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.