Evaluation of mycophenolate mofetil as a steroid-sparing agent in pemphigus: a randomized, prospective study

Background Mycophenolate mofetil (MMF) has been lately proposed as one of the most promising steroid-sparing agent in pemphigus. Objective To compare effectiveness and adverse events of two different therapeutic protocols for pemphigus: methylprednisolone alone vs. the combination of methylprednisolone and Mycophenolate mofetil. Methods A randomized prospective non-blinded trial was performed in a tertiary care medical centre. Forty-seven of 52 initially evaluated patients with newly diagnosed pemphigus vulgaris or pemphigus foliaceous that had not previously been treated with systemic corticosteroids or immunosuppressive drugs were randomly assigned to treatment with either methylprednisolone (prednisone equivalent, 1 mg/kg) or methylprednisolone plus mycophenolate mofetil (3 g/day, 1.5 g twice daily). Patients were followed up for clinical outcome based on time required until the achievement of control of disease activity, induction of partial and complete remissions on or off minimal therapy, total amount of corticosteroids administered, frequency of relapses and development of side-effects and complications. Results The two groups were similar in terms of demographics and baseline disease characteristics. There was no difference between groups in any of the variables used to measure response to treatment or total amount of corticosteroids administered. Side-effects did not differ significantly. Conclusion Combination treatment with corticosteroids and mycophenolate mofetil, 3 g/day, offers no advantage over monotherapy treatment with corticosteroids in patients with pemphigus.