Mycophenolate mofetil in combination with interferon beta-1a in the treatment of relapsing-remitting multiple sclerosis: a preliminary study

Background: the efficacy of mycophenolate mofetil (mmf) plus interferon beta-1a (ifnb-1a) in treatment of relapsing- remitting multiple sclerosis (rrms) was evaluated. methods: this was a pilot study with randomized, double-blinded, placebo-controlled design. patients with rrms and expanded disability status scale score (edss) of < 6.0 were included. those with relapse within the previous two months and prior use of immunomodulatory/immunosuppressive drugs were excluded. patients were randomized into mmf (n = 13) and placebo (n = 13) groups and received weekly intramuscular ifnb-1a plus either mmf or placebo. mmf started by 500 mg/d for one week and weekly escalated by 500 mg/d, until target divided dose of 2000 mg/d and continued for 12 months. radiologic and clinical assessments were performed at baseline and then at month 12. results: after one year of therapy, difference between the two groups in number of new t2 lesions was not statistically significant (0.54 ± 0.77 in mmf vs. 1.85 ± 3.2 in placebo group, p = 0.169). two patients in the placebo group had gadolinium-enhanced lesions and one patient had relapse. there were 3 patients in each group with more than one point progression in edss. common side effect in the mmf group included gastrointestinal upset, but no patient discontinued the treatment. conclusions: combination of mmf with ifnb-1a in patients with rrms is well tolerated, but the efficacy of such combination was not statistically significant in this pilot study and deserves further investigation with a larger sample size and a longer follow-up.