Several retrospective studies and small clinical trials have found a neuroprotective role for recombinant human erythropoietin (rhEPO) treatments for very preterm infants. However, a phase 3 randomized, double-blind, placebo-controlled, multi-center trial, recently published in JAMA, found no significant difference in cognitive development at 2 years of age between 228 preterm infants who prophylactically received early, high-dose rhEPO treatments compared to 220 preterm infants who received a saline placebo (P=0.56). Furthermore, no differences in morbidity, mean body weight, length or head circumference were found between the two groups. Data from this trial suggest that rhEPO does not have a neuroprotective effect. This finding may differ from previous studies in which rhEPO administration was started later with lower doses over a longer duration. Protective effects of rhEPO may also be more pronounced in school-aged children than 2 year olds. The results of two large ongoing trials assessing rhEPO treatment may help to clarify its neuroprotective effects.
Natalucci G, Latal B, Koller B, Ruegger C, Sick B, Held L, Bucher HU, Fauchere JC, Swiss EPONTG. Effect of Early Prophylactic High-Dose Recombinant Human Erythropoietin in Very Preterm Infants on Neurodevelopmental Outcome at 2 Years: A Randomized Clinical Trial. JAMA 2016;315: 2079-85.