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.
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