Clinical remission following monoclonal anti-CD20 therapy in two children with chronic refractory idiopathic thrombocytopenic purpura

Abstract:  Two patients, a 4-year-old boy and a 6-year-old girl who had a 2-year and a 3-year history of idiopathic thrombocytopenic purpura, respectively, were referred to our Department. Both patients had frequent haemorrhagic events. They received i.v. immunoglobulin, corticosteroids, cyclosporine, interferon α-2b and azathioprine, but no clinical remission was established. The girl also underwent splenectomy. Anti-CD20 antibody was administered to both patients at a dose of 375 mg/m² once weekly for 4 weeks. No side-effects were detected. During the 18-month follow-up period the patients received no other drug and remained in clinical remission. The B lymphocytes remained undetectable in peripheral blood for 3 months and they progressively increased during the following 4 months. Rituximab is a novel, quite effective, safe treatment of chronic refractory idiopathic thrombocytopenic purpura in childhood. More studies and follow up of patients for longer periods are necessary.