher likelihood of developing Grade 2 or Grade 3 BPD when compared to earlier intervention.

Based on this data, neonatologists at Children’s Mercy implemented a quality improvement project titled, “Using quality improvement to implement consensus guidelines for postnatal steroid treatment of preterm infants with developing bronchopulmonary dysplasia.” The project goal was to achieve over 90% adherence to consensus guidelines on use of postnatal steroids (PNS) in preterm infants for BPD within six months.⁴

Dr. Cuna’s overall objective is for the data to better inform guidelines and best practices regarding the best time to administer corticosteroids to premature infants with BPD for optimal results at other NICUs.

Researchers Use R21 Grant to Study Steroid Treatment Response in Infants with BPD

Tamorah Lewis, MD, PhD, and William Truog, MD, both Children’s Mercy neonatologists, were awarded a two-year, $455,856 R21 grant from the Eunice Kennedy Shriver National Institute of Child Health and Human Development to assess the genomic and metabolomic markers that correlate with steroid response in a group of preterm infants who have evolving BPD.

Their project is called “Less Lumping, Smarter Splitting: Genomics and Metabolomics of Systemic Steroid Response in Bronchopulmonary Dysplasia.” Their work complements the research done by Dr. Cuna, helping to clarify the genomic and metabolomics biomarkers of clinical response as a mechanism to identify a group of infants with the most favorable risk/benefit ratio, thus moving the field of BPD treatment toward precision medicine.

By better understanding steroid response variability, the team hopes to move BPD therapy into the age of personalized therapeutics in this group of infants using pharmacogenomics and pharmacometabolomics. In fact, Drs. Lewis and Truog believe their research has the potential to shift the paradigm from largely unexamined 20th Century population-based dosing regimens, with little account for patient inter-individual variability, toward a precision medicine approach.

To do this, they are using contemporary tools (genomics, metabolomics) to identify infants with the greatest likelihood of therapeutic benefit with the naturally associated minimization of development of adverse events by preventing steroid exposure to infants unlikely to respond. This precision therapeutics approach addresses a large gap in current knowledge and may lead directly to improved pharmacotherapy for infants with BPD.

Children’s Mercy is serving as the principal investigator for this research. Other sites recruiting infants for this study are the Children’s Hospital of Philadelphia, Nationwide Children’s Hospital (Columbus, Ohio), Arkansas Children’s Hospital and Children’s Hospital of Los Angeles.

Accurately Measuring Pulmonary Function in Premature Infants with BPD

Another long-standing gap in the care of premature infants with BPD is the inability to accurately measure pulmonary function and response to treatment with bronchodilators.

Though there are several types of equipment and techniques that are FDA-approved for these purposes in adults and children, there are currently no lung function tests that are practically feasible, clinically meaningful and widely used in infants.

Winston Manimtim, MD, Neonatologist and Medical Director, Infant Tracheostomy and Home Ventilator Program, is supported by intramural funding from Children’s Mercy and is investigating the use of forced oscillation technique (FOT) to measure respiratory mechanics in premature infants with BPD.

FOT is a noninvasive method that employs small amplitude pressure oscillations superimposed on normal breathing and therefore has an advantage over conventional lung function techniques because it does not require the performance of respiratory maneuvers. To date, this technique is FDA-approved in adults and children but remains largely experimental in infants and newborns.

A new device aimed specifically at newborns and infants (0 to 2 years of age) called tremoflo N-100 (“Neo”) uses a facemask to measure lung function at the patient’s bedside in only a few minutes while the infant sleeps normally. It uses an adapted version of the airwave oscillometry (AOS) to calculate the impedance of the lungs and quantify airway obstruction.

Though this device has been used to evaluate respiratory mechanics in newborn and term infants, to Dr. Manimtim’s knowledge, it has not been used in premature infants who are recovering from initial respiratory distress syndrome (RDS) and are developing BPD.

The study design is a pilot prospective cohort of 20 Children’s Mercy patients who successfully complete testing over the next year. In those subjects who are already being treated with a bronchodilator as part of their BPD medical management, a post-bronchodilator FOT measurement will be done 30 to 60 minutes after treatment to compare any changes in pulmonary mechanics.

When the study is completed, Dr. Manimtim hypothesizes that FOT and this new technology may tell researchers if a medication is actually working and, if so, how well the infant is responding, further advancing the ability to individualize these therapies for maximum benefit.

Targeting the Right Baby with the Right Medication at the Right Dose and Right Time

These researchers are focusing their collective efforts on finding better ways to individualize treatment for preterm infants at the highest risk for developing BPD, prescribing the right medication at the right dose and at the right time in their development. The division is using multiple research modalities, including database studies, prospective cohort studies and new device studies to answer these important questions related to BPD therapeutics.

By doing so, they can better inform the risk/benefit ratio for corticosteroids, ensuring the infants who receive these treatments will benefit most.

Learn more about BPD research at Children’s Mercy

Alain C. Cuna, MD, Neonatologist

accuna@cmh.edu

(816) 234-3300

Tamorah Lewis, MD, PhD, Neonatologist

trlewis@cmh.edu

(816) 234-3300

Winston Manimtim, MD, Neonatologist; Medical Director, Infant Tracheostomy & Home Ventilator Program

wmmanimtim@cmh.edu

(816) 234-3300

William Truog, MD, Director, Center for Infant Pulmonary Disorders

wtruog@cmh.edu

(816) 234-3300

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

Watterberg KL, American Academy of Pediatrics. Committee on Fetus and Newborn. Postnatal corticosteroids to prevent or treat bronchopulmonary dysplasia. Pediatrics, 2010;126(4):800-808.

Cuna A, Lewis T, Dai H, Nyp M, Truog WE. Timing of postnatal corticosteroid treatment for bronchopulmonary dysplasia and its effect on outcomes. Pediatric Pulmonology, 2019;54:165–170.

Cuna A, Lagatta JM, Savani RC, Vyas-Read S, Engle WA, Rose RS, DiGeronimo R, Logan JW. Association of time of first corticosteroid treatment with bronchopulmonary dysplasia in preterm infants. First published: 11 August 2021 https://doi.org/10.1002/ppul.25610.

Hansen TP, Oschman A, Pallotto EK, et al. Using quality improvement to implement consensus guidelines for postnatal steroid treatment of preterm infants with developing bronchopulmonary dysplasia. J Perinatol 41, 891–897 (2021). https://doi.org/10.1038/s41372-020-00862-1.

Bronchopulmonary dysplasia (BPD) remains the most common complication of premature birth.^1,2 Currently, the most accepted definition of BPD is the need for oxygen or respiratory support at 36 weeks’ postmenstrual age (PMA).

Patients with severe BPD often have severe complications, including pulmonary hypertension, poor growth, long-term pulmonary morbidity, and neurodevelopmental disability.³ The term BPD is often used interchangeably with chronic lung disease of prematurity, or CLD.

Establishing a Multidisciplinary CLD Team

There are a lack of published guidelines and national standards for treating infants with severe BPD/CLD. However, the Children’s Mercy Kansas City Neonatal Intensive Care Unit (NICU), an 84-bed Level IV regional neonatal referral center, saw the need to establish a dedicated multidisciplinary CLD team to care for these infants, and was one of the first in the nation to do so.

Launched in 2010, this team follows a standardized approach to the diagnosis and management of infants with BPD/CLD who do not respond to standard therapies.

Over the past decade, this team has grown to include dedicated neonatologists supported by pediatric pulmonology; cardiology; otolaryngology; a facilitator/coordinator; primary bedside NICU nurses; neonatal nurse practitioners; respiratory therapists; a tracheostomy coordinator; clinical pharmacology; dietary; social work; psychology; a parent advisor group; occupational, speech and language therapies; and members of the infant tracheostomy and home ventilator, pulmonary hypertension and palliative care teams. This group meets biweekly to discuss each case to determine acute and long-term management.

Patient Population Characteristics

To determine whether a multidisciplinary CLD team approach can improve outcomes for this complex infant population, the Children’s Mercy Multidisciplinary CLD team recently published its experience in the Journal of Perinatology.

A total of 267 patients hospitalized from 2008 to 2018 were identified for a retrospective analysis. Patients born from 2008 to 2009 with CLD served as a reference data point but not a comparison group prior to the establishment of the CLD Multidisciplinary team in 2010.

Characteristics of this patient population:

• 56% male; 44% female
• 62.2% Caucasian; 22.8% African American; 5.7% Hispanic; 0.7% Asian; 7.5% other
• 26 weeks median gestational age at birth; mean 28.5 weeks
• 0.85 kg median birth weight; mean birth weight 1.3 kg
• 44% primary respiratory conditions (non-BPD)
• 24% extremely preterm infants (born <28 weeks’ gestation) with severe BPD
• 14% genetic conditions
• 6% cardiac anomalies
• 4% neurologic conditions
• 8% other
• Over 75% of all patients in the cohort received diuretics, systemic and inhaled steroids, and bronchodilators. Pulmonary hypertension was diagnosed in 37.2% of the patient population; 39.7% were treated with inhaled nitric oxide, and <18% required PDE5 inhibitors.⁴

Can a Multidisciplinary CLD Team Improve Outcomes?

Since 2010, mortality has improved for infants with tracheostomy cared for by the dedicated Multidisciplinary CLD team at Children’s Mercy. Most patients were categorized as having primary pulmonary and airway conditions that evolved into what is broadly considered chronic lung disease of infancy (CLDI), while one-quarter of the cohort had the strict definition of severe BPD.

The overall survival rate reported for these infants was 88.8%, with 65% of them being ventilator-dependent through a tracheostomy at the time of NICU discharge. Tracheostomy was not associated with the odds of death prior to NICU discharge. In a cohort of infants with severe BPD/CLD, secondary pulmonary hypertension was strongly associated with the need for a tracheostomy, or death, or tracheostomy and death.

Though not addressed in this study, unique to Children’s Mercy is the continuum of care that extends beyond NICU discharge for these patients via the Infant Tracheostomy and Home Ventilator Program. This neonatology-driven program, led by Winston Manimtim, MD, Neonatologist and Medical Director, is the only one in the U.S. serving as a medical home for these patients in a multidisciplinary setting with other key subspecialists, including pulmonologists, gastroenterologists and otolaryngologists.

This team’s improved survival rates in preterm infants with tracheostomy have been highlighted in other recent studies.

Improving the Odds for Technology-Dependent Infants

A recent survey done by the Children’s Hospital Neonatal Consortium found that among 31 children’s hospital NICUs, 41% had a self-designated multidisciplinary BPD team (unpublished data), indicating that more and more NICUs are realizing the benefits of implementing a consistent, coordinated multidisciplinary approach to caring for these inpatients.

This paper includes resources, such as guidelines and quality improvement projects, to help other providers investigate and develop a multidisciplinary team approach to care for this complex infant population.

To access these supplementary materials, visit https://doi.org/10.1038/s41372-021-00974-2.

Learn more about the Children’s Mercy Multidisciplinary CLD Team

Winston Manimtim, MD, Neonatologist; Medical Director, Infant Tracheostomy and Home Ventilator Program

wmmanimtim@cmh.edu

(816) 302-3592

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

Northway WH Jr., Rosan RC, Porter DY. Pulmonary disease following respiratory therapy of hyaline-membrane disease, Bronchopulmonary dysplasia. N Engl J Med. 1967;276:357–68.

Doyle LW, Anderson PJ. Long-term outcomes of bronchopulmonary dysplasia. Semin Fetal Neonatal Med. 2009;14:391–5.

Ehrenkranz RA, Walsh MC, Vohr BR, Jobe AH, Wright LL, Fanaroff AA, et al. Validation of the National Institutes of Health consensus definition of bronchopulmonary dysplasia. Pediatrics. 2005;116:1353–60.

Hansen TP, Noel-MacDonnell J, Kuckelman S, Norberg M, Truog W, Manimtim W. A multidisciplinary chronic lung disease team in a neonatal intensive care unit is associated with increased survival to discharge of infants with tracheostomy. Journal of Perinatology. https://doi.org/10.1038/s41372-021-00974-2.

The National Institutes of Health estimates that 10,000 to 15,000 babies born in the United States develop bronchopulmonary dysplasia (BPD) each year. The risk of developing BPD increases the earlier a baby is born and the lower the birth weight. Infants born weighing less than 2.2 pounds are at the greatest risk for BPD. The number of cases of BPD has been increasing, most likely because of modern advances in medicine, which have enabled doctors to keep more very low birth weight, premature babies alive than in the past.¹

Investigating Why Some Premature Babies Develop BPD

Not all premature babies develop BPD. But what makes some more susceptible to BPD than others? That’s what Venkatesh Sampath, MBBS, MRCPCH, Neonatologist and Medical Director of the Neonatal Diseases Research Program at Children’s Mercy Kansas City, wanted to know.

Dr. Sampath noted that mammalian lung development progresses sequentially, with formation of the tiny air exchange units, called alveoli, happening after birth. In preterm infants, exposure to noxious stimuli such as infection, mechanical ventilation and hyperoxia injures lung blood vessels, disrupting formation of alveoli and resulting in BPD. ^2,3,4,5

Studies of infants who die of severe BPD have revealed severe disruptions in lung vascular growth and alveolar development. Unfortunately, little is understood about the molecular mechanisms that regulate normal lung vascular growth, limiting understanding of defective vascularization in BPD.

Identifying a Key Vascular Gene Essential for Lung Vascular and Air Sac Development

Though lung development is a highly complex and regulated process, Dr. Sampath’s lab focuses on key lung vascular genes such as the delta-like 4 (DLL4) gene that are important for lung air sac development. While DLL4’s role in embryonic and retinal vascular development is known, this is one of the first studies where anyone has looked at this powerful gene and its role in lung development in both mice and humans. Based on earlier work, Dr. Sampath hypothesized that DLL4 is required during critical stages of lung development for formation of lung blood vessels and alveoli (air-exchange units).

Dr. Sampath and his fellow researchers, Sheng Xia, Heather Menden, et al., used human autopsy samples from babies who died from causes other than lung disease, to show that DLL4 is expressed in lung blood vessels and endothelial cells (EC) very early (before birth) in lung development. DLL4 expression during lung development was also confirmed in two different mouse strains using conventional methods and single-cell RNA sequencing.

Then, they used a mouse model specially bred for this research lacking one copy of the DLL4 gene. They found that the mice also expressed DLL4 early in lung development in the lung endothelial cells. Dr. Sampath’s research showed that when the mice were missing one copy of this gene, the lung’s alveolar sacs and blood vessels didn’t develop as they should, indicating how important DLL4 is to lung development.

His research showed that DLL4 serves as a major regulator of normal lung vasculature, triggering a series of actions that signal to the endothelial cells when and where to grow and form new blood vessels. The lack of DLL4 resulted in disordered lung vessel formation initially that finally resulted in fewer lung blood vessels.

Dr. Sampath also found that when DLL4 is deficient in EC, alveolar type 1 (AT1) cells, the cells that line all alveoli and facilitate gas exchange, were also reduced. This results in impaired lung growth and air sac development. In a final step, they also showed that lung EC obtained from the human developing lung also require DLL4 for normal blood vessel branching.

Study Demonstrates Role of DLL4 in Regulating Cell Growth of Endothelial and Aveolar Type 1 Cells

This is the first study to demonstrate that DLL4 regulates microvascular growth and arborization during distal lung ontogeny and DLL4 deficiency programs deviant angiogenesis, disrupting lung vascular patterning and alveolarization. These phenotypic changes occurred concurrently with altered expression signatures of lung endothelial cell fate specification and notch signaling, as well as impaired expression of AT1 lineage markers.⁶

Studies employing drug-dependent vascular endothelial growth factor (VEGF) signaling blockade or VEGF rescue in experimental models of developing lung injury demonstrated the importance of VEGF in lung development.^7,8,9 These studies postulated “the vascular hypothesis,” i.e., vascular injury in the developing lung programming defective alveolarization.⁹

Dr. Sampath’s results support “the vascular hypothesis” from a lung development perspective by indicating that DLL4, an endothelial gene, is required for distal lung morphogenesis. This research team also demonstrated that hyperoxia, a major risk factor for BPD in premature infants, inhibits DLL4 expression in the developing mouse lung and human lung endothelial cells, indicating a potential role for DLL4 in BPD.

Future Research Direction

Future studies will examine cross-talk between endothelial cell DLL4 and other cell types required for distal lung development, and the relevance of deviant DLL4 signaling to impaired vascular development in BPD affecting preterm infants. The relevance of DLL4 signaling in BPD will be investigated in preclinical models of BPD using human genetics and cadaveric samples of infants who died of BPD. The lab is also testing therapies in preclinical models of BPD that can rescue deficient DLL4 signaling and the BPD phenotype. Ultimately, Dr. Sampath and his colleagues may one day work on therapeutic targets that may increase the DLL4 gene, protecting premature infants against lung injury.

Learn more about BPD research at Children’s Mercy

Venkatesh Sampath, MBBS, MRCPCH, Medical Director of the Neonatal Diseases Research Program

vsampath@cmh.edu

(816) 234-3591

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

1. National Organization for Rare Disorders (NORD) website: rarediseases.org/rare-diseases/bronchopulmonary-dysplasia-bpd/. Accessed March 22, 2021.

2. Jobe AH, Bancalari E. NICHD / NHLBI / ORD Workshop Summary - Bronchopulmonary Dysplasia. Am J Respir Crit Care Med. 2001;163:1723–1729.

3. Morty RE. Recent advances in the pathogenesis of BPD. Semin Perinatol. 2018;42(7):404–412.

4. Menden HL, Xia S, Mabry SM, Navarro A, Nyp MF, Sampath V. Nicotinamide adenine dinucleotide phosphate oxidase 2 regulates LPS-induced inflammation and alveolar remodeling in the developing lung. Am J Respir Cell Mol Biol. 2016 Dec;55(6):767-778. doi: 10.1165/rcmb.2016-0006OC. PMID: 27438994; PMCID: PMC5248953.

5. Menden H, Xia S, Mabry SM, Noel-MacDonnell J, Rajasingh J, Ye SQ, Sampath V. Histone deacetylase 6 regulates endothelial MyD88-dependent canonical TLR signaling, lung inflammation, and alveolar remodeling in the developing lung. Am J Physiol Lung Cell Mol Physiol. 2019 Sep 1;317(3):L332-L346. doi: 10.1152/ajplung.00247.2018. Epub 2019 Jul 3. PMID: 31268348; PMCID: PMC6766719.

6. Xia S, Menden H, Townley N, Mabry SM, Johnston J, Nyp MF, Heruth DP, Korfhagen T, Sampath V. Delta-like 4 is required for pulmonary vascular arborization and alveolarization in the developing lung. JCI Insight. 2021;6(7):e134170. https://doi.org/10.1172/jci.insight.134170.

7. Thébaud B, Abman SH. Bronchopulmonary dysplasia: Where have all the vessels gone? Roles of angiogenic growth factors in chronic lung disease. Am J Respir Crit Care Med. 2007;175(10):978–985.

8. Le Cras TD, Markham NE, Tuder RM, Voelkel NF, Abman SH. Treatment of newborn rats with a VEGF receptor inhibitor causes pulmonary hypertension and abnormal lung structure. Am J Physiol - Lung Cell Mol Physiol. 2002;283(3 27-3):555–562.

9. Thébaud B et al. Vascular endothelial growth factor gene therapy increases survival, promotes lung angiogenesis, and prevents alveolar damage in hyperoxia-induced lung injury: Evidence that angiogenesis participates in alveolarization. Circulation. 2005;112(16):2477–2486.

Dr. Srivastava’s research has validated the importance of cyclooxygenase enzyme-2 (COX2), prostaglandin E₂ (PGE₂), and prostanoid receptor EP2 in the podocyte as a critical pathway that may play an important role in hyperfiltration-mediated injury.

To study this further, he was awarded a five-year R01 grant from the NIH to work on targeting prostanoid receptor EP2, one of the four PGE₂ receptors, to mitigate hyperfiltration-mediated kidney injury to delay the progression of chronic kidney disease in children born with CAKUT.

Recently, his research has shown that elevated SNGFR results in increased fluid flow shear stress and increased expression of prostanoid receptor EP2 during early-stage CKD in animal models. He has now shown that urinary PGE₂ excretion precedes overt microalbuminuria and is a biomarker for early hyperfiltration-induced injury in children born with a solitary functioning kidney. Supported by his NIH funding, Dr. Srivastava published this research in Prostaglandins & Other Lipid Mediators in February 2020.¹

A second study, Upregulated Proteoglycan-related Signaling Pathways in Fluid Flow Shear Stress-treated (FFSS) Podocytes, was published in the American Journal of Renal Physiology in August 2020. In this study, Dr. Srivastava and his colleagues showed the importance of podocyte glycocalyx in response to FFSS, which builds on his previous work establishing the importance of COX2-PGE₂-EP2 axis and Akt-GSK3β-β-catenin signaling downstream of prostanoid receptor EP2. This was based on cell and animal work on fluid flow shear stress.^2,3,4 These studies allow us to develop potential therapeutic strategies to mitigate hyperfiltration-mediated injury.

And finally, in the original research article published in Frontiers in Physiology called Unilateral Nephrectomy Stimulates ERK and Is Associated with Enhanced Na Transport, the authors discuss the relevance of FFSS in tubular cells, resulting in activation of ERK signaling.⁵

Researching Development of Nephrotic Syndrome

Although primary nephrotic syndrome is a relatively rare condition, according to the NephCure Kidney International Foundation, it is responsible for 20% of kidney failure in children, making it an important research target.

Dr. Srivastava is well known for his expertise in idiopathic nephrotic syndrome as part of his clinical practice, which intersects with his laboratory research. He is recognized as a nephrotic syndrome specialist by NephCure, and he has studied the role of innate immunity in podocytes to better understand the mechanisms behind the development of nephrotic syndrome.

Dr. Srivastava is involved in clinical trials related to idiopathic nephrotic syndrome and is part of the National Institutes of Health multicenter studies, NEPTUNE, CureGN and FONT; American Heart Association (ECHO-NEPTUNE); and industry investigations (Abatacept, ATLANTIS, Duplex and DUET) as the Children’s Mercy site principal investigator.

Many of these trials are investigating therapies for children with treatment-resistant nephrotic syndrome. Children’s Mercy also participates in the Pediatric Nephrology Research Consortium, which provides Dr. Srivastava an opportunity to collaborate with other pediatric nephrologists around the country to participate in collaborative research.

Committed to Transformational Research

The Division of Pediatric Nephrology at Children’s Mercy is one of the most academically productive departments in the country, publishing 63 peer-reviewed manuscripts in 2020. Children’s Mercy physician-scientists are committed to leading transformational research, from bench to bedside, to create a world of well-being for all children with pediatric kidney disease.

To learn more about our research or to collaborate with us, contact Bradley Warady, MD, Division Director, Children’s Mercy Nephrology, at bwarady@cmh.edu.

Learn More About Pediatric Kidney Disease Research at Children's Mercy

Tarak Srivastava, MD, Director, Nephrology Research Laboratory at Children’s Mercy

tsrivastava@cmh.edu

(816) 234-3010

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

1. Srivastava T, Ju W, Milne GL, Rezaiekhaligh MH, Staggs VS, Alon US, Sharma R, Zhou J, El-Meanawy A, McCarthy ET, Savin VJ, Sharma M. Urinary prostaglandin E2 is a biomarker of early adaptive hyperfiltration in solitary functioning kidney. Prostaglandins & Other Lipid Mediat. 2020 Feb;146:106403.

2. Srivastava T, Joshi T, Jiang Y, Heruth DP, Rezaiekhaligh MH, Novak J, Staggs VS, Alon US, Garola RE, El-Meanawy A, McCarthy ET, Zhou J, Boinpelly VC, Sharma R, Savin VJ, Sharma M. Upregulated proteoglycan-related signaling pathways in fluid flow shear stress-treated podocytes. Am J Physiol Renal Physiol. 2020;319(2):F312-F322.

3. Srivastava T, Dai H, Heruth DP, Alon US, Garola RE, Zhou J, Duncan RS, El-Meanawy A, McCarthy ET, Sharma R, Johnson ML, Savin VJ, Sharma M. Mechanotransduction signaling in podocytes from fluid flow shear stress. Am J Physiol Renal Physiol. 2018 Jan 1;314(1):F22-F34.

4. Srivastava T, Alon US, Cudmore PA, Tarakji B, Kats A, Garola RE, Duncan RS, McCarthy ET, Sharma R, Johnson ML, Bonewald LF, El-Meanawy A, Savin VJ, Sharma M. Cyclooxygenase-2, prostaglandin E2 and prostanoid receptor EP2 in fluid flow shear stress mediated injury in solitary kidney. Am J Physiol Renal Physiol. 2014;307(12):F1323-33.

5. Repetti R, Majumder N, Carneiro De Oliveira K, Meth J, Yangchen T, Sharma M, Srivastava T, Rohatgi R. Unilateral nephrectomy stimulates ERK and is associated with enhanced Na transport. Frontiers in Physiology. Feb. 3, 2021. https://doi.org/10.3389/fphys.2021.583453.

In addition, there is pressure for pain medicine to shift away from reliance on opioids, ineffective procedures and surgeries toward comprehensive pain management that includes evidence-based nonpharmacologic options.¹

Utilizing Acupuncture to Treat Pediatric Headache and Migraine

Children’s Mercy Kansas City implemented an acupuncture clinic to treat pediatric headaches and migraines in October 2015. The program began with one medical acupuncturist, Jennifer Bickel, MD, FAAN, FAHS. Two additional medical acupuncturists, Jennifer Dilts, DO, FAAP, and Anna Esparham, MD, were added to the team to meet increasing demand for acupuncture services. All three were trained at the Helms Medical Institute.

In a retrospective chart review of 154 visits to the Headache Treatment Center from September through November 2018 of patients ages 7-18, average headache intensity scores (measured on an 11-point pain numeric rating scale) decreased after a variety of interventions.

Specifically, acupuncture provided by a board-certified medical acupuncturist at the Headache Treatment Center decreased headache intensity significantly, mean change = 2.07 + 1.90 (n=24), p<.05.²

Demand for Acupuncture in the Emergency Department 

Headache accounts for 1% to 4% of all visits to the pediatric emergency department, making headaches the ninth most common reason for all emergency department visits for patients under age 15.

In addition to variable efficacy of headache treatments received in the emergency department, there are other limitations associated with this setting that can make it suboptimal for the acute management of migraine. Such limitations include a lack of headache-trained or -informed providers, limited targeted interventions and comfort measures used for active headaches, a higher rate of treatment with opioids, and unnecessary imaging. Developing alternative viable models for early therapeutic intervention of headaches may be advantageous to improve management and limit disability.²

Expanding Acupuncture Treatment to the Emergency Department and Other Subspecialties

To address the growing demand for acupuncture in the Emergency Department and in other subspecialities, the medical acupuncturists within the Children’s Mercy Headache Treatment Clinic received a 2019 education grant from the American Academy of Pediatrics Section on Integrative Medicine.

As grant winners, the Children’s Mercy acupuncturists created an abbreviated basic acupuncture training curriculum that was implemented at the academic medical center. The 10-hour acupuncture course offered interested clinicians an accessible way to learn two basic acupuncture techniques: battlefield auricular acupuncture (BFA) and Four Gates.

Once learned, both techniques could be integrated into care at the bedside, making it possible for the patient to receive a clinical assessment and acupuncture treatment during the same emergency department visit from the same clinician.

Learning methods included an online module with videos and articles, two live workshops, and additional home practice.

Over a period of approximately 18 months, 38 pediatricians from nine pediatric subspecialties (22 emergency medicine providers, plus specialists in gastroenterology, hospital medicine, palliative care, hematology/oncology, rehabilitation, sleep medicine, developmental and behavioral pediatrics, and neurology) completed the course.

Participants completed pre- and post-tests measuring knowledge and attitudes, and a skills-based exam. Participants earned 10 American Board of Pediatrics Maintenance of Certification Part 2 points and 10 continuing medical education credits for completing the course.

Evaluation of Basic Acupuncture Curriculum

To determine the effectiveness of the acupuncture curriculum, the course instructors surveyed participants. Results included:

• Knowledge regarding acupuncture significantly increased following curriculum completion.
• The course significantly increased participants’ perceived efficacy of acupuncture for acute and chronic pain.
• All participants successfully completed a skills-based exam.
• All participants felt that the course would influence their current medical practice.
• All participants felt confident utilizing basic acupuncture.
• All participants felt that they would recommend the basic acupuncture curriculum to a colleague.

In a separate study on acupuncture from the Children’s Mercy Emergency Department, the researchers found most staff, parents and patients reported willingness to use acupuncture. In fact, patients (60%) and parents (73%) were willing to add 15 to 60 minutes to their Emergency Department length of stay to complete an acupuncture treatment.

AAP Section on Integrative Medicine Awards Acupuncture Research First Place Honor

As the AAP grant winners, the team was invited to present their research abstract at the AAP National Conference & Exhibition in October 2020 at the Section on Integrative Medicine’s joint session on pain with the Section on Pediatric Emergency Medicine.

The project received the best abstract award for the meeting and generated significant interest from other pediatric institutions interested in collaborating to develop and implement similar programs for their physicians and patients.

Collaborate With Children's Mercy to Bring the Basic Acupuncture Curriculum to Your Institution

Jennifer Bickel, MD, FAAN, FAHS, Medical Director, Center for Professional Well-Being, Division of Neurology, Chief Headache Section

jlbickel@cmh.edu

(816) 302-3331

Jennifer Dilts, DO, FAAP, Division of Neurology, Headache Section

jjdilts@cmh.edu

(816) 302-3328

Anna Esparham, MD, FAAP, Director of Headache Treatment Center, Division of Neurology

aeesparham@cmh.edu

(816) 302-3320

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

1. Tick H, Nielsen A, Pelletier KR, Bonakdar R, Simmons S, Glick R, Ratner E, Lemmon RL, Wayne P, Zador V. Evidence-based Nonpharmacologic Strategies for Comprehensive Pain Care: The Consortium Pain Task Force White Paper.

2. Esparham A, Boorigie M, Ablatt S, Connelly M, Bickel J. Improving Acute Treatment of Pediatric Primary Headache Disorders With a Novel Headache Treatment Center: Retrospective Review of Preliminary Outcomes. Journal of Child Neurology. DOI: 10.1177/0883073820952997journals.sagepub.com/home/jcn.

Pediatric tracheostomy is increasing, with 40% of tracheostomies performed in children <1 year of age.¹ The significant increase in the rate of tracheostomy in infants is partly attributed to the increased survival of extremely preterm infants with severe BPD who require long-term ventilation for several years.^2,3

Significant variation in practice has led to conflicting opinions on indications, feasibility and long-term prognosis of infants <1 year old undergoing tracheostomy.^1,4 Decannulation time, mortality and neurodevelopmental outcomes vary significantly based on the indication for tracheostomy and underlying diagnosis.⁴

Home Ventilator Program Provides Unique Opportunity to Study Tracheostomy Population

The outcomes of infants with tracheostomy have not been well described in the literature. However, Children’s Mercy Kansas City developed the Infant Tracheostomy and Home Ventilator Program in 2005. This neonatology-led program, headed by Winston Manimtim, MD, Neonatologist and Medical Director, is the only program in the U.S. serving as a medical home for these patients in a multidisciplinary setting with other key subspecialists, including pulmonologists, gastroenterologists and otolaryngologists.

The program has collected the data necessary to systematically examine several key factors related to outcomes in this specific patient population, contributing important research to this field. Their most recent retrospective study on tracheostomy and decannulation included:

• 204 infants who met the inclusion criteria from 2005 through 2015 with tracheostomy at <1 year of age and follow-up in the Home Ventilator Clinic. No patients were excluded from the study.
• Primary indications for tracheostomy were abnormalities related to: upper airway (13.7%); lower airway (16.7%); combined airway and lungs (BPD) (40.2%); cardiac (7.3%); neurologic (6.9%); and others (15.2%).
• 93% of infants were G-tube dependent for their nutritional support at discharge; the proportion of infants who were able to feed orally (7%) had tracheostomy only and did not need assisted ventilation at discharge. No patients were discharged with nasogastric tube.
• Mean age at hospital discharge for all infants was 6.5 months and median age was 7 months.⁵

Highlights Document Age at Tracheostomy, Decannulation

The team’s goal was to describe the respiratory, growth and survival outcomes of these patients. Results were published in the high-impact medical journal Pediatric Research.

Highlights included:

• The mean age at tracheostomy was 4.5 months with a median age of 3 months.
• Median age of decannulation was 32 months.
• The time from tracheostomy placement to complete discontinuation of mechanical ventilation was 15.4 months; and from tracheostomy to decannulation was 33.8 months.
• Mortality rate was 21% and median age of death was 18 months.
• Preterm infants with acquired airway and lung disease (BPD) and born at <28 weeks’ gestation had a significantly higher survival rate compared to term infants with various tracheostomy indications.
• The z-scores for weight and weight for length improved from the time of discharge (mean chronological age 6.5 months) to first year and remained consistent through 3 years of age.⁵

Findings Offer Guidance for Counseling and Care

With 204 infants in this cohort, it was one of the largest studies of its kind, a significant strength of this research. These infants had regular and consistent outpatient follow-up care from a dedicated team throughout the first 3-4 years of age, thanks to the Children’s Mercy Infant Tracheostomy and Home Ventilator Program. This large number allows for more extensive statistical modeling that can help guide families and care providers on important prognostic factors, outcomes and follow-up care plans throughout the first few critical years of life.⁵

Specific to the Children’s Mercy cohort, the team found through regression analysis that the premature infants had a much higher rate of survival compared to term infants with tracheostomy, as well as a higher rate of decannulation. The authors postulated that the improved overall survival outcomes in these very high-risk infants are the result of providing a dedicated multidisciplinary approach that ensures timely, regular and consistent delivery of both primary and subspecialty care, along with optimizing nutritional support to enhance pro-growth state, incorporating developmental surveillance in every clinic visit, and addressing caregivers’ psychosocial issues.

The team’s goal is that these insights will assist other clinicians as they provide families with counseling based on this data, helping them manage expectations for tracheostomy and home ventilator support, and aiding them in making better informed decisions on behalf of this vulnerable population.

Learn more about the Children's Mercy Infant Tracheostomy and Home Ventilator Program

Winston Manimtim, MD, Neonatologist; Medical Director, Infant Tracheostomy Care and Home Ventilator Program

wmmanimtim@cmh.edu

(816) 302-3592

Gangaram Akangire, MD, Neonatologist

gakangire@cmh.edu

(816) 302-3592

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

1. Muller RG, Mamidala MP, Smith SH, Smith A, Sheyn A. Incidence, Epidemiology, and Outcomes of Pediatric Tracheostomy in the United States from 2000 to 2012. Otolaryngol. Head Neck Surg. 160, 332–338 (2019).

2. Overman AE, et al. Tracheostomy for Infants Requiring Prolonged Mechanical Ventilation: 10 Years’ Experience. Pediatrics 131, e1491–e1496 (2013).

3. Ehrenkranz RA, et al. Validation of the National Institutes of Health Consensus Definition of Bronchopulmonary Dysplasia. Pediatrics 116, 1353–1360 (2005).

4. Salley J, Kou YF, Shah GB, Mitchell RB, Johnson RF. Survival Analysis and Decannulation Outcomes of Infants with Tracheotomies. Laryngoscope 130, 2319–2324 (2020).

5. Akangire G, Taylor JB, McAnany S, Noel-MacDonnell J, Lachica C, Sampath V, Manimtim W. Respiratory, Growth, and Survival Outcomes of Infants with Tracheostomy and Ventilator Dependence. Pediatr Res. 2020 Oct. 3:1-9. doi:10.1038/s41390-020-01183-x. Online ahead of print.

Precise cardiovascular anatomy is required for neonatal surgical planning. CT angiogram is commonly used to complement echocardiography. However, in small neonates, CT angiogram can be challenging due to a lack of power-injectable vascular access and rapid contrast transit, secondary to higher heart rates.

At the Ward Family Heart Center at Children’s Mercy Kansas City, Non-Invasive Advanced Cardiac Imaging and Echocardiography are utilizing MRI techniques that make it possible to obtain high-resolution images in small neonates, yet limit their exposure to ionizing radiation.

In these cases, a swaddling technique is used to calm the infant for the MRI exam, eliminating the need for sedating anesthesia. Utilizing a blood pool contrast agent for MR angiography that can be infused in a small peripheral intravenous line enables the acquisition of these images necessary for surgical planning. These techniques are used in neonates weighing down to 2 kilograms.

MR angiograms and additional functional imaging also have been used in clarifying critical details in lesions, like vascular rings, Tetralogy of Fallot with pulmonary atresia and atrioventricular septal defect in neonates. This demonstrates that these MRI techniques are diagnostic in neonates, thus reducing the risks of CT and anesthesia while complementing echocardiography.

Utilizing Point-of-Care Ultrasound for Cardiac Evaluation During COVID-19

The novel coronavirus SARS-CoV-2 is known to cause significant cardiac dysfunction, effusion, thromboembolism and coronary dilation. As the COVID-19 pandemic spread across the nation and these cardiac complications began to surface in the pediatric population, Non-Invasive Advanced Cardiac Imaging and Echocardiography at Children’s Mercy began searching for ways to safely and effectively detect these issues.

In addition to traditional ultrasound equipment, the team quickly pivoted to adopt a hand-held ultrasound probe that can be connected to an iPad. Because the unit is significantly smaller and more compact, cleaning and sanitizing it after each patient exam is much simpler. Plus, the technology can be readily utilized at the bedside with diagnostic image quality.

The team’s workflow was to perform the point-of-care exam, and if the scan revealed findings requiring more comprehensive evaluation, a follow-up detailed echocardiography exam was planned. For those patients with normal exams, the detailed echo was deferred.

Overall, point-of-care ultrasound has helped improve workflow and expedite care for pediatric patients admitted for COVID-19, while ensuring safety of patients and health care workers. Thanks to its portability and ease of use, the heart center imaging team is now training critical care specialists and neonatologists at Children’s Mercy in how to perform these ultrasound examinations at the bedside, putting access to this lifesaving testing at their fingertips.

Serving as the Echocardiography Core Lab for FDA Investigation of New Transcatheter Pulmonary Valves

Since 2012, the Non-Invasive Advanced Cardiac Imaging and Echocardiography Lab at Children’s Mercy has served as the Echocardiography Core Lab for FDA investigation of the safety and effectiveness of transcatheter pulmonary valves.

These valves are being developed by Edwards Lifesciences and include the SAPIEN 3 and Alterra transcatheter heart valves. The lab continues surveillance of patients who received the SAPIEN valve, which has been recently approved by FDA for clinical use.

In order to monitor valve safety and effectiveness over a five-year time frame, patients receive from five to nine different echo studies pre- and post-implant for each device under investigation.

The lab has performed more than 1,500 echo studies related to these valves since 2012, validating the team’s expertise in pediatric echocardiography while providing access to longitudinal follow-up data on hundreds of patients.

Developing New Apps for Advanced Imaging

Like many non-invasive pediatric cardiology programs, Children’s Mercy utilizes Lean methodology and meets all guidelines from the American College of Cardiology and the American Heart Association for advanced cardiac imaging. The program utilizes 3D printing capabilities for surgical planning; has a robust telemedicine platform that offers image transfer, archiving and expert interpretation of cardiac imaging studies; and provides 3D echocardiography and myocardial strain imaging surveillance for oncology patients post-treatment through the hospital’s Cardio-Oncology Clinic.

A program under development places the program on the leading edge of pediatric cardiology by using a novel online module-based curriculum to enhance pediatric cardiology fellowsʼ echo education. Four interactive modules, focused on principles and interpretation of a normal echo, were delivered to fellows. All fellows completed pre- and post-tests, validated by independent expert imagers and surveys. All of the fellows participated in the pilot study. Test scores improved after completing the modules, and most fellows maintained a higher score at four weeks, suggesting good knowledge retention.

Learn More About Advanced Imaging at Children’s Mercy

Sanket Shah, MD, MHS; Co-Director of Advanced Cardiac Imaging and Director of Echocardiography

ssshah@cmh.edu

Erin Opfer, DO; Co-Director of Advanced Cardiac Imaging

ekopfer@cmh.edu

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

Though sleep disorders are common in the pediatric population, infants represent an understudied minority. Little research has been published on the subject of sleep-disordered breathing (SDB) in this population, even though it can have a significant impact on health during the formative years of neurocognitive development.

To address this issue, Zarmina Ehsan, MD, Pediatric Sleep Medicine, Children’s Mercy Kansas City, joined forces with the data science team led by Mark Hoffman, PhD, Chief Research Information Officer with the Children’s Mercy Research Institute, home to one of the most progressive and dynamic pediatric research programs in the world.

They utilized big data to conduct the first large-scale study on SDB in infants. Dr. Ehsan identified the gaps in currently available literature as:

1. Limited understanding of the evolution of SDB during infancy and beyond.
2. Paucity of centers performing infant polysomnography (PSG) and, therefore, available literature generated based on information from select centers.
3. Management of infant obstructive sleep apnea (OSA) based on extrapolation of available literature from older children, potentially increasing the risk of providing inappropriate therapy to this understudied and underserved population with SDB.

These gaps also make this population an ideal focus of data science.

Database Highlights

Children’s Mercy utilizes the de-identified electronic health record Cerner Health Facts. The hospital also manages the Standardized Health and Research data Exchange (SHaRE), a group of Health Facts contributors who have agreed to collaborate and share de-identified patient data. By employing this big data methodology, the researchers determined the demographics, sleep diagnoses, comorbid medication conditions, healthcare utilization and economic outcomes for this patient database. Earl Glynn, a data scientist in Dr. Hoffman’s group, developed an innovative method to characterize comorbidities in this data resource.

Highlights included:

• In a cohort of 68.7 million unique patients, over a nine-year period, there were 9,773 infants and young children with a diagnosis of SDB (obstructive sleep apnea [OSA], nonobstructive sleep apnea, and “other” sleep apnea) who met inclusion criteria, encompassing 17,574 encounters, and a total of 27,290 diagnoses across 62 U.S. health systems, 172 facilities and three patient encounter types (inpatient, clinic and outpatient).
• Thirty-nine percent of patients were female; 39% were 1 year of age (6,429); 50% were 1-2 years of age; and 11% were 2 years of age.
• The most common comorbid diagnoses were micrognathia, congenital airway abnormalities, gastroesophageal reflux, chronic tonsillitis/adenoiditis and anomalies of the respiratory system.
• Payer mix was dominated by government-funded entities.

Big Data Yields Insight into Comorbidities

This is the first study to utilize big data in this understudied population and to demonstrate the utility of large-scale de-identified electronic health record data in sleep research in general.

Interestingly, this study confirmed what we see clinically: that sleep-disordered breathing in infants and young children is multifactorial and closely linked to comorbid medical conditions.

Additional insights that are not widely known include:

• Chronic tonsillitis and adenoiditis were prevalent in 21% of the OSA cohort, compared to 0.6% of the base population within this age group (prevalence ratio of 36%).
• Adenotonsillar hypertrophy is a well-known risk factor for OSA in older children, although few studies also report that it can be prevalent as early as infancy.
• About 2.9% of this cohort had a diagnosis of jaw abnormality, compared to 0.1% in the general population within this age group. This phenotype had the second largest prevalence ratio (27.9), but was not present in the nonobstructive sleep apnea or “other” sleep apnea cohorts, which is not unexpected as micrognathia is a known risk factor for OSA in infants, particularly in those with a cleft palate.
• Congenital anomalies of the respiratory system were prevalent in 11% of the OSA cohort, compared to 0.8% of the base population. Retrospective clinical research has shown that congenital soft tissue airway abnormalities or bone abnormalities involving the face also predispose to OSA.
• Laryngomalacia is one of the leading causes of stridor, upper airway obstruction, and therefore OSA, in infants. Other airway abnormalities such as subglottic stenosis or tracheomalacia are often present as well.
• Prevalence ratios for “dependence on respirator/ventilator” and “other diseases of the lung” also ranked high at 11% each.
• A diagnosis of otitis media was also prevalent within the cohort. There are a handful of studies suggesting a high prevalence of middle ear effusion and otitis media in infants with OSA (particularly those with Down syndrome).
• Chromosomal abnormalities consistently scored high on prevalence ratio in all three cohorts, with a higher prevalence than in the base population.
• The link between gastroesophageal reflux and OSA in infants has been reported in the literature; however, the evidence for this is weak. In all three cohorts, this research found a higher prevalence of gastroesophageal reflux compared to the base population, but prevalence ratios did not appear to be significant.

Conclusions:

Dr. Ehsan and her colleagues concluded large-scale aggregate, de-identified EHR data provide a rich and untapped resource to examine these conditions. The team is in the process of pursuing further research targeting infants and young children to better understand the long-term outcomes of SDB in this patient population.¹

Learn more about Pulmonary and Sleep Telemedicine Services at Children’s Mercy

Zarmina Ehsan, MD, Pediatric Pulmonology, Pediatric Sleep Medicine; Clinical Assistant Professor of Pediatrics, University of Missouri-Kansas City School of Medicine

zehsan@cmh.edu • (816) 234-3700

For consults, admissions or transport call: 1 (800) GO MERCY / 1 (800) 466-3729.

References:

1. Ehsan Z, Glynn EF, Hoffman MA, Ingram DG, Al-Shawwa B. Small Sleepers, Big Data: Leveraging Big Data to Explore Sleep-Disordered Breathing in Infants and Young Children. SLEEPJ, 2020, 1–13. doi: 10.1093/sleep/zsaa176.