No Difference in Outcomes for Premature Infants Enrolled in RCTs

Jun 23 2015

No Difference in Outcomes for Premature Infants Enrolled in RCTs

premature infantsMany families of premature infants quickly become familiar with a powerful research tool called a randomized clinical trial (RCT). Neonatologists may invite them to participate in RCTs, as they investigate ways to help prevent, treat, and manage the myriad complications that can occur when these babies’ organs are not ready for life outside their mothers’ wombs.

In a RCT, study participants are randomly assigned to two or more groups, which helps to reduce any potential bias and compare research results. For example, one group receives a new drug being tested, and one group receives a placebo, no treatment, or a different drug. Parents, however, may wonder if the decision to include their newborns in a RCT could be associated with any detrimental differences in their outcomes.

A research letter published in JAMA by several neonatologists from The Children’s Hospital of Philadelphia and the University of Pennsylvania may help to alleviate these concerns. They reported results from a study of 5,000 extremely preterm infants and determined that important in-hospital outcomes, such as severe brain injury and a chronic lung disorder called bronchopulmonary dysplasia, were neither better nor worse in infants enrolled in RCTs compared with infants who were eligible but not enrolled in RCTs.

“These results are important, as they provide reassurance that participating in randomized trials is not detrimental to preterm infants,” said Elizabeth E. Foglia, MD, an attending neonatologist with the Division of Neonatology at CHOP, who co-authored the letter.

The new findings are in line with previous research of adults and older children that demonstrated no significant differences in outcomes between trial participants and nonparticipants who were treated similarly outside trials, the authors pointed out.

The researchers performed their analysis of trial enrollment and outcomes using data from six RCTs performed at National Institute of Child Health and Human Development Neonatal Research Network (NRN) sites between January 1999 and December 2012. All of the infants had outcomes recorded in the NRN’s very low-birth-weight registry.

“Many therapies that are commonly used in preterm and sick infants have never been rigorously tested, and novel interventions are being developed all the time,” said Dr. Foglia, who also is an instructor in Pediatrics at the Perelman School of Medicine, University of Pennsylvania. “The only way we can know with confidence that a given therapy is safe and effective in our patients is by performing well-designed and appropriately regulated randomized trials.”

Several RCTs involving premature infants are underway at The Children’s Hospital of Philadelphia, such as the Sustained Aeration of Infant Lungs Trial (SAIL) trial, which is looking at the standard of care for resuscitation of these babies at delivery. Another RCT is comparing two oral feeding schedules for premature infants, and researchers are collecting data on milk transfer, sucking strength, growth, and medical complications.

More information about making the decision to participate in a clinical trial is available in the Parents resources section of The Children’s Hospital of Philadelphia’s Research Institute’s website.

Read a press release for additional details about the JAMA research letter.