Journal of the neurological sciences
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Evidence for the effectiveness of immunosuppressive agents in MS is scanty. There are few good quality trials; most have methodological limitations, such as a small sample size and short duration. ⋯ Although azathioprine appears to reduce the relapse rate in MS patients, its effect on disability progression has not been demonstrated. Methotrexate may alter the course of disease favourably in patients with progressive MS, but the evidence is again sparse.
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The recent publication of the MIMS-study (Mitoxantrone In Multiple Sclerosis) and the approval of several health authorities support the use of mitoxantrone in patients with active relapsing-remitting or secondary progressive multiple sclerosis. This review provides information on data thus far present on the putative mechanisms of action of mitoxantrone in the immunopathogenesis of this disabling disease.
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Based on the encouraging results of transplantation in animals with experimental autoimmune encephalomyelitis (EAE), small-scale phase I/II trials of autologous hematopoietic stem cell transplantation (autoHSCT) were initiated in 1995 for the treatment of severe cases of multiple sclerosis (MS). More than 200 patients with treatment-resistant multiple sclerosis have been transplanted so far, mainly in Europe and the USA. The results of these studies appear promising in terms of impact on MRI disease parameters and, to a lesser extent, clinical stabilization or even improvement. Despite concerns raised by the morbidity and mortality noted in the initial pilot studies, a controlled, randomized, phase III trial of autoHSCT against the best currently available treatment, i.e., mitoxantrone, seems justified and is under way.