Eltrombopag: the Emerging Evidence of Its Therapeutic Value in Thrombocytopenia

Overview

Journal Core Evid

Publisher Dove Medical Press

Specialty Pharmacology

Date 2012 Apr 13

PMID 22496678

Citations 2

Authors

Louise Profit

Affiliations

Soon will be listed here.

Abstract

Introduction: Idiopathic (immune) thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by low platelet counts and bleeding episodes. Current therapy options are associated with unwanted side effects, and although patients initially respond to treatment the platelet count is not sustained in many individuals. There is a need for safe and well-tolerated treatments that provide a sustained platelet response.

Aims: This review summarizes the emerging evidence for the potential use of eltrombopag in the treatment of ITP in adults.

Disease And Treatment: Eltrombopag is a nonpeptide, small molecular weight thrombopoietin receptor agonist that is orally administered. It mimics the activity of thrombopoietin, a cytokine that promotes growth and production of platelets, primarily inducing proliferation and differentiation of megakaryocytes from bone marrow progenitor cells.

Profile: Eltrombopag is still in the early stages of development but initial phase I and phase II results are promising. Potential advantages of eltrombopag may include a sustained platelet response and a good tolerability profile. Its once-daily oral dosing regimen would also be an advantage over many of the existing therapies. It is expected that patients who have failed first-line therapy with corticosteroids and wish to avoid the need for a splenectomy, and patients with chronic refractory ITP, may benefit from eltrombopag treatment.

Citing Articles

[The efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia].

Huang Y, Liu X, Chen Y, Fu R, Liu W, Zhang L Zhonghua Xue Ye Xue Za Zhi. 2018; 39(1):32-36.

PMID: 29551030 PMC: 7343115. DOI: 10.3760/cma.j.issn.0253-2727.2018.01.007.

From chronic immune thrombocytopenia to severe aplastic anemia: recent insights into the evolution of eltrombopag.

Gill H, Wong R, Kwong Y Ther Adv Hematol. 2017; 8(5):159-174.

PMID: 28473904 PMC: 5407506. DOI: 10.1177/2040620717693573.

References

Kuhne T, Imbach P, Bolton-Maggs P, Berchtold W, Blanchette V, Buchanan G . Newly diagnosed idiopathic thrombocytopenic purpura in childhood: an observational study. Lancet. 2002; 358(9299):2122-5. DOI: 10.1016/S0140-6736(01)07219-1. View

Chong B, Ho S . Autoimmune thrombocytopenia. J Thromb Haemost. 2005; 3(8):1763-72. DOI: 10.1111/j.1538-7836.2005.01376.x. View

Broudy V, Lin N . AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine. 2003; 25(2):52-60. DOI: 10.1016/j.cyto.2003.05.001. View

Aledort L, Hayward C, Chen M, Nichol J, Bussel J . Prospective screening of 205 patients with ITP, including diagnosis, serological markers, and the relationship between platelet counts, endogenous thrombopoietin, and circulating antithrombopoietin antibodies. Am J Hematol. 2004; 76(3):205-13. DOI: 10.1002/ajh.20104. View

. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol. 2003; 120(4):574-96. DOI: 10.1046/j.1365-2141.2003.04131.x. View

Neylon A, Saunders P, Howard M, Proctor S, Taylor P . Clinically significant newly presenting autoimmune thrombocytopenic purpura in adults: a prospective study of a population-based cohort of 245 patients. Br J Haematol. 2003; 122(6):966-74. DOI: 10.1046/j.1365-2141.2003.04547.x. View

Fujisawa K, Tani P, Piro L, McMillan R . The effect of therapy on platelet-associated autoantibody in chronic immune thrombocytopenic purpura. Blood. 1993; 81(11):2872-7. View

Stoll D, Cines D, Aster R, Murphy S . Platelet kinetics in patients with idiopathic thrombocytopenic purpura and moderate thrombocytopenia. Blood. 1985; 65(3):584-8. View

Stasi R, Stipa E, Masi M, Cecconi M, Scimo M, Oliva F . Long-term observation of 208 adults with chronic idiopathic thrombocytopenic purpura. Am J Med. 1995; 98(5):436-42. DOI: 10.1016/s0002-9343(99)80342-8. View

10.

Waters A . Autoimmune thrombocytopenia: clinical aspects. Semin Hematol. 1992; 29(1):18-25. View

11.

Cines D, Bussel J . How I treat idiopathic thrombocytopenic purpura (ITP). Blood. 2005; 106(7):2244-51. DOI: 10.1182/blood-2004-12-4598. View

12.

Dickerhoff R, von Ruecker A . The clinical course of immune thrombocytopenic purpura in children who did not receive intravenous immunoglobulins or sustained prednisone treatment. J Pediatr. 2000; 137(5):629-32. DOI: 10.1067/mpd.2000.110123. View

13.

Andres E, Zimmer J, Noel E, Kaltenbach G, Koumarianou A, Maloisel F . Idiopathic thrombocytopenic purpura: a retrospective analysis in 139 patients of the influence of age on the response to corticosteroids, splenectomy and danazol. Drugs Aging. 2003; 20(11):841-6. DOI: 10.2165/00002512-200320110-00005. View

14.

Cohen Y, Djulbegovic B, Mozes B . The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med. 2000; 160(11):1630-8. DOI: 10.1001/archinte.160.11.1630. View

15.

Fabris F, Tassan T, Ramon R, Carraro G, Randi M, Luzzatto G . Age as the major predictive factor of long-term response to splenectomy in immune thrombocytopenic purpura. Br J Haematol. 2001; 112(3):637-40. DOI: 10.1046/j.1365-2141.2001.02615.x. View

16.

Bell Jr W . Long-term outcome of splenectomy for idiopathic thrombocytopenic purpura. Semin Hematol. 2000; 37(1 Suppl 1):22-5. DOI: 10.1016/s0037-1963(00)90114-8. View

17.

Bennett C, Weinberg P, Golub R, Bussel J . The potential for treatment of idiopathic thrombocytopenic purpura with anti-D to prevent splenectomy: a predictive cost analysis. Semin Hematol. 2000; 37(1 Suppl 1):26-30. DOI: 10.1016/s0037-1963(00)90115-x. View

18.

George J, Woolf S, Raskob G, Wasser J, Aledort L, Ballem P . Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood. 1996; 88(1):3-40. View

19.

George J, Davidoff F . Idiopathic thrombocytopenic purpura: lessons from a guideline. Ann Intern Med. 1997; 126(4):317-8. DOI: 10.7326/0003-4819-126-4-199702150-00009. View

20.

Geddis A, Linden H, Kaushansky K . Thrombopoietin: a pan-hematopoietic cytokine. Cytokine Growth Factor Rev. 2001; 13(1):61-73. DOI: 10.1016/s1359-6101(01)00030-2. View