Inside Pediatrics Fall/Winter 2024

DIVISION ROUNDS

For a closer look at these stories and more, visit InsidePeds.org

NEONATOLOGY

Studies identify risk factors for BPD-PH in preterm infants Two studies led by Children’s of Alabama neonatologists are shedding light on the risk factors for bronchopulmonary dysplasia–associated pulmonary hypertension (BPD-PH), a condition that can lead to long-term breathing and health problems. It’s a major threat: nearly half of preterm infants develop BPD. About 20% of these babies also develop PH, and an estimated 40% of them will die of BPD-PH before turning 2 years old.

neonatologist Samuel Gentle, M.D., wanted to fill the vacuum. That’s why he and colleagues at the University of Alabama at Birmingham (UAB) published two studies that assessed factors that may contribute to BPD-PH in preterm babies. Published in the American Journal of Respiratory and Critical Care Medicine , they found two important associations: The duration of intermittent hypoxia events and the presence and persistence of a patent ductus arteriosus (PDA) after birth are both novel risk factors for BPD-PH. But far more study is needed, Gentle pointed out. “While this research focused on a specific phenotype of BPD,” he said, “we need a comprehensive approach to identifying each infant’s specific type of lung disease from which we can decide on the best therapeutic course for every child.”

will also develop PH 20 %

die before the age of 2 40 %

NEARLY HALF

preterm infants develop BPD

Since research has been scant about what causes the development of BPD-PH—as well as how to screen, treat and prevent it—

NEPHROLOGY

Testing the first potential treatment for AMKD

A team of clinicians and researchers from Children’s and the University of Alabama Birmingham (UAB) has launched a potentially groundbreaking clinical trial testing the first potential treatment for APOL1-mediated kidney disease, or AMKD, a genetic condition linked to mutations in the APOL1 gene that can lead to rapid kidney function decline and, ultimately, kidney failure. This genetic factor is particularly common in African Americans, contributing significantly to the higher rates of kidney failure seen in this population. The trial, called AMPLITUDE, is testing the efficacy and safety of a first-in-its-class oral medication called VX-147, or inaxaplin. The drug is designed to inhibit the inflammatory pathway triggered by the APOL1 gene variant. The hope is that blocking this pathway will slow or even stop the progression of AMKD.

“It’s exciting because these patients have no other options for therapy,” said Children’s pediatric nephrologist Daniel Feig, M.D., Ph.D. The trial is enrolling about 500 adult and pediatric participants, including about 150 children ages 10 to 18. More than 200 centers in the U.S. and other countries are involved.

2