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fter many decades of an orphan existence, immune thrombocytopenic purpura (ITP) is a central focus for the clinical development of mul- tiple, novel thrombopoietic agents. These agents have the potential to transform the management of ITP and possibly other thrombocytopenic disorders. Now, with the approval of the first agents for clinical use, their role in patient care will become established and their poten- tial risks may be more clearly revealed. The availability of novel thrombopoietic agents for the treatment of patients with thrombocytopenia is remark- able both as a scientific achievement and also as an immediate benefit for patient care. The history of the isolation and characterization of thrombopoietin (TPO) in 1994 and the development of therapeutic throm- bopoietic agents have been recently reviewed. 1 This year the first two agents, romiplostim (previously known as AMG 531) and eltrombopag, will be approved by the US Food and Drug Administration (FDA) for treatment of patients with ITP. The success of these thrombopoietic agents in clinical trials for treatment of ITP 2-4 has stimu- lated the development of multiple other compounds to enhance platelet production, including the novel small non-peptidyl molecule described in this issue of the jour- nal by Nogami et al. 5 The molecular diversity of these agents, their therapeutic effectiveness, and their poten- tial risks are an important story for hematologists. In this Perspective, we will describe (1) the evolution of understanding of the pathogenesis of ITP, that provided the insight for treatment with thrombopoietic agents; (2) the experience of individual patients with ITP who have received thrombopoietic agents; (3) the molecular spec- trum of thrombopoietic agents that are currently in development; (4) the clinical spectrum of targeted thrombocytopenic disorders; and (5) the potential harm from these agents. Immune thrombocytopenic purpura: a target for drug development The clinical development of thrombopoietic agents has been intimately associated with ITP. The low preva- lence of ITP, estimated at 9 patients per 10 5 population, 6

Novel thrombopoietic agents: a new era for management of patients with thrombocytopenia