Anchored by a robust research program and leading-edge clinical offerings such as nasal epithelial cell theratyping, the Cystic Fibrosis Care Center at Riley Children’s Health is a leader in advancing treatment and enhancing care for children and young adults with cystic fibrosis (CF).
The CF Center at Riley Children’s, accredited by the Cystic Fibrosis Foundation, is one of the nation’s largest, treating more than 430 patients from Indiana and neighboring states.
As one of the few pediatric centers in the U.S. to offer theratyping, Riley Children’s has helped patients gain access to highly effective CF transmembrane conductance regulator (CFTR) modulators.
“CFTR modulators are transformative for patients and have made a huge difference in quality of life and outcomes,” said Don B. Sanders, MD, MS, director of the Cystic Fibrosis Care Center at Riley Children’s. “However, these medications aren’t available unless we can demonstrate that patients will respond, and that’s where theratyping becomes so critically important.”
Working to ensure as many people as possible can take advantage of these breakthrough medicines, physician-scientists at Riley Children’s have been able to show efficacy of CFTR modulators in patients without a qualifying mutation.
“We've discovered that some patients who possess mutations we thought were not responsive to modulators actually do respond to these medications in nasal epithelial cell theratyping studies,” Dr. Sanders explained. “With these findings, we’ve been able to get insurance approval for these CFTR modulators for some of our patients who otherwise would not qualify.”
Also related to clinical care, Riley Children’s is home to a leading pulmonary function laboratory and is a recognized leader in infant pulmonary function measurements, forced oscillometry and multiple breath washout (MBW). Riley experts in this specialized lab have helped train other labs across the nation to perform MBW and are instrumental in ensuring MBW data are acceptable for research and clinical use.
Advancing care and quality improvement
Comprised of more than 30 multidisciplinary professionals, the Cystic Fibrosis Care Center team at Riley Children’s is dedicated to continuous quality improvement and elevating patient care. In addition to Riley pulmonologists, the team includes partners in gastroenterology, endocrinology, psychology, genetics, nursing, respiratory therapy, nutrition, social work, pharmacy and child life.
“Our team is constantly innovating and proactively identifying ways we can make things better for our patients,” said Dr. Sanders. “They step up even before issues become apparent, to address and improve the quality of care we deliver.”
The Riley team has established a highly successful CF pediatric-to-adult transition of care program, working in collaboration with adult pulmonologists at Indiana University Health. The program is led by Riley pulmonologist Pi Chun (Jenny) Cheng, MD, who sees both pediatric and adult CF patients. The program includes age-appropriate patient goals beginning in elementary school and educational assessments to gauge patients’ understanding of their treatment and disease.
With a grant from the CF Foundation, Riley Children’s is working with other centers on a project to improve the quality of home spirometry data. Additionally, the CF team is engaged in a social determinants of health project designed to identify issues such as food insecurity and other socioeconomic factors that could impact the health of CF patients.
“Our model of care is to always stay vigilant about disease progression and to work closely with our families on optimizing care with the goal of preventing progression of CF lung disease,” Dr. Sanders said.
Leader in CF research
With 10 physician-scientists and researchers involved in CF studies, Riley Children’s participates in research through the Cystic Fibrosis Foundation and the Therapeutics Development Network Center, as well as investigator-initiated studies. Riley physician-scientists are recognized experts in the conduct of CF clinical trials.
Dr. Sanders is a leader of two national research programs, STOP360 and STOP PEDS. These studies are enrolling children and adults with CF in clinical trials to optimize treatment for exacerbations of CF lung disease. Riley Children’s also serves as a Midwest coordinating center for clinical study enrollment for CF lung disease in Indiana, Ohio and Kentucky.
