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Clinical applications of thrombopoietic growth factors

David J Kuter, MD, DPhil
Section Editor
Lawrence LK Leung, MD
Deputy Editor
Jennifer S Tirnauer, MD


The use of hematopoietic growth factors has markedly changed the practice of medicine. Erythroid growth factors (eg, erythropoietin, darbepoetin) and myeloid growth factors (eg, granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor) have allowed the specific stimulation of the production of erythrocytes and neutrophils, respectively. (See "Introduction to recombinant hematopoietic growth factors".)

With the discovery of thrombopoietin (TPO) and the development of a variety of peptide and non-peptide TPO receptor agonists, clinically effective thrombopoietic growth factors have now entered the clinical armamentarium. This topic review will discuss the thrombopoietins and TPO receptor agonists that have been developed, their activity in preclinical models, and the available clinical studies. The biology of TPO is discussed separately. (See "Biology and physiology of thrombopoietin".)


The potential clinical applications of thrombopoietic growth factors are suggested by analyzing the use of platelet transfusions (figure 1). (See "Clinical and laboratory aspects of platelet transfusion therapy", section on 'Indications for platelet transfusion'.)

Two TPO receptor agonists (romiplostim, eltrombopag) have been approved for the treatment of chronic immune thrombocytopenia (chronic ITP). (See "Immune thrombocytopenia (ITP) in adults: Second-line and subsequent therapies", section on 'Thrombopoietin receptor agonists'.)

One thrombopoietic growth factor (eltrombopag) has been approved for the treatment of thrombocytopenia in patients with hepatitis C who are being treated with interferon (see "Management of the side effects of peginterferon and ribavirin used for treatment of chronic hepatitis C virus infection", section on 'Thrombocytopenia') and for the treatment of aplastic anemia. (See "Treatment of aplastic anemia in adults", section on 'Eltrombopag'.)


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Literature review current through: Sep 2016. | This topic last updated: Sep 6, 2016.
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