Abstract
Objective: To evaluate the clinical and radiological outcome in patients with aggressive MS following treatment with mitoxantrone (MX).
Background: Mitoxantrone is one of the treatment options for patients with aggressive or refractory multiple sclerosis. In most cases MX was used for relapsing –remitting MS (RRMS) with frequent and disabling relapses and for rapid secondary progressive MS (SPMS).
Design/Methods: This retrospective study included mitoxantrone-treated patients (n=21) diagnosed with worsening RRMS, SPMS, or primary progressive MS. All received intravenous (IV) MX infusion every 3 months at a dose of 12mg/m2 body surface area per infusion plus 1g IV methylprednisolone. Patients regularly received cardiac monitoring before and during the treatment phase. Twenty-one MX-treated MS patients (14 females, 7 males) with mean age 43.5 years (range, 25–61 years), 4 had RRMS, 2 had PPMS, and 15 had SPMS. Mean disease duration to MX initiation was 11.7 years.
Results: Ten years following MX treatment 3 of our patients had died, 3 developed cancer, 5 were clinically improved, 4 had little change, and 12 continued deterioration. Four out of 5 patients with improvement had RRMS with gadolinium positive MRI lesions. One patient had transient atrial fibrillation.
Conclusions: Mitoxantrone was an effective and safe treatment in selected cases of rapidly progressive RRMS with active MRI lesions. Evidence for benefit in progressive forms of MS without MRI activity was lacking.