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Short-term effects of erythropoietin on neurodevelopment in infants with cerebral palsy: a pilot study
Hee Song Lee1, Junyoung Song1, Kyunghoon Min2
1Department of Rehabilitation Medicine, CHA University, Gyeonggi-do, South Korea.
Insights
Recombinant human erythropoietin (rhEPO) showed short-term benefits in motor function for infants with cerebral palsy (CP) during active rehabilitation. Further research is needed to confirm long-term effects and therapeutic mechanisms in CP treatment.
Area of Science:
- Neuroscience
- Pediatrics
- Pharmacology
Background:
- Cerebral palsy (CP) is a disabling motor impairment in infants.
- Neuroinflammation is a key pathological mechanism in infant CP.
- Erythropoietin (EPO) exhibits neuroprotective, anti-inflammatory, and anti-apoptotic properties.
Purpose of the Study:
- To investigate the therapeutic efficacy of recombinant human EPO (rhEPO) in infants with CP.
- To evaluate rhEPO's potential benefits during active rehabilitation.
- To explore rhEPO's anti-inflammatory mechanisms in CP.
Main Methods:
- A randomized controlled trial involving twenty infants with CP undergoing hospitalization and active rehabilitation.
- Infants were divided into two groups: rhEPO treatment and control.
- Outcomes were assessed using the Gross Motor Function Measure (GMFM) and Bayley Scales of Infant Development-II (BSID-II) at 1 and 6 months.
Main Results:
- Significant improvements in GMFM A and total scores were observed in the rhEPO group after 1 month compared to the control group (p = 0.003, p = 0.04).
- No significant differences were found between groups at the 6-month follow-up.
- Bayley Scales of Infant Development-II scores showed no significant differences between groups at either time point.
Conclusions:
- rhEPO demonstrates short-term therapeutic efficacy in improving motor function for infants with CP during active rehabilitation.
- Anti-inflammation is suggested as a potential mechanism of rhEPO's therapeutic action.
- Long-term efficacy and optimal use of rhEPO in CP management require further investigation.
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.

