TREATMENT OF FULMINANT MULTIPLE SCLEROSIS WITH INTRAVENOUS CYCLOPHOSPHAMIDE

BACKGROUNDThe role of intravenous (IV) cyclophosphamide (CTX) for patients with progressive multiple sclerosis (MS) remains controversial, despite numerous controlled and uncontrolled clinical trials. OBJECTIVETo present the results of open label treatment with CTX and intravenous methylprednisolone (MP) in 17 consecutive patients with corticosteroid-resistant, fulminant MS. METHODOpen-label, nonblind, noncontrolled consecutive series of patients meeting the following criteria were treated with CTX and MP. Fulminant MS was defined as objectively documented, continuous deterioration by ≥1.5 points on the Kurtzke Expanded Disability Status Scale (EDSS) for ≥ 3 months. Corticosteroid resistance was defined as continued deterioration for 2 months after high-dose IV MP. After completion of the CTX/MP treatment protocol, all patients were subsequently treated with maintenance immunotherapy at the discretion of their treating neurologist. Each patient was followed by a single neurologist, who determined the EDSS according to a standard protocol at successive visits. RESULTSBaseline EDSS scores at the time of CTX/MP treatment ranged from 6.0 to 8.5. There were no serious complications of treatment. Follow-up time ranged from 12 to 24 months (median 24 months, mean 22 months). At 12 months, 1 3 of 1 7 (75.5%) patients were stable or improved by ≥1.0 EDSS point. At 24 months, 9 of 13 (69%) of patients were stable or improved. CONCLUSIONThis consecutive case series suggests that CTX/MP represents an effective therapeutic option for those rare MS patients with a fulminant progressive course who are refractory to corticosteroid therapy. The limitations related to the uncontrolled nature of the study and comparisons with other CTX studies are discussed.