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Background and Purpose Te aim of this study was to determine the efcacy and tolerability of granulocyte colony-stimulating factor (G-CSF) in subjects with amyotrophic lateralsclerosis (ALS). Methods Forty subjects with ALS were randomly assigned to two groups, which received either subcutaneous G-CSF (5 μg/kg/q12h) or placebo for 5 days. Te subjects were then followed up for 3 months using the ALS Functional Rating Scale-Revised (ALSFRS-R), manualmuscle testing, ALS Assessment Questionnaire-40, and nerve conduction studies. CD34+/CD133+ cell count and monocyte chemoattractant protein-1 (MCP-1) levels were evaluated atbaseline. Results Te rate of disease progression did not difer signifcantly between the two groups. Te reduction in ALSFRS-R scores was greater in female subjects in the G-CSF group than intheir counterparts in the placebo group. Tere was a trend toward a positive correlation between baseline CSF MCP-1 levels and the change in ALSFRS-R scores in both groups (Spearman’s ρ=0.370, p=0.070). Conclusions With the protocol implemented in this study, G-CSF is not a promising optionfor the treatment of ALS. Furthermore, it may accelerate disease progression in females.

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