Objective: To investigate the safety, tolerability, and short-term efficacy of treatment with erythropoietin in patients with optic neuritis as a first demyelination event.
Methods: We conducted this randomized double-blind pilot study in the Shiraz University of Medical Sciences, Shiraz, Iran, from March 2007 to January 2009. The participants were patients aged 18-45 years with optic neuritis and at least 3 hyperintense lesions on T2-weighted and FLAIR MRI, but no clinically definite multiple sclerosis (MS). They were randomized into 2 groups. The case group (5 patients) received intravenous methyl prednisolone (1000 mg/24 hours) and intravenous erythropoietin (20,000 unit/24 hours) for 5 consecutive days, and the control group (5 patients) received intravenous methyl prednisolone at the same dose as the case group, and a placebo. The groups were followed for one year and compared for adherence to protocol, adverse drug effects, mean duration of conversion to clinically definite MS, and MRI changes.
Results: All patients tolerated the protocol. One patient who received erythropoietin developed cerebral venous sinus thrombosis and anti-cardiolipin antibody positivity. One patient in the control group, but no patients in the case group, fulfilled the McDonald criteria for MS during the follow-up period, but none of the participants in either group developed clinically definite MS according to the Poser criteria.
Conclusion: Erythropoietin may be effective, but should be used with caution.