Mechanisms and therapeutic prospects of thrombopoietin receptor agonists

James Bussel, Austin Kulasekararaj, Nichola Cooper, Amit Verma, Ulrich Steidl, John W. Semple, Britta Will

Research output: Contribution to journalReview articlepeer-review

22 Scopus citations


The second-generation thrombopoietin (TPO) receptor agonists eltrombopag and romiplostim are potent activators of megakaryopoiesis and represent a growing treatment option for patients with thrombocytopenic hematological disorders. Both TPO receptor agonists have been approved worldwide for the treatment of children and adults with chronic immune thrombocytopenia. In the EU and USA, eltrombopag is approved for the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy and in the USA for the first-line treatment of severe aplastic anemia in combination with immunosuppressive therapy. Eltrombopag has also shown efficacy in several other disease settings, for example, chemotherapy-induced thrombocytopenia, selected inherited thrombocytopenias, and myelodysplastic syndromes. While both TPO receptor agonists stimulate TPO receptor signaling and enhance megakaryopoiesis, their vastly different biochemical structures bestow upon them markedly different molecular and functional properties. Here, we review and discuss results from preclinical and clinical studies on the functional and molecular mechanisms of action of this new class of drug.

Original languageEnglish (US)
Pages (from-to)262-278
Number of pages17
JournalSeminars in hematology
Issue number4
StatePublished - Oct 2019


  • Eltrombopag
  • Mechanism of action
  • Megakaryopoiesis
  • Romiplostim
  • Thrombocytopenia

ASJC Scopus subject areas

  • Hematology


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