News at CHOP: Summer 2022

Children’s Hospital of Philadelphia awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the U.S. Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 166-year history, the Gold Medal highlights High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.

“It is a tremendous honor and a distinct pleasure to present this award to Katherine High, an inspirational scientist who did something that few thought possible: developing gene therapies and taking them from bench to bedside,” said Madeline Bell, CHOP President and CEO. “Through her innovative vision, she and her colleagues restored the vision of others, helping children around the world and laying the foundation for future breakthroughs for those with genetic diseases. Her hemophilia research has revolutionized the field, bringing ‘once and done’ therapy for this bleeding disease one step closer to reality.”

The CHOP Gold Medal is given to individuals or institutions that have improved the health and welfare of children by making significant discoveries in medicine, surgery, and other specialties; psychiatry and social sciences; education; and research. The award was created to recognize transformative achievements that are making an impact across the globe.

Treatment Developed by CHOP Researchers Prevents Hypoglycemia in Children with Hyperinsulinism

Children with hyperinsulinism may soon have a new medication to control their hypoglycemia after exendin-(9-39), a medication developed at CHOP, showed it was effective at preventing dangerously low blood sugar levels
in younger children. It could spare patients without other effective drug options the need for a pancreatectomy.

Exendin-(9-39) works to inhibit GLP-1 signaling, which blocks insulin secretion in those with the KATPHI form of the disease, the most common type. This study confirmed an earlier study in adults that exendin-(9-39) reduced the likelihood of hypoglycemia — in this study by 76% to 84% (depending on the dose) and increased fasting glucose by 20% to 28%. “

We are very excited about this study because by targeting the underlying pathophysiology, exendin-(9-39) offers potential therapeutic advantages over currently available therapies for HI, which could make a huge difference in the lives of the children we care for,” says senior study author Diva D. De León-Crutchlow, MD, Chief of the Division of Endocrinology and Diabetes and Director of the Congenital Hyperinsulinism Center.

During Pandemic, Kids and Young Adults Exercised Less, Study Confirms

Children and young adults experienced a decline in cardiopulmonary exercise performance during the COVID-19 pandemic, according to a new study by researchers at CHOP. The study was published in Pediatric Cardiology.

Both males and females in the study saw an increase in BMI during the pandemic and a significant decrease in aerobic capacity, as measured by cardiopulmonary exercise tests. The greatest decrease in exercise performance among those who were the most aerobically fit pre- pandemic. One explanation for this finding, researchers suggest, is that the most aerobically fit subjects were likely participating in competitive, structured sports pre-pandemic, and those activities were canceled during the pandemic period.

“Although social distancing measures were a necessary public health measure during the pandemic, this study demonstrates unintended consequences that should be kept in mind as further policy is developed and implemented,” says Danielle S. Burstein, MD, a cardiologist in the CHOP Cardiac Center and first author of the study.

Opioid Prescriptions for Pediatric Patients Following Surgical Procedures Have Dropped Significantly

Researchers from CHOP and the Perelman School of Medicine, led by Tori Sutherland, MD MPH, found that opioid prescriptions for children who underwent 1 of 8 common outpatient surgeries declined substantially between 2014 and 2019. These findings, published in Pediatrics, suggest that clinicians are using more discretion when considering which pediatric patients require an opioid prescription after procedures associated with mild to moderate pain, where they are commonly prescribed but are often discretionary.

Investigators used data from a private insurance database to study 124 249 opioid-naïve patients under the age of 18 undergoing surgery. They found the percentage of children who had an opioid prescription filled after their surgery dropped from 78.2% to 48% for adolescents; from 53.9% to 25.5% for school-aged children; and from 30.4% to 11.5% for preschoolers. Additionally, the average morphine milligram equivalent dispensed declined by approximately 50% across all age groups.