A research team at UT Southwestern Medical Center has been approved for a five-year, $7 million funding award from the Patient-Centered Outcomes Research Institute (PCORI). The funding will support multisite clinical research on hypothermia therapy, a cooling treatment that aims to help protect the brains of babies deprived of oxygen at birth.
“I am grateful for this PCORI funding that will allow us to address important questions to improve patient care in vulnerable populations,” said Lina Chalak, M.D., Professor of Pediatrics at UT Southwestern and interim Division Chief of Neonatal-Perinatal Medicine at Children’s Health, who will lead the project. “This will be the first study to address the effectiveness of cooling for infants suffering mild hypoxic-ischemic encephalopathy (HIE), a previously unstudied population at risk of brain injury. I look forward to working with families who will participate in the design and dissemination of the trial.”
Every year, millions of newborns worldwide receive brain damage caused by this oxygen deprivation, a condition known as neonatal HIE. Although the World Health Organization estimates that birth asphyxia is responsible for nearly a quarter of all neonatal deaths, those babies that survive oxygen deprivation are often left with neurological injuries, especially those infants who do not receive targeted treatment and follow-up, Dr. Chalak explained.
To help improve outcomes, babies diagnosed with moderate and severe HIE are treated with therapeutic hypothermia, using a cooling blanket that brings the body temperature down to as low as 33.5 C (92.3 F) – a treatment implemented 15 years ago by the National Institutes of Health’s (NIH) Neonatal Research Network, of which UT Southwestern has long been a member.
Dr. Chalak has focused her clinical and translational research on the prevention of neonatal brain injury and optimization of HIE outcomes, supported by NIH funding and collaborations with nationally funded networks including the National Institute of Neurological Disorders and Stroke and the Neonatal Research Network.
Randomized trials targeting infants with moderate to severe HIE have shown that therapeutic hypothermia reduces death and disability in this population. Research published by Dr. Chalak and her team last year showed that HIE-affected babies treated with hypothermia therapy are at an elevated risk of seizures and brain damage during the rewarming period, which could be a precursor of disability or death.
The new study will examine the use of hypothermia therapy for patients with mild HIE by comparing the effectiveness of hypothermia to room temperature on neurodevelopmental outcomes at two years; determine the adverse effects of hypothermia therapy on the infant and his/her family; and determine the heterogeneity of treatment effects across mild HIE subgroups as determined by physiological biomarkers. The clinical trial aims to include 460 children at 15 sites across the United States, with UT Southwestern as the primary site. In Dallas, infants will be enrolled for the study at UTSW and Parkland Health, with follow-ups at Children’s Health.
“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other health care stakeholders in a study conducted in real-world settings, but also for its potential to answer important questions about hypothermia therapy and fill a crucial evidence gap,” said PCORI Executive Director Nakela L. Cook, M.D., M.P.H. “We look forward to following the study’s progress and working with UT Southwestern to share its results.”
Dr. Chalak’s award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better informed health care decisions.
About UT Southwestern Medical Center
UT Southwestern, one of the nation’s premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institution’s faculty has received six Nobel Prizes, and includes 26 members of the National Academy of Sciences, 17 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,900 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in more than 80 specialties to more than 100,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 4 million outpatient visits a year.
