Thrombopoietin receptor agonists: a critical review

Abstract

Thrombopoietic agents have created a paradigm shift in the management of chronic or poorly responsive immune thrombocytopenia (ITP). There are ample randomized, placebo-controlled trial data, as well as long-term data gathered for more than 5 years; short-term efficacy and safety are well documented and long-term efficacy and safety data are emerging. The purpose of this review will be to focus critically on what we know or do not know at this point about these agents. We review the evolution, efficacy and clinical use, side effects, special populations, and off-target effects of the thrombopoietic agents.