Improving the health of premature babies through support for clinical research
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 from across the country that conduct clinical research to investigate the safety and effectiveness of treatments for sick or 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 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 statistical 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 birth asphyxia, which can lead to severe brain damage. We not only provided the statistical design for the study, but we also implemented randomization as well as data management systems and participated in the data analyses.
In addition, we 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 a similar 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. We recently supported a genomics study to identify genetic variations like bronchopulmonary dysplasia (BPD), a chronic lung disease found in infants.
Relying on our multidisciplinary expertise, we also develop tools for the NRN such as the Neonatal BPD Outcome Estimator, which predicts the risk of BPD and Very Low Weight Postnatal Growth Charts that provide individualized expected growth curves.
Research Findings that Save Lives
To date, we have supported more than 200 NRN studies aimed at understanding and improving health practices associated with premature births. Findings from these studies have led to changes in care practices and improved outcomes for high-risk infants. These findings include, for example, identifying that steroids improve survival and reduce the chances of certain birth defects for extremely premature infants—even if the treatment course is not finished before delivery—and that increased oxygen can raise the chances for survival in babies born prematurely.
RTI continues to collaborate as part of the NRN—developing studies to examine therapies for sepsis, anemia, and lung disease, and studying the effects of donor human milk compared to formula in improving developmental outcomes for extremely premature babies.
