Role of Cyclosporine A in Pediatric Patients with Refractory Chronic Idiopathic Thrombocytopenic Purpura

Purpose: Since the treatment of chronic idiopathic thrombocytopenic purpura (ITP) remains unsatisfactory in patients refractory to standard therapy, alternative therapies such as splenectomy, danazol, rituximab and cyclosporine A (CsA) are being considered as recently. The efficacy and safety of CsA has already been proved to adult patients with chronic ITP, however, there was no previous study in pediatric patients. The purpose of this study was to evaluate the efficacy and side effect of CsA in pediatric chronic ITP patients. Methods: We reviewed medical records of nine steroid refractory chronic ITP patients diagnosed as chronic ITP who were treated with CsA during 2007 and 2009 retrospectively. Results: All nine patients received standard treatment of intravenous immunoglobulin G and steroid before receiving CsA. Average time duration to start CsA from diagnosis was 52 months. Three children (33%) showed more than partial response maintaining platelet count over 50×109/L, the other six patients did not show any effect. There were no remarkable toxicities other than mild chest discomfort and headache in two patients. Conclusion: CsA therapy is considered as a safe and effective treatment option in adult chronic ITP patients recently, however, there is nearly no study in pediatric patients only with few case reports. In this study only small portion of patients showed response to CsA. Since it was a small sized study with short term follow up, long term follow up with larger patient number is required to make conclusionope