Authors' objectives:

This report reviews the evidence of the value of mitoxantrone in the treatment of multiple sclerosis (MS), estimates the direct costs to the MUHC of such treatment, and formulates recommendations concerning its use in the MUHC for the treatment of the relapsing-remitting, and secondary progressive forms of the disease.

Authors' results and conclusions: Evidence of its benefit is based on three randomized clinical studies. These are consistent in providing evidence of a beneficial effect of mitoxantrone on the progression of MS. Over the short term there is a reduction in attack rate, a reduction in the rate of development of new cerebral lesions detected by MRI, and a reduction in the number of patients who experience deterioration in function. However, the amount by which progression of disability can be retarded is not yet clear. In the biggest study (188 subjects) with the longest follow-up (3 years), although fewer treated individuals experienced functional deterioration, there was no significant difference between the average change in disability levels from baseline, between the treated and control groups. It is still too early to know whether those benefits that are experienced during treatment will persist. Compared to other forms of chemotherapy, mitoxantrone has relatively few side effects. Patients receiving high doses are at risk of cardiomyopathy, but at the dosage levels envisaged in the current treatment protocol for multiple sclerosis treatment this risk is low. There is concern that mitoxantrone use may increase the risk of developing malignancies.

Authors' recommendations: There is relatively good evidence that treatment with mitoxantrone can be expected to reduce the relapse rate and the rate of clinical deterioration, as well as MRI evidence of diminished CNS activity, at least during the course of treatment. The clinical benefits to be expected, although not very substantial and not yet shown to be permanent, are still sufficient to justify offering patients with very active forms of MS, similar to those in reported studies, the possibility of treatment. In view of the above, and in light of the present budget situation, it is recommended that a programme limited to 20 new enrollments per year should be approved at this time. This decision should be reviewed in one year in light of the experience accumulated, and of any new evidence concerning benefits and side effects of mitoxantrone and of competing treatments.

Authors' methods: Review

Project Status: Completed

URL for project: http://www.mcgill.ca/tau/publications/2002

Year Published: 2002

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Canada

Organisation Name: Technology Assessment Unit of the McGill University Health Centre (MUHC)

Contact Address: Technology Assessment Unit of the MUHC, Centre for Outcomes Research and Evaluation (CORE), Research Institute of the McGill University Health Centre, 5252 boul. de Maisonneuve, Bureau 3F.50, Montreal, Quebec H4A 3S5

Contact Name: nandini.dendukuri@mcgill.ca

Contact Email: nandini.dendukuri@mcgill.ca

Copyright: Technology Assessment Unit of the McGill University Health Centre (MUHC)