Rituximab and Pemphigus — A Therapeutic Advance

Pemphigus vulgaris and pemphigus foliaceus are rare autoimmune blistering diseases of obscure causes. (...) The autoimmune nature of pemphigus was used as a rationale for the introduction of immunosuppressive drugs and plasmapheresis, both aimed at eliminating pathogenic autoantibodies from the patient.(...)During the past 10 years we have witnessed the arrival of new and exciting therapies for pemphigus. Infusions of intravenous immune globulins and B-cell–targeted humanized monoclonal antibodies are two examples. Rituximab is an anti-CD20 chimeric monoclonal antibody approved by the Food and Drug Administration for the treatment of B-cell non-Hodgkin's lymphoma and rheumatoid arthritis that is refractory to anti–tumor necrosis factor . (...) The article by Joly et al. supports previous reports that describe the beneficial effects of rituximab in the treatment of pemphigus without the need for infusions of intravenous immune globulins; however, its use must be restricted to a limited number of patients with pemphigus vulgaris or pemphigus foliaceus that is not responsive to conventional therapy (e.g., systemic corticosteroids and immunosuppressive agents) or to patients in whom these drugs may be harmful. Caution should be exercised in the use of rituximab because of the risk of serious short-term complications such as viral and bacterial infections and the potential for as yet unknown long-term complications. Finally, this study demonstrates the value of a multicenter approach to accomplish relevant clinical research in orphan diseases such as pemphigus. A U.S. national registry for pemphigus and standardized tools to assess the disease activity of pemphigus are necessary to accelerate research progress.