Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis: A Pilot Study of Safety and Feasibility

Title
Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis: A Pilot Study of Safety and Feasibility
Author
김주한
Keywords
erythropoietin; pilot study.; amyotrophic lateral sclerosis
Issue Date
2014-10
Publisher
대한신경과학회
Citation
Journal of Clinical Neurology , Vol.10 No.4 [2014] ,342-347(6쪽)
Abstract
Background and Purpose It has been shown that erythropoietin is neuroprotective in animalmodels of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). The aim ofthis study was to determine the safety and feasibility of repetitive high-dose recombinant humanerythropoietin (rhEPO) therapy in ALS patients.Methods Two consecutive studies were conducted. We first recruited 26 subjects for an initialsingle-arm safety study. After a lead-in period of 3 months to assess the disease progression, rhE-PO was infused intravenously (35,000 IU) once per month for 3 months, and the subjects werefollowed for an additional 3 months. The ALS Functional Rating Scale-Revised (ALSFRS-R)was used for clinical assessment. After confirming the safety of rhEPO, 60 subjects were recruited for the second controlled study (rhEPO and control groups), which involved a total of 6 infusions at a rate of 1/month.Results There were no serious adverse events in the first study. The mean rate of decline in theALSFRS-R score was lower during the treatment period than during the lead-in period (mean±SD: 2.6±1.8 and 3.7±2.6, respectively; p=0.02). However, the rate of decline during the subsequent 3 months returned to that observed in the lead-in period. In the second study, the mean rateof decline in ALSFRS-R score was significantly lower in the rhEPO group than in the controlgroup (during months 0?3, 1.8±1.7 vs. 3.1±2.3, p=0.03; during months 4?6, 2.1±2.2 vs. 3.5±2.3,p=0.02).Conclusions Intravenous high-dose rhEPO is both safe and feasible for the treatment of ALS.Further investigation using different intervals and doses should be considered.
URI
https://synapse.koreamed.org/DOIx.php?id=10.3988/jcn.2014.10.4.342http://hdl.handle.net/20.500.11754/51074
ISSN
1738-6586; 2005-5013
DOI
10.3988/jcn.2014.10.4.342
Appears in Collections:
COLLEGE OF MEDICINE[S](의과대학) > MEDICINE(의학과) > Articles
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