Severe chronic refractory immune thrombocytopenic purpura during childhood: A survey of physician management

Background. Physician attitudes regarding management of children with severe chronic immune thrombocytopenic purpura (ITP) have not been recently characterized. Procedure. We designed a survey of members of the American Society of Pediatric Hematology-Oncology (ASPHO) that described a 5-year-old female with ITP for 1 year who was unresponsive to steroids, IVIG, and anti-D immune globulin and having frequent epistaxis causing interference with her daily activities. A 13-item questionnaire evaluated physician decision-making in this setting. Results. Two hundred and ninety-seven surveys (35% response rate) were returned, and 295 were evaluable. Thirty-three percent of respondents stated that they would recommend splenectomy for such a child. Of those who would not recommend splenectomy, 67% reported that they would instead treat with rituximab. If initial drug therapy failed, 47% would proceed with splenectomy. Those who reported treating with rituximab initially were more likely to recommend splenectomy following failure than those who preferred other drug therapy (P < 0.0001). Conclusions. Physician management of patients with chronic ITP is diverse. With the advent of new treatments such as rituximab and thrombopoetic agents it is critically important to compare their cost, adverse effects and efficacy with splenectomy in order to optimally guide treatment practices.