Immune thrombocytopenic purpura - current management practices

The treatment of patients with immune thrombocytopenic purpura (ITP) is changing rapidly, as new agents demonstrate the capability of improving outcomes and decreasing toxicity. Prior to 1981, the only effective treatment options available to increase platelet counts in persons with ITP were corticosteroids and splenectomy. In recent years, intravenous immunoglobulin (IVIg) and intravenous Rh immunoglobulin (IV RhIg) have demonstrated efficacy comparable to that of corticosteroids for increasing platelet counts in ITP. In addition, IVIg and IV RhIg have demonstrated efficacy for maintaining corticosteroid-induced increased platelet counts by periodic infusion, causing a transient impairment of reticuloendothelial clearance function (medical splenectomy). Thus, the time-proven efficacy of corticosteroids for initial treatment of ITP (induction) may now be supplemented with IVIg or IV RhIg infusions for patients requiring ongoing treatment to support a timely and complete steroid taper, while sustaining the increased platelet count (maintenance) with less toxicity. Several investigators have reported that rituximab (anti-CD20) induced sustained remissions with minimal toxicity, in patients with chronic ITP. These reports are promising and, if confirmed, will provide another effective (spleen-sparing) option for managing acute ITP and a long-awaited option for patients who have had a splenectomy and are refractory to conventional agents. Other treatments, including danazol, azathioprine, cyclophosphamide, vinca alkaloids and cyclosporin A, have advocates, but evidence of their efficacy is limited to relatively small and mostly uncontrolled clinical trials. In our opinion, these agents should be reserved for symptomatic thrombocytopenia after refractoriness to corticosteroids, IVIg, IV RhIg, splenectomy and rituximab has been clearly established.