Effect of open label pulse cyclophosphamide therapy on MRI measures of disease activity in five patients with refractory relapsing-remitting multiplesclerosis

Objective. To evaluate the response to cyclophosphamide (CTX) of five patie nts who failed an average three treatments with multiple other therapeutic agents, using serial monthly MRI measures. Methods. Five patients with rela psing-remitting multiple sclerosis (MS) and documented MRT disease activity were started on monthly pulse intravenous CTX at a dose of I g/m(2). CTX w as administered without an induction phase according to the protocol simila r to the treatment of lupus nephritis. The five patients were followed with monthly MRI and clinical evaluation for a mean of 28 months. Results. All the patients showed a rapid reduction in the contrast-enhancing lesion freq uency and in three patients there was a decrease in the T2, lesion load wit hin the first 5 months after starting CTX treatment. The administration of CTX during overnight hospitalization was safe and well tolerated. Conclusio ns. These findings suggest that aggressive immunosuppressive therapy may be useful in some rapidly deteriorating refractory patients and further contr olled study should be considered in order to full evaluate this type of tre atment as a potential therapy in MS. (C) 1999 Elsevier Science B.V. All rig hts reserved.