Rituximab in childhood pemphigus vulgaris: a long-term follow-up case and review of the literature

Abstract

Pemphigus vulgaris is an infrequent but life-threatening autoimmune blistering disease that is rare in the pediatric age. Although the mainstay of therapy for childhood pemphigus vulgaris (CPV) is steroids, adjuvant immunosuppressive drugs are often needed to control the disease. Thus, an important part of CPV morbidity can be related to treatment. We report the youngest CPV patient described in the English literature, an 18-month-old boy, who has been followed up for 16 years. During this period, the patient has received several immunosuppressive therapies with variable response. He has finally achieved a long-lasting and complete remission with rituximab. Successful treatment with rituximab has previously been reported in 6 cases of CPV, in whom rituximab presented a good side effect profile. Our patient has experienced a chronic and severe clinical course with multiple flares eventually developing vegetative lesions. Since he presented refractoriness to multiple therapies, we administered rituximab. The introduction of this drug led to a dramatic clinical response and a long-term clinical remission. Based on the experience of this case and the data reported in the literature, we believe that rituximab may be a safe and efficacious agent for the treatment of severe CPV.