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Eltrombopag

Eltrombopag is a small-molecule, nonpeptide thrombopoietin receptor agonist with megakaryopoiesis-stimulating activity. Eltrombopag binds to and stimulates the transmembrane domain of the platelet thrombopoietin receptor (TPO-R or CD110), a member of the hematopoietin receptor superfamily. Activation of TPO-R leads to the proliferation and differentiation of cells in the megakaryocytic lineage and an increase in platelet production. Eltrombopag was approved by FDA in November 20, 2008. Check for active clinical trials or closed clinical trials using this agent. (NCI Thesaurus).

Eltrombopag (rINN, codenamed SB-497115-GR) is a drug candidate that has been developed for conditions that lead to thrombocytopenia (abnormally low platelet counts). It is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin. Eltrombopag was discovered as a result of research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals. Designated an orphan drug in the USA and European Union, it is being manufactured and marketed by GlaxoSmithKline under the trade name Promacta in the USA and will be marketed as Revolade in the EU.  Eltrombopag was approved by the U.S. Food and Drug Administration on November 20, 2008.

In preclinical studies, the compound was shown to interact selectively with the thrombopoeitin receptor, leading to activation of the JAK-STAT signaling pathway and increased proliferation and differentiation of megakaryocytes. Animal studies confirmed that administration could increase platelet counts. In 73 healthy volunteers, higher doses of eltrombopag caused larger increases in the number of circulating platelets without tolerability problems.

Eltrombopag has been studied, and shown to be effective, in two major clinical syndromes: idiopathic thrombocytopenic purpura and cirrhosis due to hepatitis C (in which low platelet counts may be a contraindication for interferon treatment). After 6 weeks of therapy in a phase III trial, eltrombopag 50mg/day was associted with a significantly higher resonse rate than placebo in adult patients with chronic ITP.

1: Scully M, Kaczmarski R, Myers B, Collins R, Emohare O, Bolton-Maggs PH. Eltrombopag named patient programme for patients with chronic immune thrombocytopenia. Br J Haematol. 2011 Nov 17. doi: 10.1111/j.1365-2141.2011.08954.x. [Epub ahead of print] PubMed PMID: 22091659.

2: Zhang Y, Kolesar JM. Eltrombopag: an oral thrombopoietin receptor agonist for the treatment of idiopathic thrombocytopenic purpura. Clin Ther. 2011 Nov;33(11):1560-76. Epub 2011 Nov 4. PubMed PMID: 22054810.

3: Garnock-Jones KP. Spotlight on eltrombopag in treatment-refractory chronic primary immune thrombocytopenia†. BioDrugs. 2011 Dec 1;25(6):401-4. doi: 10.2165/11207620-000000000-00000. PubMed PMID: 22050343.

4: Duic JP, Grewal J, McConie K, Staszewski H, Haas J, Kesari S. Eltrombopag for radiation-induced thrombocytopenia in a glioblastoma patient. J Neurooncol. 2012 Jan;106(2):427-9. Epub 2011 Aug 11. PubMed PMID: 21833801.

5: Garnock-Jones KP. Eltrombopag: a review of its use in treatment-refractory chronic primary immune thrombocytopenia. Drugs. 2011 Jul 9;71(10):1333-53. doi: 10.2165/11207390-000000000-00000. Review. PubMed PMID: 21770480.

6: Nieto M, Calvo G, Hudson I, Feldschreiber P, Brown D, Lee CC, Lay G, Valeri A, Abadie E, Thomas A, Pignatti F. The European Medicines Agency review of eltrombopag (Revolade) for the treatment of adult chronic immune (idiopathic) thrombocytopenic purpura: summary of the scientific assessment of the Committee for Medicinal Products for Human Use. Haematologica. 2011 Sep;96(9):e33-40. Epub 2011 Jun 28. Review. PubMed PMID: 21712542; PubMed Central PMCID: PMC3166089.

7: Cheng G. Eltrombopag for the treatment of immune thrombocytopenia. Expert Rev Hematol. 2011 Jun;4(3):261-9. PubMed PMID: 21671710.

8: Deng Y, Rogers M, Sychterz C, Talley K, Qian Y, Bershas D, Ho M, Shi W, Chen EP, Serabjit-Singh C, Gorycki PD. Investigations of hydrazine cleavage of eltrombopag in humans. Drug Metab Dispos. 2011 Sep;39(9):1747-54. Epub 2011 Jun 6. PubMed PMID: 21646438.

9: Deng Y, Madatian A, Wire MB, Bowen C, Park JW, Williams D, Peng B, Schubert E, Gorycki F, Levy M, Gorycki PD. Metabolism and disposition of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist, in healthy human subjects. Drug Metab Dispos. 2011 Sep;39(9):1734-46. Epub 2011 Jun 6. PubMed PMID: 21646437.

10: Tanimoto T, Fukunaga S, Hori A, Yagasaki F, Ono S. Eltrombopag for chronic immune thrombocytopenia. Lancet. 2011 Jun 4;377(9781):1919; author reply 1919-20. PubMed PMID: 21641478.

11: Haselboeck J, Pabinger I, Ay C, Koder S, Panzer S. Platelet activation and function during eltrombopag treatment in immune thrombocytopenia. Ann Hematol. 2012 Jan;91(1):109-13. Epub 2011 May 7. PubMed PMID: 21553010.

12: Allred AJ, Bowen CJ, Park JW, Peng B, Williams DD, Wire MB, Lee E. Eltrombopag increases plasma rosuvastatin exposure in healthy volunteers. Br J Clin Pharmacol. 2011 Aug;72(2):321-9. doi: 10.1111/j.1365-2125.2011.03972.x. PubMed PMID: 21434975; PubMed Central PMCID: PMC3162661.

13: Takeuchi K, Sugiura T, Umeda S, Matsubara K, Horikawa M, Nakamichi N, Silver DL, Ishiwata N, Kato Y. Pharmacokinetics and hepatic uptake of eltrombopag, a novel platelet-increasing agent. Drug Metab Dispos. 2011 Jun;39(6):1088-96. Epub 2011 Mar 21. PubMed PMID: 21422191.

14: Cela I, Miller IJ, Katz RS, Rizman A, Shammo JM. Successful treatment of amegakaryocytic thrombocytopenia with eltrombopag in a patient with systemic lupus erythematosus (SLE). Clin Adv Hematol Oncol. 2010 Nov;8(11):806-9. PubMed PMID: 21326158.

15: Benham L, Guo L, Turner R. What is publication? The case of eltrombopag. Lancet. 2011 Jan 29;377(9763):360-1. PubMed PMID: 21277427.

16: Hayes S, Ouellet D, Zhang J, Wire MB, Gibiansky E. Population PK/PD modeling of eltrombopag in healthy volunteers and patients with immune thrombocytopenic purpura and optimization of response-guided dosing. J Clin Pharmacol. 2011 Oct;51(10):1403-17. Epub 2010 Dec 8. PubMed PMID: 21148042.

17: Bussel JB, Pinheiro MP. Eltrombopag. Cancer Treat Res. 2011;157:289-303. Review. PubMed PMID: 21052963.

18: Pecci A, Gresele P, Klersy C, Savoia A, Noris P, Fierro T, Bozzi V, Mezzasoma AM, Melazzini F, Balduini CL. Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations. Blood. 2010 Dec 23;116(26):5832-7. Epub 2010 Sep 15. PubMed PMID: 20844233.

19: Bowen CJ, Lobb KM, Park JW, Sanderson B, Ferguson J. Eltrombopag (75 mg) does not induce photosensitivity: results of a clinical pharmacology trial. Photodermatol Photoimmunol Photomed. 2010 Oct;26(5):243-9. doi: 10.1111/j.1600-0781.2010.00538.x. PubMed PMID: 20831698.

20: Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, Arning M, Stone NL, Bussel JB. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011 Jan 29;377(9763):393-402. Epub 2010 Aug 23. Erratum in: Lancet. 2011 Jan 29;377(9763):382. PubMed PMID: 20739054.

21: Kellum A, Jagiello-Gruszfeld A, Bondarenko IN, Patwardhan R, Messam C, Mostafa Kamel Y. A randomized, double-blind, placebo-controlled, dose ranging study to assess the efficacy and safety of eltrombopag in patients receiving carboplatin/paclitaxel for advanced solid tumors. Curr Med Res Opin. 2010 Oct;26(10):2339-46. PubMed PMID: 20735290.

22: Cook L, Cooper N. Eltrombopag--a novel approach for the treatment of chronic immune thrombocytopenic purpura: review and safety considerations. Drug Des Devel Ther. 2010 Jul 21;4:139-45. Review. PubMed PMID: 20689640; PubMed Central PMCID: PMC2915538.

23: Mavroudi I, Pyrovolaki K, Pavlaki K, Kozana A, Psyllaki M, Kalpadakis C, Pontikoglou C, Papadaki HA. Effect of the nonpeptide thrombopoietin receptor agonist eltrombopag on megakaryopoiesis of patients with lower risk myelodysplastic syndrome. Leuk Res. 2011 Mar;35(3):323-8. Epub 2010 Aug 4. PubMed PMID: 20688394.

24: Gibiansky E, Zhang J, Williams D, Wang Z, Ouellet D. Population pharmacokinetics of eltrombopag in healthy subjects and patients with chronic idiopathic thrombocytopenic purpura. J Clin Pharmacol. 2011 Jun;51(6):842-56. Epub 2010 Jul 27. PubMed PMID: 20663993.

25: Bauman JW, Vincent CT, Peng B, Wire MB, Williams DD, Park JW. Effect of hepatic or renal impairment on eltrombopag pharmacokinetics. J Clin Pharmacol. 2011 May;51(5):739-50. Epub 2010 Jul 27. PubMed PMID: 20663991.

26: Sperati CJ, Streiff MB. Acute renal failure in a patient with antiphospholipid syndrome and immune thrombocytopenic purpura treated with eltrombopag. Am J Hematol. 2010 Sep;85(9):724-6. PubMed PMID: 20652968.

27: Matthys G, Park JW, McGuire S, Wire MB, Zhang J, Bowen C, Williams D, Jenkins JM, Peng B. Eltrombopag does not affect cardiac repolarization: results from a definitive QTc study in healthy subjects. Br J Clin Pharmacol. 2010 Jul;70(1):24-33. PubMed PMID: 20642544; PubMed Central PMCID: PMC2909804.

28: Chouhan JD, Herrington JD. Treatment options for chronic refractory idiopathic thrombocytopenic purpura in adults: focus on romiplostim and eltrombopag. Pharmacotherapy. 2010 Jul;30(7):666-83. Review. PubMed PMID: 20575632.

29: Corman SL, Mohammad RA. Eltrombopag: a novel oral thrombopoietin receptor agonist. Ann Pharmacother. 2010 Jun;44(6):1072-9. Epub 2010 May 11. Review. Erratum in: Ann Pharmacother. 2010 Jul-Aug;44(7-8):1352-3. PubMed PMID: 20460556.

30: Eltrombopag. Idiopathic thrombocytopenic purpura after treatment failure: romiplostim is a better option. Prescrire Int. 2010 Feb;19(105):15. PubMed PMID: 20455333.

31: Matthys G, Park JW, McGuire S, Wire MB, Bowen C, Williams D, Jenkins J, Peng B. Clinical pharmacokinetics, platelet response, and safety of eltrombopag at supratherapeutic doses of up to 200 mg once daily in healthy volunteers. J Clin Pharmacol. 2011 Mar;51(3):301-8. Epub 2010 Apr 23. PubMed PMID: 20418510.

32: Koehrer S, Keating MJ, Wierda WG. Eltrombopag, a second-generation thrombopoietin receptor agonist, for chronic lymphocytic leukemia-associated ITP. Leukemia. 2010 May;24(5):1096-8. Epub 2010 Mar 25. PubMed PMID: 20339441.

33: Erickson-Miller CL, Kirchner J, Aivado M, May R, Payne P, Chadderton A. Reduced proliferation of non-megakaryocytic acute myelogenous leukemia and other leukemia and lymphoma cell lines in response to eltrombopag. Leuk Res. 2010 Sep;34(9):1224-31. Epub 2010 Mar 3. PubMed PMID: 20202683.

34: Danish FA, Koul SS, Subhani FR, Rabbani AE, Yasmin S. Considerations in the management of hepatitis C virus-related thrombocytopenia with eltrombopag. Saudi J Gastroenterol. 2010 Jan-Mar;16(1):51-6. Review. PubMed PMID: 20065578; PubMed Central PMCID: PMC3023106.

35: Bussel JB. Update on eltrombopag for ITP. Oncology (Williston Park). 2009 Nov 30;23(13):1177-8. PubMed PMID: 20043469.

36: Dmytrijuk A, Robie-Suh K, Rieves D, Pazdur R. Eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura. Oncology (Williston Park). 2009 Nov 30;23(13):1171-7. PubMed PMID: 20043468.

37: Jenkins J, Williams D, Deng Y, Collins DA, Kitchen VS. Eltrombopag, an oral thrombopoietin receptor agonist, has no impact on the pharmacokinetic profile of probe drugs for cytochrome P450 isoenzymes CYP3A4, CYP1A2, CYP2C9 and CYP2C19 in healthy men: a cocktail analysis. Eur J Clin Pharmacol. 2010 Jan;66(1):67-76. Epub 2009 Sep 16. PubMed PMID: 19756558.

38: Will B, Kawahara M, Luciano JP, Bruns I, Parekh S, Erickson-Miller CL, Aivado MA, Verma A, Steidl U. Effect of the nonpeptide thrombopoietin receptor agonist Eltrombopag on bone marrow cells from patients with acute myeloid leukemia and myelodysplastic syndrome. Blood. 2009 Oct 29;114(18):3899-908. Epub 2009 Aug 26. PubMed PMID: 19710504; PubMed Central PMCID: PMC2773490.

39: Rice L. Treatment of immune thrombocytopenic purpura: focus on eltrombopag. Biologics. 2009;3:151-7. Epub 2009 Jul 13. PubMed PMID: 19707404; PubMed Central PMCID: PMC2726065.

40: Erhardt JA, Erickson-Miller CL, Aivado M, Abboud M, Pillarisetti K, Toomey JR. Comparative analyses of the small molecule thrombopoietin receptor agonist eltrombopag and thrombopoietin on in vitro platelet function. Exp Hematol. 2009 Sep;37(9):1030-7. Epub 2009 Jul 24. PubMed PMID: 19631713.

41: Serebruany VL, Eisert C, Sabaeva E, Makarov L. Eltrombopag (Promacta), a thrombopoietin receptor agonist for the treatment of thrombocytopenia: current and future considerations. Am J Ther. 2010 Jan-Feb;17(1):68-74. PubMed PMID: 19451811.

42: Williams DD, Peng B, Bailey CK, Wire MB, Deng Y, Park JW, Collins DA, Kapsi SG, Jenkins JM. Effects of food and antacids on the pharmacokinetics of eltrombopag in healthy adult subjects: two single-dose, open-label, randomized-sequence, crossover studies. Clin Ther. 2009 Apr;31(4):764-76. PubMed PMID: 19446149.

43: Garnock-Jones KP, Keam SJ. Eltrombopag. Drugs. 2009;69(5):567-76. doi: 10.2165/00003495-200969050-00005. PubMed PMID: 19368418.

44: Panzer S. Eltrombopag in chronic idiopathic thrombocytopenic purpura and HCV-related thrombocytopenia. Drugs Today (Barc). 2009 Feb;45(2):93-9. Review. PubMed PMID: 19343229.

45: Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. PubMed PMID: 19231632.

46: Panzer S, Pabinger I. Eltrombopag in chronic idiopathic thrombocytopenic purpura. Lancet. 2009 Feb 21;373(9664):607-8. Erratum in: Lancet. 2009 May 9-2009 May 15;373(9675):1606. PubMed PMID: 19231614.

47: Stasi R. Eltrombopag for the treatment of idiopathic thrombocytopenic purpura. Expert Rev Hematol. 2008 Dec;1(2):145-52. Review. PubMed PMID: 21082919.

48: Erickson-Miller CL, Delorme E, Tian SS, Hopson CB, Landis AJ, Valoret EI, Sellers TS, Rosen J, Miller SG, Luengo JI, Duffy KJ, Jenkins JM. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009 Feb;27(2):424-30. PubMed PMID: 19038790; PubMed Central PMCID: PMC2729672.

49: Molecule of the month. Eltrombopag. Drug News Perspect. 2008 Jul-Aug;21(6):344. PubMed PMID: 18836592.

50: Bussel JB, Marks KM. How effective is eltrombopag for the treatment of thrombocytopenia in patients with HCV infection? Nat Clin Pract Gastroenterol Hepatol. 2008 Aug;5(8):424-5. Epub 2008 Jul 8. PubMed PMID: 18607406.

51: Mondelli MU. Eltrombopag: an effective remedy for thrombocytopaenia? J Hepatol. 2008 Jun;48(6):1030-2. Epub 2008 Mar 31. PubMed PMID: 18433923.

52: Zekry A, Freiman J. Eltrombopag: Is this "24 karat gold platelet" treatment for thrombocytopenia in cirrhosis associated with hepatitis C? Hepatology. 2008 Apr;47(4):1418-21. PubMed PMID: 18366111.

53: Zimmer J, Hentges F, Andrès E. Eltrombopag in thrombocytopenia. N Engl J Med. 2008 Mar 6;358(10):1072; author reply 1072-3. PubMed PMID: 18326076.

54: Lawson A. Eltrombopag in thrombocytopenia. N Engl J Med. 2008 Mar 6;358(10):1072; author reply 1072-3. PubMed PMID: 18322290.

55: Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. PubMed PMID: 18046028.

56: McHutchison JG, Dusheiko G, Shiffman ML, Rodriguez-Torres M, Sigal S, Bourliere M, Berg T, Gordon SC, Campbell FM, Theodore D, Blackman N, Jenkins J, Afdhal NH; TPL102357 Study Group. Eltrombopag for thrombocytopenia in patients with cirrhosis associated with hepatitis C. N Engl J Med. 2007 Nov 29;357(22):2227-36. PubMed PMID: 18046027.

57: [Eltrombopag increases thrombocyte count and reduces the bleeding risk]. Med Monatsschr Pharm. 2007 Oct;30(10):390-1. German. PubMed PMID: 17966290.

58: Jenkins JM, Williams D, Deng Y, Uhl J, Kitchen V, Collins D, Erickson-Miller CL. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood. 2007 Jun 1;109(11):4739-41. Epub 2007 Feb 27. PubMed PMID: 17327409.