QUESTIONS 1. Describe the treatment for pemphigus vulgaris.
What is mycophenolate mofetil?3. How did the study protocol affect the outcome of the study? 4. What were the results? 5. What were the conclusions and implications of the study?The results of well-performed randomized clinical trials (RCT) are considered the highest-quality evidence for altering or supporting clinical practice. Therapeutic options for the treatment of pemphigus vulgaris (PV) have evolved over the 50 years since the initial dramatic results with corticosteroids. Experts have no consensus for the optimal treatment for PV, which is a rare, potentially life-threatening disease (Mimouni and Nousari, 2003; Martin and Murrell, 2008). Indeed, a recent systematic review (Martin et al., 2009) discovered insufficient information for determining the safest and most effective treatment regimen. Therefore, an RCT is imperative to allow treatments to be thoroughly evaluated. In this issue, Beissert et al. (2010) report the results of a 1-year comparison of two doses of mycophenolate mofetil (MMF; 2 or 3 g/d) combined with corticosteroids and steroids alone in treating PV.The authors found no difference in the number of patients responding after 1 year between the treatment groups but found more side effects in the MMF-treated group, particularly in those treated with 3 g/d. Patients treated with MMF, however, were quicker to respond, had a shorter time to sustained response, and exhibited a longer time before relapse. MMF also resulted in steroid-sparing effects because the cumulative dose of prednisone was approximately 1 g less in MMF-treated patients. Unfortunately, this latter finding was not associated with decreased side effects or decreased morbidity during the study period. The implications of this study remain uncertain. Will dermatologists abandon (or at least use lower doses of) MMF, or will the reported secondary outcomes of faster response and steroid-sparing effects sustain its use? Through the following questions, we examine this paper, as well as the subject of PV treatment, in greater detail. For brief answers, please refer to the supplementary information online RefeRences beissert S, Mimouni D, Kanwar aj et al. (2010) Treating pemphigus vulgaris with prednisone and mycophenolate mofetil: a multicenter, randomized, placebo-controlled trial. J Invest Dermatol 130:2041-8 Martin LK, Murrell DF (2008) Treatment of pemphigus: the need for more evidence. Arch Dermatol 144:100-1 Martin LK, Werth V, Villanueva E et al. (2009) Interventions for pemphigus vulgaris and pemphigus foliaceus. Cochrane Database Syst Rev CD006263 Mimouni D, Nousari CH, Cummins DL et al. (2003) Differences and similarities among expert opinions on the diagnosis and treatment of pemphigus vulgaris.
