10/21/2026
As a leader in the treatment of complex diseases in multiple pediatric specialties, Riley Children’s Health is wrapping up an impactful year of firsts in patient care and clinical research.
“Riley is a place with exceptional people, a strong collaborative culture and a clear commitment to innovation,” said Douglas K. Graham, MD, PhD, who recently joined Riley Children’s as physician-in-chief and leads the Indiana University School of Medicine Department of Pediatrics, ranked No. 6 for National Institutes of Health (NIH) funding. “By strengthening the integration of research and clinical medicine across specialties, we are uniquely positioned to further distinguish ourselves as a sought-after national destination for pediatric care.”
Firsts in clinical trials and patient care
Gastroenterology
- First site in the world to enroll a pediatric patient in the QUASAR Jr. trial, the first approved clinical trial evaluating guselkumab for children with ulcerative colitis.
“A coordinated team effort supported by a strong research infrastructure allowed us to quickly identify this patient as a candidate and move efficiently into enrollment,” said Marian Pfefferkorn, MD. “For children with limited remaining options, participation in a trial like this can offer a meaningful opportunity to potentially avoid colectomy.”
Nephrology
- Enrolled the world’s first pediatric participant in PIONEER, a first-of-its kind international multisite clinical trial to combat immunoglobulin A (IgA) nephropathy.
“Without pediatric clinical trials, children must wait until they reach adulthood to get access to IgA treatments,” said Myda Khalid, MD. “For a 12-year-old child, that would mean six years of risk of permanent kidney function decline before accessing new therapies.”
- Enrolled the first patient in the U.S. in the EMPA Kidney Kids global clinical trial to study the safety and efficacy of empagliflozin in children with chronic kidney disease.
Hematology, Oncology & Stem Cell Transplant
- Performed the world’s first successful pediatric bone marrow transplant using cells from a deceased donor.
“This treatment has the potential to dramatically expand access to transplant for children who previously had few or no remaining treatment options, giving us a way to close critical gaps in care and offer hope to families facing impossible circumstances,” said Jodi Skiles, MD.
Notable firsts in Indiana
- For children and adolescents whose emotional, behavioral and medical conditions intersect, Riley Children’s opened a Pediatric Medical-Behavioral Health Unit—one of the first units of its kind in the nation and the first in Indiana.
“By integrating behavioral health expertise into medical care, we can better support patients experiencing eating disorders, severe anxiety, depression, behavioral dysregulation and other conditions that require a specialized approach,” said Leslie Hulvershorn, MD.
- Riley Children’s joined the small number of health systems nationwide able to deliver a complex groundbreaking gene therapy treatment for transfusion-dependent beta-thalassemia.
Other premier programs at Riley Children’s
- Cardiothoracic Surgery: The Riley Heart Center meets the criteria for a “Comprehensive Care Center”— a top-tier designation based on updated pediatric heart surgery national standards.
- Diabetes & Endocrinology: One of the first in the Midwest to administer teplizumab to delay Type 1 diabetes (T1D), Riley Children’s is an active clinical trial site for therapies designed to modify the course of T1D.
- Neonatology: Advancing precision medicine in the NICU, Riley’s Perinatal Genomics Program has been the subject of seven scientific presentations at national conferences.
- Nephrology: Riley Children’s is home to the nation’s first comprehensive neonatal kidney health program.
- Neurosurgery: Riley neurosurgeons and maternal fetal medicine specialists perform mini-laparotomy fetoscopic repair—an innovative, minimally invasive surgical technique—for prenatal closure of myelomeningocele.
- Orthopedics: With three fellowship-trained orthopedic oncologists, Riley Children’s has one of the largest pediatric orthopedic oncology groups in the nation.
- Pulmonology: Riley Pulmonology is one of the few pediatric programs in the U.S. with an NIH program project grant to develop asthma treatments.
- Urology: Riley Children’s is one of the largest-volume centers for pediatric robotic urologic surgeries in the U.S.
Read about more program distinctions and highlights in the latest Riley Children’s Health Annual Report, published in October 2026.
Physician-scientists and researchers at Riley Children’s Health are supported by numerous National Institutes of Health (NIH) awards. Content related to these awards and included in this article is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.
