Treating Pemphigus Vulgaris with Prednisone and Mycophenolate Mofetil: A Multicenter, Randomized, Placebo-Controlled Trial

Abstract

Non-blinded trials of pemphigus vulgaris suggest that mycophenolate mofetil (MMF) may be beneficial. In a prospective, multicenter trial, outpatients with mild or moderate pemphigus vulgaris were randomized to MMF (2 or 3 g day(-1)) plus oral corticosteroids or placebo plus oral corticosteroids for 52 weeks. The primary end point was the proportion of patients in the placebo and combined MMF groups responding to treatment (absence of new, persistent oral or cutaneous lesions, and prednisone dose < or = 10 mg day(-1) from weeks 48 to 52). Of 96 randomized patients, 94 were given treatment and 75 completed the study. Treatment responses occurred in 40 of 58 patients (69.0%) in the combined MMF group and 23 of 36 (63.9%) in the placebo group (P=0.6558, 95% confidence interval -17.4 to 27.6). MMF-treated patients showed faster and more durable responses. In post hoc analyses, more patients taking MMF showed sustained responses for 3 or 6 months than did placebo patients. MMF was well tolerated. Although MMF did not show an advantage on the primary end point, there seemed to be a beneficial treatment effect on several secondary end points, including time to response and duration of response. Thus, MMF may be a potentially useful agent in patients with mild or moderate pemphigus vulgaris.

Efficacy of Dapsone in the Treatment of Pemphigus Vulgaris: A Single-Center Case Study

“…It is noteworthy that in this retrospective series, which is one of the largest reported to date, we evaluated dapsone's efficacy in 26 patients with PV using clinical observation end points defined by the International Pemphigus Committee [9] . The trend of efficacy demonstrated in the current study is similar to that reported in the past for other adjuvants such as mycophenolate mofetil [3] , methotrexate [21] , and others.…”

“…Although dapsone can be a reasonable nonimmunosuppressant steroid-sparing agent for some patients, at least for a limited period of time, and despite the current results as well as previous results indicating dapsone's efficacy in PV, its use is debatable in light of newer drugs, which are efficacious and well tolerated [3,4] . Dapsone should be an accessible tool in the armamentarium of PV treatments as it does not cause immunosuppression.…”

“…This reduction in the daily dose of prednisone is statistically signifi- 2 The mean Hb reduction in the study group was 1.3 mg/dl. 3 Three patients did not complete due to treatment failure (as defined by the consensus statement) after 3 months but before 6 months, 2 due to remission at 4 months, and 1 due to adverse effects at 4 months (headaches). 581 cant (p < 0.001).…”

Mycophenolate Mofetil for the Management of Autoimmune Bullous Diseases

“…The patients given MMF showed faster and more durable responses, but the lack of difference in the response rate may have been attributable to the milder disease of the placebo group, which may not have needed the additional immunosuppressive therapy. Similarly to the results reported by Powell et al [ 59 ], the 2 and 3 g/day doses of MMF displayed similar effi cacy, but infectious adverse events were more frequent with the higher dose [ 63 ].…”

Current Management of Pemphigus

“…Prospektif kontrollü bir çalış-mada iki adjuvan yani azatioprin ve mikofenolat mofetil karşılaştırılmış ve birikmiş kortikosteroid dozu, etkinlik ve yan etkiler açısından bir fark gözlenmemiştir (17). Bir çalışmada oral kortikosteroide adjuvan olarak mikofenolat mofetil eklenmesinin plasebo eklenmesine göre daha yararlı olduğu, başka bir çalışmada ise bu kombinasyonun kortikosteroidin tek başına kullanımına göre üstün olmadığı bildirilmiştir (11,18).…”