Treatment of immune thrombocytopenic purpura: Focus on eltrombopag

Biologics: Targets and Therapy

Volumn 3, Issue , 2009, Pages 151-157

  Rice, Lawrence ^a  

^a HOUSTON METHODIST HOSPITAL   (United States)

Author keywords

Autoimmune disorder; Eltrombopag; Immune thrombocytopenic purpura; Serum thrombopoietin

Indexed keywords

EID: 77953412868     PISSN: 11775475     EISSN: 11775491     Source Type: Journal    
DOI: 10.2147/BTT.S3130     Document Type: Review

References (56)

1. Altman, LK. Who goes first. The story of self experimentation in medicine. 1st. Ed. New York: Random House; 1987.
2. Arimura G, Harrington WJ, Minnich V. The autoimmune thrombocytopenias. Prog Hematol. 1956;1:166-192.
3. McMillan R. Antiplatelet antibodies in chronic adult immune thrombocytopenic purpura: assays and epitopes. J Pediatr Hematol Oncol. 2003;25 Suppl 1:S57-S61.
4. George JN, Woolf SH, Raskob GE. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood. 1996;88(1):3-46.
5. Seagal JB, Powe NR. Prevalence of immune thrombocytopenia: analyses of administrative data. J Thromb Haemost. 2008;4(11):2377-2383.
6. Fuedjo-Tepie MA, Robinson D, Bennett D. Prevalence of diagnosed chronic immune thrombocytopenic purpura in the US: analysis of a large US claim database: a rebuttal. J Thromb Haemost. 2008;6(4):711-712.
7. Satia JA. Epidemiology of immune thrombocytopenic purpura in the United Kingdom, Germany, and the Netherlands. Blood. 2005;106(Suppl):Abstract 5554.
8. Blanchette V, Bolton-Maggs P. Childhood immune thrombocytopenic purpura: diagnosis and management. Pediatr Clin North Am. 2008;55(2):393-420.
9. Chang M, Nakagawa PA, Williams SA, et al. Immune thrombocytopenic purpura (ITP) plasma and purified ITP monoclonal autoantibodies inhibit megakaryocytopoiesis in vitro. Blood. 2003;102(3):887-895.
10. Mazzucconi MG, Fazi P, Bernasconi S, et al; for Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA) Thrombocytopenia Working Party. Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood. 2007;109(4):1401-1407.
11. Zaja F, Baccarani M, Mazza P, et al. A prospective randomized study comparing rituximab and dexamethasone vs dexamethasone alone in ITP: Results of final analysis and long term follow up. Blood. 2008;112(suppl):Abstract 1.
12. AR Gaines. Acute onset hemoglobinemia and/or hemoglobinuria and sequelae following Rho(D) immune globulin intravenous administration in immune thrombocytopenic purpura patients. Blood. 2000;95:2523-2529.
13. Simanek R, Panzer S, Lechner K, Pabinger I. Late spontaneous remissions in severe adult autoimmune thrombocytopenia. Ann Hematol. 2007;86:705-710.
14. Kojouri K, Vesely SK, Terrell DR, George JN. Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess long-term platelet count responses, prediction of response, and surgical complications. Blood. 2004;104(9):2623-2634.
15. Dolan JP, Sheppard BC, DeLoughery TG. Splenectomy for immune thrombocytopenic purpura: surgery for the 21st century. Am J Hematol. 2008;83(2):93-96.
16. Arnold DM, Dentali F, Crowther MA, et al. Systematic review: efficacy and safety of rituximab for adults with idiopathic thrombocytopenic purpura. Ann Intern Med. 2007;146(1):25-33.
17. Zhou Z, Yang R. Rituximab treatment for chronic refractory idiopathic thrombocytopenic purpura. Crit Rev Oncol Hematol. 2008;65(1):21-31.
18. Medeot M, Zaja F, Vianelli N, et al. Rituximab therapy in adult patients with relapsed or refractory immune thrombocytopenic purpura: longterm follow-up results. Eur J Haematol. 2008;81(3):165-169.
19. Patel V, Mihatov N, Cooper N, Stasi R, Cunningham-Rundles S, Bussel JB. Long term follow-up of patients with immune thrombocytopenic purpura whose initial response to rituximab lasted a minimum of 1 year. Blood (suppl). 2006;Suppl:abstract 479.
20. Kim SW, Rice L, McCarthy JJ. Efficacy of danazol with autoimmune thrombocytopenia. Clin Appl Thrombosis/Hemostasis. 1997;3:251-255.
21. Maloisel F, Andrès E, Zimmer J, et al. Danazol therapy in patients with chronic idiopathic thrombocytopenic purpura: long-term results. Am J Med. 2004;116(9):590-594.
22. Rice L. Danazol, idiopathic thrombocytopenic purpura, and thrombopoietin. Am J Med. 2004;117(12):972-973.
23. George JN, Kojouri K, Perdue JJ, Vesely SK. Management of patients with chronic, refractory idiopathic thrombocytopenic purpura. Semin Hematol. 2000;37(3):290-298.
24. Snyder HW Jr, Cochran SK, Balint JP Jr, Bertram JH, Mittelman A, Guthrie TH Jr, Jones FR. Experience with protein A-immunoadsorption in treatment-resistant adult immune thrombocytopenic purpura. Blood. 1992;79:2237-2245.
25. Kaushansky K. The molecular mechanisms that control thrombopoiesis. J Clin Invest. 2005;115(12):3339-3347.
26. Kuter DJ, Begley CG. Recombinant human thrombopoietin: basic biology and evaluation of clinical studies. Blood. 2002;100(10): 3457-3469.
27. Li J, Yang C, Xia Y, et al. Thrombocytopenia caused by the development of antibodies to thrombopoietin. Blood. 2001;98(12):3241-3248.
28. 111Indium-oxine-labeled platelets in idiopathic thrombocytopenic purpura: a quantitative study. Am J Hematol. 1982;12(2):167-177.
29. Ballem PJ, Segal GM, Stratton JR, et al. Mechanisms of thrombocytopenia in chronic autoimmune thrombocytopenic purpura. Evidence of both impaired platelet production and increased platelet clearance. J Clin Invest. 1987;80(1):33-40.
30. Siegel RS, Rae JL, Barth S, et al. Platelet survival and turnover: important factors in predicting response to splenectomy in immune thrombocytopenic purpura. Am J Hematol. 1989;30(4):206-212.
31. Louwes H, Zeinali Lathori OA, Vellenga E, de Wolf JT. Platelet kinetic studies in patients with idiopathic thrombocytopenic purpura. Am J Med. 1999;106(4):430-434.
32. Hoffman R, Zaknoen S, Yang HH, et al. An antibody cytotoxic to megakaryocyte progenitor cells in a patient with immune thrombocytopenic purpura. N Engl J Med. 1985;312(18):1170-1174.
33. Takahashi R, Sekine N, Nakatake T. Influence of monoclonal antiplatelet glycoprotein antibodies on in vitro human megakaryocyte colony formation and proplatelet formation. Blood. 1999;93(6):1951-1958.
34. McMillan R, Wang L, Tomer A, Nichol J, Pistillo J. Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP. Blood. 2004;103(4):1364-1369.
35. Emmons RVB, Reid DM, Cohen RL, Meng G, Young NS, Dunbar CE, Shulman RE. Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction. Blood. 1996;87:4068-4071.
36. Nichol JL. Endogenous (e)TPO levels in health and disease. Possible clues for therapeutic intervention. Stem Cells. 1998;16(suppl 2):165-175.
37. Rice L, Nichol JL, McMillan R, Roskos LK, Bacile M. Cyclic immune thrombocytopenia responsive to thrombopoietic growth factor therapy. Am J Hematol. 2001;68(3):210-214.
38. Bose P, Hussein KK, Terrell DR, Berger D, Rice L, George JN. Successful treatment of cyclic thrombocytopenia with thrombopoietin-mimetic agents: A report of two patients. [submitted].
39. Nomura S, Dan K, Hotta T, et al. Effects of pegylated recombinant human megakaryocyte growth and development factor in patients with idiopathic thrombocytopenic purpura. Blood. 2002;100(2):728-730.
40. Harker LA, Marzec UM, Novembre F, et al. Treatment of thrombocytopenia in chimpanzees infected with human immunodeficiency virus by pegylated recombinant human megakaryocyte growth and development factor. Blood. 1998;91(12):4427-4433.
41. Rice L, Drug evaluation: AMG-531 for the treatment of thrombocytopenias, Curr Opin Invest Drugs. 2006;7(9):834-841.
42. Bussel JB, Kuter DJ, George JN, et al. AMG 531, a thrombopoiesisstimulating protein, for chronic ITP. N Engl J Med. 2006;355(16): 1672-1681.
43. Kuter DJ, Bussel JB, Lyons RM. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008;371(9610):395-403.
44. Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2008. In press.
45. George JN, Mathias SD, Go RS, et al. Improved quality of life for romiplostim-treated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo-controlled trials. Br J Haematol. 2009;144:409-415.
46. Kuter DJ, Bain B, Mufti G, Bagg A, Hasserjian RP. Bone marrow fibrosis: pathophysiology and clinical significance of increased bone marrow stromal fibres. Br J Haematol. 2007;139(3):351-362.
47. Erickson-Miller CL, Delorme E, Tian SS, et al. Preclinical Activity of Eltrombopag (SB-497115), an Oral, Non-peptide Thrombopoietin Receptor Agonist. Stem Cells. 2008. In press.
48. FDA website. http://www.fda.gov/ohrms/dockets/ac/08/briefing/2008-4366b1-02-GSK.pdf. Accessed 11:00CET 02 December 2008. Copy on file.
49. Jenkins JM, Williams D, Deng Y, et al. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood. 2007;109(11):4739-4741.
50. McHutchison JG, Dusheiko G, Shiffman ML, et al. Eltrombopag for thrombocytopenia in patients with cirrhosis associated with hepatitis C. N Engl J Med. 2007;357(22):2227-2236.
51. Bussel JB, Cheng G, Saleh MN, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007;357(22):2237-2247.
52. Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009;373:641-648.
53. Bussel JB, Cheng G, Saleh MN, et al. Safety and efficacy of long-term treatment with oral eltrombopag for chronic idiopathic thrombocytopenic purpura. Blood. 2008;112 suppl:Abstract 3432.
54. Cheng G, Bussel, JB, Saleh MN, et al. Eltrombopag delivers clinical benefit in chronic idiopathic thrombocytopenic purpura (ITP) patients not achieving platelet counts ≥50,000/μL-data from the EXTEND study. Blood. 2008;112 suppl:Abstract 3430.
55. Saleh MN, Bussel JB, Cheng G, et al. Eltrombopag is efficacious in patients with refractory chronic idiopathic thrombocytopenic purpura (ITP)-data from the EXTEND Study. Blood. 2008;112 suppl:Abstract 401.
56. Promacta: Highlights of Prescribing Information. Available at: http:// www.fda.gov/cder/foi/label/2008/022291lbl.pdf1