Use of Mitoxantrone in Early Secondary Progressive Multiple Sclerosis: An Observational Study of 48 Patients with Clinical and MRI Outcomes

OBJECTIVE: To report the clinical follow-up, side effects, and magnetic resonance imaging (MRI) findings of mitoxantrone treatment in patients in the early phase of secondary progressive and exacerbating relapsing-remitting multiple sclerosis. METHODS: Patients that completed and/or were undergoing treatment between 2001 and 2008 were included in the study. All patients were definitive progressive multiple sclerosis patients and met ‘non-responder’ criteria for immunomodulatory the- rapy. They did not have active infection, cardiac disease, or any contraindication for immunosuppressive treatment. Baseline comp- lete blood count, liver function tests, and transthoracic echocardiography were performed. Mitoxantrone was given as induction the- rapy (1 dose per month for 3 months) and then as maintenance therapy (with 3 month periods, 12 mg/m2 until to the cumulative dose was reached). Patients were followed-up regularly for the appearance of any side effects. RESULTS: The study included 33 female and 15 male patients. Mean age was 36.6 ± 7.8 years, mean disease duration was 9.38 ± 4.8 years, mean expanded disease severity scale (EDSS) score was 5.8, and mean treatment duration was 11.16 ± 7.4 months. Fol- lowing the end of treatment, 17 patients were stabilized, 10 improved, and 14 progressed. There were no side effects observed du- ring or after treatment in 16 patients. Among the other 32 patients, the observed side effects were as follows: nausea (n= 18), per- manent leucopenia (n= 1), transient thrombocytopenia (n= 4), alopecia (n= 13), neutropenic fever (n= 1), and amenorrhea (n= 18). MRI follow-up was performed in a subgroup of patients and showed that the lesion loads were stabilized. CONCLUSION: We reported the efficacy, and clinical and MRI follow-up results of multiple sclerosis patients treated with mitoxantro- ne. Patient selection and therapy timing was very important for maximum efficacy. Patients that responded to mitoxantrone treat- ment benefited beginning with the induction phase and their disability was limited or improve