Thrombopoietin Receptor Agonists: Characteristics, Adverse Effects, and Indications

  • Jenny Despotovic
  • Amanda Grimes


While the most commonly utilized first-line therapeutics for the treatment of immune thrombocytopenia (ITP) target the peripheral destruction of platelets, a preponderant second-line therapeutic agent targeting increased megakaryopoiesis and/or platelet production has now emerged, in the form of thrombopoietin receptor agonists (TPO-RAs). These TPO-RAs, eltrombopag and romiplostim, have been proven safe and effective in treating ITP in both children and adults and have also been approved for use in severe aplastic anemia and chronic hepatitis, with ongoing investigation in the treatment of other clinical entities as well, including myelodysplastic syndrome and acute myelogenous leukemia, chemotherapy-induced thrombocytopenia, congenital thrombocytopenia, and other diseases. This chapter will detail TPO-RA use in all of these different clinical settings.


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© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Pediatric Hematology/Oncology, Baylor College of Medicine/Texas Children’s HospitalHoustonUSA

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