Short-term effects of erythropoietin on neurodevelopment in infants with cerebral palsy: a pilot study

Objective: Cerebral palsy (CP) is a disabling condition characterized by the motor impairment, which is difficult to be ameliorated. In the brain of infants with CP, there are persistent pathomechanisms including accentuated neuroinflammation. Since erythropoietin was demonstrated to have neuroprotective effect via anti-inflammatory and anti-apoptotic properties, we hypothesized that the administration of recombinant human EPO (rhEPO) could help children with CP, especially young infants.

Materials and method: We investigated the therapeutic efficacy of rhEPO for infants with CP, who had been undergoing active rehabilitation in hospitalized setting to eliminate treatment bias. Twenty infants with CP were randomly divided into EPO or control group equally. We compared the changes in the Gross Motor Function Measure (GMFM) and the Bayley Scales of Infant Development-II (BSID-II) scores during one month of hospitalization between two groups.

Results: The improvements after 1 month on the GMFM A and GMFM total scores differed significantly between the groups (p = 0.003, p = 0.04, respectively). However, the changes after 6 months were not different between the two groups. The scores of BSID-II did not show any differences at 1-month and 6-months post-treatment.

Conclusion: These results indicated that rhEPO could have therapeutic efficacy for infants with CP during the active rehabilitation and anti-inflammation was suggested to be one of its therapeutic mechanisms.