Erythropoietin fails to improve outcomes for newborns with hypoxic-ischemic encephalopathy

Erythropoietin, a treatment for newborns with critically low levels of oxygen or blood supply to the brain at birth, does not prevent death or disability, according to a new multinational study. The research will be presented at the Pediatric Academic Societies (PAS) 2025 Meeting, held April 24-28 in Honolulu. 

Researchers found that a high-dose treatment with erythropoietin, paired with standard cooling treatments, does not reduce death, rate of cerebral palsy, or physical or cognitive impairment for newborns with hypoxic-ischemic encephalopathy (HIE).

HIE, which leads to brain damage, is caused by labor and delivery problems, or problems with how the placenta is functioning just before or during birth.

The randomized trial (PAEAN) studied 311 babies born with HIE at or after 35 weeks in Australia, New Zealand, and Singapore. Researchers compared outcomes at age two between babies treated only with controlled cooling of the baby to a few degrees below normal, and those treated with both erythropoietin and cooling in the first days after birth.

Our research supports the findings of the HEAL study, published by Dr. Yvonne Wu and her colleagues in 2022, which also indicated no benefit of erythropoietin for babies receiving cooling for HIE. The PAEAN study increases the certainty of evidence from HEAL, and together, they do not support prior studies showing erythropoietin could improve outcomes, which brought hope to lower-income countries where access to intensive treatment is not assured. Hypoxic-ischemic encephalopathy is a leading cause of death and disability among newborns, and the search for ways to prevent, detect, and treat it remains critical."

Helen G. Liley, MBChB, FRACP, professor at the University of Queensland in Australia and presenting author

Between one and five babies per 1,000 born in countries with high health care resources have HIE, while rates are significantly higher in countries with fewer resources for prenatal care and birth, according to researchers.