Analysis of Current Therapy and Clinical Outcome in Childhood Pemphigus Vulgaris

Pemphigus vulgaris (PV) is a rare, potentially fatal chronic autoimmune disease of the skin and mucous membrane. The objective of this study was to analyze the clinical outcomes and side effects associated with treatment of childhood PV (CPV). A retrospective review of the English language literature was conducted through PUBMED using the words childhood pemphigus vulgaris, and treatment or clinical outcome. Only patients under 12 years of age were included. Thirty-three cases were found in 29 reports. Mean age at onset was 8.3 years (range 1.5-12 yrs). Mucosal involvement (97.0%) was more common than cutaneous involvement (84.8%). Oral mucosa was the most common site of mucosal involvement (93.9%), followed by genital (20.6%), ocular (11.8%), and nasal mucosa (2.9%). Mean duration of therapy was 4.5 years (range 0.6-14.5 yrs), and mean duration of follow-up was 5.2 years (range 0.6-16 yrs). Complete recovery with no further therapy was achieved in 18.2% and partial recovery with minor relapses while on maintenance therapy in 78.8%. One patient died due to infection (3.0%). Serious side effects were present in 60.6%. The most common were cushingoid features (65.0%), growth retardation (50.0%), and infection (50.0%). Two patients who were refractory to systemic corticosteroids and immunosuppressive agents (ISA) had a favorable clinical response to Rituximab. Current therapy for CPV involving the use of long-term systemic corticosteroids in conjunction with ISA results in prolonged immunosuppression, causing systemic infections and growth retardation. Safer and more effective therapies need to be explored.