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In the News: FUEL Study Results, Better Screening for Retinopathy of Prematurity, Prenatal Stress, and New 2019 Annual Report

Published on
Nov 29, 2019
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The holiday season is in full swing, and so is our research at Children’s Hospital of Philadelphia Research Institute. This edition of In the News highlights the clinical benefit of a medication for a unique population of children with single-ventricle heart disease, describes an improved method for predicting retinopathy of prematurity, and shares surprising outcomes from a study assessing prenatal stress. And if you’re looking for an inspirational read this holiday, you’ll find plenty in our newly released Research Institute Annual Report!

Milestone Results Reported for Fontan Survivors

The largest-ever clinical trial of a medication for pediatric cardiology patients found that an oral drug significantly improved exercise capacity in adolescent patients with severe, congenital single ventricle heart defects. 

Pediatric cardiologist David J. Goldberg, MD, of the Cardiac Center at CHOP and co-principal investigator of the multicenter Fontan Udenafil Exercise Longitudinal Assessment Trial (FUEL), reported the results at the 2019 Scientific Sessions of the American Heart Association in Philadelphia and was the lead author of an article published concurrently in the journal Circulation. The principal investigator of the trial, also from CHOP’s Cardiac Center, was Stephen Paridon, MD

The physiologic benefits represent a milestone in the care of those who have undergone the Fontan procedure, a palliative operation for single ventricle disease.

“Exercise capacity is a surrogate for morbidity and mortality outcomes in children with single ventricle congenital heart disease,” Dr. Goldberg said. “It is our hope that an improvement in exercise capacity will translate into better long-term outcomes.” 

The Phase 3 randomized, double-blind, placebo-controlled clinical trial, sponsored by Mezzion Pharma Co. Ltd., enrolled 400 male and female participants aged 12 to 18 years old from 30 centers in the United States, Canada, and South Korea within the Pediatric Heart Network. The researchers reported that participants in the FUEL trial had statistically significant improvements in oxygen consumption and other measures of exercise capacity during moderate levels of activity.

“This study of udenafil provides the first evidence of clinical benefit for a medication in this unique population of children with single-ventricle heart disease,” Dr. Goldberg said.

Disclosure: Drs. Goldberg and Paridon both receive grant support from Mezzion and are co-inventors of patent US10137128B2 which is for the use of Udenafil in Fontan physiology. CHOP holds these patent rights in conjunction with Mezzion. 

For more information, see the CHOP press release.

New Screening Approach for Retinopathy of Prematurity Developed 

A multicenter group of 41 hospitals led by CHOP researchers confirmed that an improved method for predicting retinopathy of prematurity (ROP), a leading cause of blindness in children, reduced the number of babies having invasive diagnostic examinations by nearly a third, while raising disease detection up to 100 percent.

ROP is a disorder of the blood vessels of the retina that affects premature babies born with immature retinas. In general, the more premature the baby and the lower the birth weight, the greater the risk for developing ROP. Since the disease has no external signs or symptoms when it first develops, it can only be properly detected through an eye exam.

The new method described by the study team in the journal JAMA Ophthalmology adds slow weight gain to the existing criteria for ROP screening. Slow growth is a sign of low growth hormones that are involved in the development of ROP. Because nurses routinely measure weight gain in infants, these simple measurements are readily available. If implemented, this screening approach could considerably reduce both unnecessary healthcare costs and physically stressful retinal examinations for premature infants.

In a previous study, Gil Binenbaum, MD, an attending surgeon in the Division of Ophthalmology, the Richard Shafritz Endowed Chair of Ophthalmology Research at CHOP, and Chair of the Postnatal Growth and ROP (G-ROP) Study Group that performed the research, and his colleagues developed what they called the “G-ROP criteria” using a hybrid modeling approach that combined birth weight and gestational age criteria, weight gain comparisons to expected growth from infants without ROP, and user-friendly screening criteria.

They tested the G-ROP criteria in this latest study prospectively among 3,981 premature infants at risk of developing ROP across 41 hospitals in the United States and Canada. When the cohorts from the two studies were combined, the G-ROP criteria correctly predicted 677 of 677 Type 1 ROP cases, while reducing the number of infants receiving examinations by 33 percent among the 11,464 babies.

“This study successfully validated the accuracy of the G-ROP screening criteria, which now can be used clinically to reduce the number of infants receiving eye examinations for ROP,” Dr. Binenbaum said. “Based upon these new findings and the findings of our previous study, we recommend that these criteria are incorporated into national ROP screening guidelines.”

Read more in the CHOP press release.

Study Assessing Prenatal Stress Reveals Surprising Outcomes

Researchers from the Lifespan Brain Institute (LiBI) of CHOP and the University of Pennsylvania, in collaboration with Clalit Research Institute in Israel, wanted to learn how prenatal exposure to a limited one-month stressor, in the form of repeated rocket attack on a civilian population, may affect neuropsychiatric outcomes in children. 

Researchers looked at almost 7,000 children born in the months following the 2006 Second Lebanon War in northern Israel and compared them to children from the same province who were born one year later when rocket attacks had ceased. The team found these children did not have higher rates of neuropsychiatric outcomes compared to non-exposed children when followed up to age nine. 

“The findings were very surprising to us,” said Ran Barzilay, MD, PhD, lead author, child and adolescent psychiatrist at CHOP, and research scientist at LiBI. “We assumed going into the study that the children who were prenatally exposed to the bombings would be at an increased risk for a variety of neuropsychiatric conditions, but that was not the case.”

Furthermore, mothers who were exposed to the rocket attack had a lower rate of peripartum depression than non-exposed mothers.

When asked why this may be, Dr. Barzilay explained, “It is possible that there are resilience factors that prevent the possible deleterious effects of prenatal stress on brain development. But it is also possible that adverse neuropsychiatric effects may present in the adolescent or early adult years, which were not assessed in the current study since we followed the children until age nine.” 

Dr. Barzilay and his team, along with LiBI Director Raquel Gur, MD, PhD, are starting new studies to prospectively follow pregnant mothers and their children to identify risk and resilience factors that affect the development of brain and behavior. 

The findings appeared in European Child & Adolescent Psychiatry. Find out more in a CHOP press release.

Celebrate Our Scientific Achievements With the 2019 Annual Report

Our 2019 Research Institute Annual Report is here! This year’s collection of stories capture the innovative and exciting breakthroughs in pediatric science made by our research community over the fiscal year 2019. Read about game-changing collaborations, discover novel research initiatives, and meet the visionaries, leaders, families, and scientists who inspire us to drive pediatric medicine forward every day.  

ICYMI

Catch up on our headlines from our last edition

  • Newsweek Covers Allergy Research by World-renowned CHOP Doc
  • Finding a Way to Accelerate Cures for Children With Cancer 
  • Tagging CAR T Cells With Imaging Markers to Monitor Cancer Therapy in Body
  • Paul Offit, MD, Honored With Prestigious Science Award
  • Patterns of Spinal Curvature in Children May Predict Scoliosis in Teens

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