N Engl J Med 2021 06 19;384(24):2295-2305. Epub 2021 May 19.
From the Perelman School of Medicine, University of Pennsylvania, and the Corporal Michael J. Crescenz Veterans Affairs Medical Center, Philadelphia (V.P.W.); the Department of Dermatology, Rouen University Hospital and INSERM 1234, Normandie Université, Rouen (P.J.), and the Department of Dermatology, Groupe Hospitalier Paris Seine-Saint-Denis, Assistance Publique-Hôpitaux de Paris and INSERM Unité Mixte de Recherche 1125, Bobigny (F.C.) - all in France; the Sackler School of Medicine, Tel Aviv University, Tel Aviv, and Rabin Medical Center, Petah Tikva - both in Israel (D.M.); the Department of Dermatology, University of California, Davis, School of Medicine, Sacramento (E.M.), and Genentech, South San Francisco (A.K., D.M.C.) - both in California; Roche Products, Welwyn Garden City, United Kingdom (P.L.); F. Hoffmann-La Roche, Basel, Switzerland (L.G.); and Roche Products, Mississauga, ON, Canada (P.P.).
Background: Rituximab and mycophenolate mofetil are used to treat pemphigus vulgaris, but they have not been adequately compared in clinical trials.
Methods: In a randomized, controlled trial, we assigned patients with moderate-to-severe pemphigus vulgaris in a 1:1 ratio to receive intravenous rituximab (1000 mg on days 1, 15, 168, and 182) or oral mycophenolate mofetil (2 g per day), in addition to an oral glucocorticoid administered on the same tapering schedule in the two groups. The primary end point was sustained complete remission at week 52, defined as the healing of lesions with no new active lesions, as reflected by a Pemphigus Disease Area Index (PDAI) activity score of 0 (on a scale of 0 to 250, with higher scores indicating greater disease severity), for at least 16 weeks without the use of glucocorticoids. Read More