title: Mitoxantrone treatment in multiple sclerosis: a 5-year clinical and MRI follow-up.
abstract: Mitoxantrone (MTX) is an antineoplastic agent approved for treatment of secondary progressive and rapidly worsening relapsing-remitting multiple sclerosis (MS). We designed a longitudinal open-label prospective study to evaluate the efficacy and toxicity of MTX over a 2-year treatment period with a further 3-year follow-up. Fifty consecutive MS patients were included and received MTX intravenously (8 mg/m(2) every 2 months for a total of 12 infusions). Efficacy was assessed clinically and by brain MRI performed before MTX therapy, at the end of treatment and at the end of each year of follow-up. Forty-nine patients completed the 5-year study, 44 (89.8%) completed the MTX course, five (10.2%) interrupted the treatment because of side effects. Fifteen (30.6%) patients showed Expanded Disability Status Scale (EDSS) progression on treatment and nine (18.4%) during follow-up. Seventeen (34.7%) patients had enhancing lesions at baseline, nine (18.4%) at the end of treatment, but none at the end of follow-up. In conclusion, we observed EDSS progression in about 1/3 of the patients during the treatment period and in 1/5 during the further 3-year follow-up period. This evidence suggests a delayed beneficial effect after MTX treatment is completed with only a minority of patients showing disability progression once the drug was suspended.
authors.affiliation: Department of Neurological Science, Neurological Unit, S. Andrea Hospital, University La Sapienza, Rome, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Buttinelli
authors.forename: A
authors.initials: A
authors.lastname: Clemenzi
authors.forename: G
authors.initials: G
authors.lastname: Borriello
authors.forename: F
authors.initials: F
authors.lastname: Denaro
authors.forename: C
authors.initials: C
authors.lastname: Pozzilli
authors.forename: C
authors.initials: C
authors.lastname: Fieschi
mesh_terms: D000328:Adult; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods*; D008297:Male; D008875:Middle Aged; D008942:Mitoxantrone / Q000627:therapeutic use*; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000188:drug therapy* / Q000473:pathology*; D011446:Prospective Studies
chemical_list: D008942:Mitoxantrone