Use of low-dose mitozantrone to treat aggressive multiple sclerosis: a single-centre open-label study using patient self-assessment and clinical measures of multiple sclerosis status

Background: There is significant evidence supporting the use of mitozantrone in the treatment of multiple sclerosis (MS) but few data on the subtypes of MS that respond or which measures of disease status are most useful. Aims: To assess the efficacy of low-dose (5 mg/m 2 3 monthly) mitozantrone using patient self-assessment questionnaire (SAQ), expanded disability status score (EDSS), multiple sclerosis functional composite score (MSFC), and the fatigue severity scale (FSS). Then, to compare the responses of a subgroup of relapsing remitting MS (RRMS) and secondary progressive MS (SPMS) patients to treatment, and to assess which measures of MS disease status are the most useful in a study of this type. Method: Thirty-one patients with definite (McDonald criteria) active MS were commenced on mitozantrone 5 mg/m 2 every 3 months. EDSS, MSFC and FSS data collected before treatment and after 12 months were analysed. The SAQ was administered after at least 12 months of therapy. Results: RRMS patients showed significantly more response to mitozantrone than SPMS patients in terms of MSFC (P = 0.02) and SAQ (P = 0.01). Conclusions: Low-dose mitozantrone was well tolerated and useful in active RRMS in the short term; however, mitozantrone did not display any useful activity in SPMS patients over this time interval or at the mitozantrone dose used. Patient perception of treatment is a worthwhile outcome measure and the MSFC is the most useful objective measure of MS status change in this type of study.