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High-Dose Cyclophosphamide Appears to Reduce Disease Activity, Disability in Patients With MS NEW YORK -- June 9, 2008 -- Treatment with high doses of cyclophosphamide appears to reduce disease activity and disability in individuals with aggressive multiple sclerosis (MS), according to an article that will appear in the August issue of Archives of Neurology. The article is available online today. Immune-related therapies to treat MS have been suboptimal because of the distinct autoimmune process in each of the 4 subtypes of MS. Cyclophosphamide has been used to treat MS with mixed results and is often used in combination with bone marrow transplantation. Chitra Krishnan, MHS, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland, and colleagues studied high doses of cyclophosphamide without bone marrow transplantation in a 2-year, open-label trial involving 9 patients (6 men, 3 women, mean age 35 years) with aggressive relapsing-remitting MS. The patients received cyclophosphamide 50 mg/kg QD intravenously for 4 consecutive days. Over an average of 23 months of follow-up, there were no deaths or unexpected, or serious, adverse events. The patients experienced an average 39.4% reduction in disability and an 87% improvement in scores on a composite test measuring physical and mental function. In addition, MRI showed a decrease in the average number of MS-related brain lesions, from 6.5 to 1.2 lesions. "High-dose cyclophosphamide treatment of patients with aggressive MS was safe and well tolerated and did not lead to excess morbidity or accelerated brain atrophy," the authors wrote. "Moreover, high-dose cyclophosphamide induced a functional improvement in most of the patients we studied. In many of those patients, the functional improvement was sustained through the length of the study (up to 24 months) despite the absence of any immunomodulatory therapies beyond the initial high-dose cyclophosphamide treatment." "This immunoablative regimen of cyclophosphamide for patients with aggressive MS is worthy of further study and may be an alternative to bone marrow transplantation," they concluded. SOURCE: Archives of Neurology E-mail this page to a friend or colleague! To print, use this version