B-Cell Depletion for Autoimmune Thrombocytopenia and Autoimmune Hemolytic Anemia in Pediatric Systemic Lupus Erythematosus

OBJECTIVES. Our goal was to determine long-term efficacy and safety of B-cell-depletion therapy for children with autoimmune thrombocytopenia and autoimmune hemolytic anemia in pediatric systemic lupus erythematosus. PATIENTS AND METHODS. A retrospective, single-center cohort study was conducted including all patients with pediatric systemic lupus erythematosus who were diagnosed with autoimmune thrombocytopenia and/or autoimmune hemolytic anemia and treated with rituximab. Treatment efficacy and safety parameters were monitored and recorded. RESULTS. Nine patients with pediatric systemic lupus erythematosus were included in the study: 5 had autoimmune thrombocytopenia, 3 had autoimmune hemolytic anemia, and 1 had both. There were 5 female and 4 male patients; median age at diagnosis of pediatric systemic lupus erythematosus was 14 years (range: 8-16 years); and median pediatric systemic lupus erythematosus disease duration to time of rituximab treatment was 6 months (range: 2-30 months). Complete response was achieved in all 6 children with autoimmune thrombocytopenia (median time to complete response: 2 weeks [range: 1-12 weeks]). Two patients' conditions flared at 48 and 68 weeks, respectively, and were re-treated. The remaining 4 patients continued to be in remission at 24, 32, 36, and 88 weeks, respectively. All 4 children with autoimmune hemolytic anemia achieved complete response at a median time of 4 weeks (range: 4-32 weeks). All patients remained in complete response at 24, 44, 84, and 100 weeks of follow-up. Complete B-cell depletion was seen in all children after rituximab treatment. No serious infections occurred, but 1 patient had an infusion reaction. CONCLUSIONS. Preliminary evidence suggests that B-cell-depletion therapy with rituximab is an efficacious and safe treatment for autoimmune thrombocytopenia and autoimmune hemolytic anemia in pediatric systemic lupus erythematosus. Despite the prolonged effect on B-cell numbers and function, no serious infections were observed. Pediatrics 2009; 123: e159-e163