PHASE I/II TRIAL OF RITUXIMAB FOR CHRONIC, SEVERE ITP IN CHILDREN AND ADOLES

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Immune thrombocytopenia (ITP) in childhood is most commonly a sef-limited disorder, resolving within a year in about 70% of affected children. In a minority of patients, the condition becomes chronic. Whereas many patients with chronic ITP have benign courses, requiring little or no ongoing therapy, a fraction of these individuals has persistent severe disease. Refractoriness to first line agents (steriods, anti-D globulin, IVIG or splenectomy) in severly affected patients is a vexing clinical problem. These patients are at relatively high risk for blieeding, and no available literature suggests that any of the many available interventions is safer or more effective than any other ( including no intervention at all). In the long run, the best way to answer this type of question is by randomized clinical trial. We propose this pilot study as prologue to such a randomized trial. Rituximab is an attractive choice in severe ITP. The pathophysiology of the disorder is thought to be anitbody mediated platelet destruction in the majority of cases, and the toxicity profile is reasonable for initial trials in severly affected patients. Durable responses have been reported.