PHILADELPHIA – Premature babies who are enrolled in clinical trials for therapies to treat and prevent complications from preterm birth are no more likely to die or experience poor outcomes than babies who are not trial participants, according to a retrospective analysis of more than 5,000 babies born before 29 weeks of gestation. Results of the study, led by researchers at the Perelman School of Medicine at the University of Pennsylvania, are published this week in JAMA.
“Performing randomized trials is the only way we can know with confidence whether the therapies we use in preterm babies are safe and effective,” said the study’s lead author, Elizabeth E. Foglia, MD, a neonatologist and instructor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and the Children’s Hospital of Philadelphia (CHOP). “We studied over 5,000 premature babies who were eligible for clinical trials spanning many years, and found no differences in the rates of death or other serious medical outcomes between infants who participated in trials and those who did not. Our findings provide reassurance that trial participation itself is not detrimental to this small and vulnerable patient population.”
The analysis included six studies that took place between 1999 and 2012. Investigated therapies included phototherapy (light therapy for jaundice), glutamine supplementation to prevent infections, nitric oxide to treat respiratory failure, timing of umbilical cord clamping, continuous positive airway pressure (a form of respiratory support) immediately after birth, and Vitamin E supplementation.
There were a total of 5,389 infants who were eligible for at least one of six clinical trials, 3,795 of whom were enrolled in at least one randomized clinical trial, and 1,594 who were not enrolled in a trial. The researchers found that 68 percent of the enrolled group and 69 percent in the group that did not enroll either died or experienced at least one major complication of prematurity: bronchopulmonary dysplasia (a chronic lung disease in preterm infants), severe brain injury, or severe retinopathy of prematurity (a sight-threatening disorder of the eye).
There were also no differences observed in rates of sepsis (a potentially life-threatening complication of an infection), and necrotizing enterocolitis (severe gut injury and inflammation). The authors note that their results are similar to findings of studies of adults and older children, which also demonstrated no significant differences in outcomes between clinical trial participants and nonparticipants.
Barbara Schmidt, MD MSc and Sara DeMauro, MD MSCE of Penn Medicine and CHOP are co-authors, as are Tracy Nolen, DrPH, Abhik Das, PhD, Edward Bell, MD, and Barbara Stoll, MD.
The National Institutes of Health and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) provided grant support for Neonatal Research Network’s Generic Database Study via cooperative agreements.
Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation’s first medical school) and the University of Pennsylvania Health System, which together form a $4.9 billion enterprise.
The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 17 years, according to U.S. News & World Report‘s survey of research-oriented medical schools. The School is consistently among the nation’s top recipients of funding from the National Institutes of Health, with $409 million awarded in the 2014 fiscal year.
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