Complete recovery from refractory immune thrombocytopenic purpura in three patients treated with etanercept

Management of patients with immune thrombocytopenic purpura (ITP) who have persistent, severe, and symptomatic thrombocytopenia following splenectomy is difficult and empirical. No single agent or regimen provides long-term success for most patients, and for most treatments it is difficult to assess whether benefits outweigh risks. We report three consecutive patients with critical chronic refractory ITP, who responded promptly and completely following treatment with etanercept, an inhibitor of tumor necrosis factor-alpha. These patients had failed 6-11 previous treatments. In the first patient, etanercept was given for its approved indication: a flare of co-existing rheumatoid arthritis. The next two patients were treated with etanercept because of successful outcomes in the previous patients. Although etanercept appeared to be effective treatment for ITP in these 3 patients, the experimental nature of this treatment and the potential risks must be emphasized. On the basis of these case reports, a clinical trial has been initiated to systematically evaluate the efficacy and risks of etanercept in the management of children and adults with chronic ITP. (C) 2003 Wiley-Liss, Inc.