Recent Advances in the Treatment of Childhood

Immune thrombocytopenic purpura (ITP), the most common bleeding disorder in childhood, is a benign condition that typically resolves within 6 months following diagnosis. While all would agree on drug treatment for children with severe hemorrhage, management strategies of the minimally symptomatic child with a very low platelet count can vary widely. The case for treatment is based primarily on the risk for the uncommon complication of intracranial hemorrhage or other serious bleeding. Numerous clinical studies clearly indicate that immune globulins (intravenous immune globulin [IVIg] or anti-D) and corticosteroid therapy (prednisone and megadose methylprednisolone) are effective in rapidly raising platelet counts. However, physicians must ultimately decide whether to use drug therapy for a patient with minimal symptoms and low platelet count or adopt an “observation-only” approach. With this background, three fundamental questions emerge when evaluating different clinical strategies and potential outcomes: (1) Which children with ITP need treatment? (2) Which pharmacologic agent best accomplishes the goals of drug treatment? (3) Can physicians better predict the outcome of treatments?