Efficacy and safety of mitoxantrone use in primary and secondary progressive multiple sclerosis - study site experience based on the therapy of 104 patients.

Mitoxantrone (MX) is used in patients with primary and secondary progressive as well as relapsing-remitting type of multiple sclerosis (PPMS, SPMS, RRMS). The objective of our project was to evaluate the efficacy and safety of MX use in patients with PPMS and SPMS.

METHODS: The retrospective study included 104 patients (mean age 54.2 ± 9.0), with PPMS (13.46%) and SPMS (86.54%) treated with MX. During single cycle of the MX therapy a dose of 12 mg/m2 of body surface area was administered and next cycles every three months up to a total dose of 140 mg/m2 were realized.

RESULTS: The course of the therapy was completed by 95 patients (91.34%) including 73 patients who received a scheduled whole dose. The average cumulative dose per patient was 75.2 mg/m2 . Thirty-two patients reported nausea after MX administration, 20 revealed increase in the incidence of infection and 19 patients hair loss. Twenty-two patients discontinued therapy (seven patients because of the progress of disability). Independent risk factors for deterioration were: age and the form of PPMS (RR 1.56 [95% CI: 1.17-2.07] and RR 2.8 [95% CI: 1.08-7.21], respectively). Five patients revealed a asymptomatic decrease in EF value <50% or 10% in relation to the previous test.

CONCLUSIONS: MX therapy enables us to stabilize the disease without causing any significant side effects in most patients with progressive disease as compared to patients with primary progressive disease with a comparable safety profile. Larger benefits of MX therapy are associated with the patients with secondary progressive disease.