Impact
Improving the health of premature babies through support for clinical research
Updated
Each year, 1 in 10 babies in the United States is born prematurely and is at increased risk of health problems—including apnea, infections, and anemia. Almost 1 million premature babies will die due to these complications.
In addition to the emotional toll on parents and families, premature births cost the United States billions of dollars in health care and education costs.
Supporting a Network of Research Centers to Improve Treatments for Premature Infants
Since 1998, we have worked on behalf of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) to conduct research aimed at saving the lives of premature babies.
Formed in 1986, the NRN is a cooperative group of 15 academic medical centers comprising 39 Neonatal Intensive Care Units (NICUs) from across the country that conduct clinical research to investigate the safety and effectiveness of treatments for sick (often extremely premature) newborn babies. The NRN disseminates research findings to fellow practitioners and scientists with the goals of improving clinical practices and reducing deaths and illnesses associated with premature birth and other newborn diseases.
Providing Statistical Leadership and Essential Operational Support
We serve as the data coordinating center for the NICHD Neonatal Research Network (NRN). As a collaborator with NICHD and the clinical sites, our experts provide biostatistical leadership and operational support that are essential to NRN’s success.
We advance the scientific impact of the NRN by collaborating to develop, implement, and monitor rigorous multicenter studies. For example, we assisted with a study that found hypothermia is an effective therapy for neonatal encephalopathy, which can lead to severe brain damage. We developed the statistical design for the study, implemented randomization as well as data management systems, monitored patient safety during the study and led the data analyses.
We routinely develop innovative trial designs to address the unique challenges in neonatal trials. The rarity of some diseases affecting newborns often makes designing an appropriately sized study difficult. For example, when studying the effects of cooling to reduce the risk of moderate and severe hypoxic-Ischemic encephalopathy—brain injury due to asphyxia—in premature infants, a limited population of infants from which to enroll study participants posed a challenge. As a solution, we utilized a Bayesian design based on the previous trial in full-term infants to develop a reasonable and effective study with a limited sample size.
As the data coordinating center, we also combine trial data with data from secondary studies to answer important questions. For example, we used extensive registry data collected by the NRN to develop a risk calculator for bronchopulmonary dysplasia (BPD), a chronic lung disease in premature babies. We then applied this calculator to see if baseline risk of BPD modifies the effect of vitamin A therapy on death or BPD, using data from an old NRN trial. Importantly, we found that infants at lower risk benefited more from vitamin A therapy than those at higher risk, which has implications for clinical practice.
In addition to the BPD calculator, we have also developed other useful tools based on NRN data, such as the widely used Extremely Preterm Birth Outcomes Tool for babies born at the limits of viability, and the Neonatal Outcomes Trajectory Estimator to estimate outcomes among survivors at specific times during hospitalization in the NICU.
Research Findings that Save Lives
Our collaboration with the NRN has produced more than 400 coauthored publications aimed at understanding and improving health practices associated with premature births. These studies have been published in high-profile journals, such as NEJM, JAMA, Lancet, and Pediatrics, and findings from these studies are cited in policy statements, practice guidelines, and clinical reports by the American Academy of Pediatrics and the American Congress of Obstetricians and Gynecologists. This has led to changes in care practices and improved outcomes for high-risk infants in a variety of areas, including management of neonates at the threshold of viability, neonatal sepsis, use of surfactants, antenatal and postnatal steroids, developmental follow-up, neonatal encephalopathy, and breastfeeding.
RTI continues to collaborate as part of the NRN—developing studies to examine therapies for reducing death, brain injury, lung disease, and other rare conditions such as congenital diaphragmatic hernia in newborns.
