Sandra E. Juul
Treatment with high-dose erythropoietin, or EPO, is not associated with a lower risk for death or severe neurodevelopmental impairment at 2 years of age in extremely preterm infants, according to phase 3 trial results published in The New England Journal of Medicine.
The study was the first to explore the benefits of EPO therapy in extremely preterm infants. Previous, preclinical models of neonatal brain injury had shown a neuroprotective effect of EPO.
“[EPO] is not efficacious for neurologic purposes but is for erythropoietic purposes,” Sandra E. Juul, MD, PhD, neonatologist at Seattle Children’s Hospital, told Healio. “EPO may be used safely to decrease blood transfusions in extremely preterm infants, but its use does not prevent the neurologic consequences of extreme prematurity.”
Juul and colleagues conducted a multicenter, double-blind, randomized trial that included 741 infants born at 24 weeks to 27 weeks and 6 days of gestation, 376 of whom received EPO and 365 of whom were given a placebo within 24 hours of birth. The primary outcome was death or severe neurodevelopmental impairment — defined as severe cerebral palsy or a composite motor or cognitive score of less than 70 on the Bayley Scales of Infant and Toddler Development third edition — within 22 to 26 months of postmenstrual age.
Juul and colleagues observed no significant differences between the groups in incidences of severe neurodevelopmental impairment or death (26% vs. 26%; RR = 1.03; 95% CI, 0.81-1.32) at 2 years of age. Additionally, they observed no significant differences in rates of retinopathy of prematurity, sepsis, intracranial hemorrhage, bronchopulmonary dysplasia, necrotizing enterocolitis or in the frequency of serious adverse events.
Juul noted that the lack of EPO efficacy in reducing neurodevelopmental impairment was surprising.
“My hope, based on robust preclinical trials in multiple animal models, was that EPO would be neuroprotective,” Juul said. “Unfortunately, there are no great animal models of encephalopathy of prematurity mimicking the long-term stresses of the NICU stay.” – by Eamon Dreisbach
Disclosures: Juul reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.