The role of AMG-531 in the treatment of thrombocytopenia in idiopathic thrombocytopenic purpura and myelodysplastic syndromes

Expert Opinion on Biological Therapy

Volumn 8, Issue 7, 2008, Pages 1021-1030

  Tiu, Ramon V ^a   Sekeres, Mikkael A ^a  

^a TAUSSIG CANCER INSTITUTE   (United States)

Author keywords

AMG531; Amgen megakaryopoiesis protein 531; Immune mediated thrombocytopenic purpura; ITP; MDS; Myelodysplastic syndrome; Thrombopoeitin; TPO

Indexed keywords

ACETYLSALICYLIC ACID; CORTICOSTEROID; DANAZOL; GROWTH FACTOR; IMMUNOGLOBULIN D ANTIBODY; IMMUNOGLOBULIN G; MEGAKARYOCYTE GROWTH AND DEVELOPMENT FACTOR; PLACEBO; PROTEIN C MPL; REMIPLISTIM; RITUXIMAB; STEROID; THROMBOPOIETIN; THROMBOPOIETIN RECEPTOR; THYMOCYTE ANTIBODY; UNCLASSIFIED DRUG;

AMINO ACID SEQUENCE; ANOREXIA; ARTHRALGIA; ASTHENIA; BACKACHE; BONE NECROSIS; BREAST TUMOR; CHRONIC DISEASE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTUSION; COUGHING; DIARRHEA; DISEASE SEVERITY; DIZZINESS; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG MECHANISM; DRUG METABOLISM; DRUG RECEPTOR BINDING; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; DYSPNEA; ECCHYMOSIS; EPISTAXIS; EXCORIATION; FATIGUE; FEBRILE NEUTROPENIA; FEVER; FLU LIKE SYNDROME; GINGIVA BLEEDING; HEADACHE; HEMATOMA; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; INJECTION SITE BLEEDING; INSOMNIA; JAPAN; LIMB PAIN; MENORRHAGIA; MUSCULOSKELETAL PAIN; MYALGIA; MYELODYSPLASTIC SYNDROME; NAUSEA AND VOMITING; NONHUMAN; ORAL BLEEDING; ORAL BLISTER; ORAL MUCOSAL DISEASE; PATHOGENESIS; PATHOPHYSIOLOGY; PERIPHERAL EDEMA; PETECHIA; RANDOMIZED CONTROLLED TRIAL; RASH; REGULATORY MECHANISM; REVIEW; RHINOPHARYNGITIS; SCLERITIS; SEQUENCE HOMOLOGY; SIDE EFFECT; SIGNAL TRANSDUCTION; SKIN DEFECT; SPLENECTOMY; THROMBOCYTE COUNT; THROMBOCYTOPENIA; THROMBOCYTOPOIESIS; THROMBOSIS; TREATMENT RESPONSE; UPPER RESPIRATORY TRACT INFECTION; VAGINA BLEEDING;

ANIMALS; BLOOD PLATELETS; CARRIER PROTEINS; CLINICAL TRIALS AS TOPIC; HUMANS; INFUSIONS, INTRAVENOUS; MODELS, BIOLOGICAL; MYELODYSPLASTIC SYNDROMES; PLATELET COUNT; PURPURA, THROMBOCYTOPENIC, IDIOPATHIC; RECEPTORS, FC; RECEPTORS, THROMBOPOIETIN; RECOMBINANT PROTEINS; THROMBOCYTOPENIA; THROMBOPOIETIN; TREATMENT OUTCOME;

ANIMALIA;

EID: 47549100894     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.8.7.1021     Document Type: Review

References (44)

1. Basser RL, O'Flaherty E, Green M, et al. Development of pancytopenia with neutralizing antibodies to thrombopoietin after multicycle chemotherapy supported by megakaryocyte growth and development factor. Blood 2002;99:2599-602
2. Kuter DJ. New thrombopoietic growth factors. Blood 2007;109:4607-16
3. Li J, Yang C, Xia Y, et al. Thrombocytopenia caused by the development of antibodies to thrombopoietin. Blood 2001;98:3241-8 • This paper discusses the mechanism of antibody formation against TPO resulting in thrombocytopenia seen with older thrombopoietic agonists.
4. Panzer S. New therapeutic options for adult chronic immune thrombocytopenic purpura: a brief review. Vox Sang 2008;94:1-5
5. FDA Grants Fast Track Designation for Amgen's AMG 531 and AMG 706 Experimental Therapies to Potentially Treat Life-Threatening Conditions. Business Wire Published online 2004 December 6. Available from: http://findarticles.com/p/articles/mi_m0EIN/is_2004_Dec_6/ai_n7638614 [Last accessed 17 May 2008]
6. Bussel JB, Kuter DJ, George JN, et al. AMG 531, a thrombopoiesis- stimulating protein, for chronic ITP. N Engl J Med 2006;355:1672-81 •• Major Phase 1/2 study on AMG531 in ITP.
7. Wang B, Nichol JL, Sullivan JT. Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin Pharmacol Ther 2004;76:628-38
8. Kumagai Y, Fujita T, Ozaki M, et al. Pharmacodynamics and pharmacokinetics of AMG 531, a thrombopoiesis-stimulating peptibody, in healthy Japanese subjects: a randomized, placebo-controlled study. J Clin Pharmacol 2007;47:1489-97
9. Sun Y, Arends R, Smithson A, et al. A novel thrombopoiesis-stimulating agent, AMG 531: pharmacokinetics and pharmacodynamics in FcRn knock-out and wild type mice. ASH Annual Meeting Abstracts; 2005;106:3575
10. Harrington WJ, Minnich V, Hollingsworth JW, Moore CV. Demonstration of a thrombocytopenic factor in the blood of patients with thrombocytopenic purpura. J Lab Clin Med 1951;38:1-10
11. Harrington WJ, Sprague CC, Minnich V, et al. Immunologic mechanisms in idiopathic and neonatal thrombocytopenic purpura. Ann Intern Med 1953;38:433-69
12. George JN, Woolf SH, Raskob GE, et al. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood 1996;88:3-40
13. Schiavotto C, Rodeghiero F. Twenty years experience with treatment of idiopathic thrombocytopenic purpura in a single department: results in 490 cases. Haematologica 1993;78:22-8
14. 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;80:33-40
15. Branehog I, Kutti J, Weinfeld A. Platelet survival and platelet production in idiopathic thrombocytopenic purpura (ITP). Br J Haematol 1974;27:127-43
16. McMillan R, Luiken GA, Levy R, et al. Antibody against megakaryocytes in idiopathic thrombocytopenic purpura. JAMA 1978;239:2460-2
17. Emmons RV, Reid DM, Cohen RL, et al. Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction. Blood 1996;87:4068-71
18. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet 2008;371:395-403 •• Major Phase 3 study of AMG-531 in ITP.
19. Newland A, Caulier MT, Kappers-Klunne M, et al. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol 2006;135:547-53 •• Major Phase 1/2 study of AMG-531 in ITP.
20. Kantarjian H, Giles F, List A, et al. The incidence and impact of thrombocytopenia in myelodysplastic syndromes. Cancer 2007;109:1705-14
21. Menke DM, Colon-Otero G, Cockerill KJ, et al. Refractory thrombocytopenia. A myelodysplastic syndrome that may mimic immune thrombocytopenic purpura. Am J Clin Pathol 1992;98:502-10
22. Sekeres MA, Fu AZ, Maciejewski JP, et al. A decision analysis to determine the appropriate treatment for low-risk myelodysplastic syndromes. Cancer 2007;109:1125-32
23. Steensma DP, Bennett JM. The myelodysplastic syndromes: diagnosis and treatment. Mayo Clin Proc 2006;81:104-30
24. Hofmann WK, Kalina U, Wagner S, et al. Characterization of defective megakaryocytic development in patients with myelodysplastic syndromes. Exp Hematol 1999;27:395-400
25. Houwerzijl EJ, Blom NR, van der Want JJ, et al. Increased peripheral platelet destruction and caspase-3-independent programmed cell death of bone marrow megakaryocytes in myelodysplastic patients. Blood 2005;105:3472-9
26. van de Loosdrecht AA, Vellenga E. Myelodysplasia and apoptosis: new insights into ineffective erythropoiesis. Med Oncol 2000;17:16-21
27. Biesma DH, van den Tweel JG, Verdonck LF. Immunosuppressive therapy for hypoplastic myelodysplastic syndrome. Cancer 1997;79:1548-51
28. Bourgeois E, Caulier MT, Rose C, et al. Role of splenectomy in the treatment of myelodysplastic syndromes with peripheral thrombocytopenia: a report on six cases. Leukemia 2001;15:950-3
29. Molldrem JJ, Caples M, Mavroudis D, et al. Antithymocyte globulin for patients with myelodysplastic syndrome. Br J Haematol 1997;99:699-705
30. Stadtmauer EA, Cassileth PA, Edelstein M, et al. Danazol treatment of myelodysplastic syndromes. Br J Haematol 1991;77:502-8
31. Wattel E, Cambier N, Caulier MT, et al. Androgen therapy in myelodysplastic syndromes with thrombocytopenia: a report on 20 cases. Br J Haematol 1994;87:205-8
32. Lambertenghi-Deliliers G, Annaloro C, Oriani A, Soligo D. Myelodysplastic syndrome associated with bone marrow fibrosis. Leuk Lymphoma 1992;8:51-5
33. Li X, Pu Q. Megakaryocytopoiesis and apoptosis in patients with myelodysplastic syndromes. Leuk Lymphoma 2005;46:387-91
34. Mattia G, Vulcano F, Milazzo L, et al. Different ploidy levels of megakaryocytes generated from peripheral or cord blood CD34+ cells are correlated with different levels of platelet release. Blood 2002;99:888-97
35. Kantarjian H, Fenaux P, Sekeres MA, et al. Phase 1/2 Study of AMG 531 in thrombocytopenic patients (pts) with low-risk myelodysplastic syndrome (MDS): Update including extended treatment. ASH Annual Meeting Oral Presentation. Blood 2007;110:250
36. Zwierzina H, Rollinger-Holzinger I, Nuessler V, et al. Endogenous serum thrombopoietin concentrations in patients with myelodysplastic syndromes. Leukemia 1998;12:59-64
37. Golshayan AR, Jin T, Maciejewski J, et al. Efficacy of growth factors compared to other therapies for low-risk myelodysplastic syndromes. Br J Haematol 2007;137:125-32
38. Hellstrom-Lindberg E, Gulbrandsen N, Lindberg G, et al. A validated decision model for treating the anaemia of myelodysplastic syndromes with erythropoietin + granulocyte colony-stimulating factor: significant effects on quality of life. Br J Haematol 2003;120:1037-46
39. Adams JA, Liu Yin JA, Brereton ML, et al. The in vitro effect of pegylated recombinant human megakaryocyte growth and development factor (PEG rHuMGDF) on megakaryopoiesis in normal subjects and patients with myelodysplasia and acute myeloid leukaemia. Br J Haematol 1997;99:139-46
40. Cheson BD, Greenberg PL, Bennett JM, et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood 2006;108:419-25
41. Amgen: Statement on successful outcome of romiplostim panel meeting. Available from: http://www.amgen.com/media/media_pr_detail.jsp?releaseID=1118084 [Last accessed May 17 2008]
42. Amgen: An open label extension study evaluating the safety of long term dosing of AMG 531 in thrombocytopenic subjects with myelodysplastic syndromes (MDS). 2007. Available from: http://www.amgentrials.com/trial_details.cfm?tid= 14878&local=0 [Last accessed May 17 2008]
43. Amgen: A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of AMG 531 Treatment of Subjects With Low or Intermediate Risk Myelodysplastic Syndrome (MDS) Receiving Hypomethylating Agents. 2006. Available from: http://www.amgentrials.com/trial_details.cfm?tid= 9817&local=0 [Last accessed May 17 2008]
44. Amgen: A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of AMG 531 Treatment of Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) Receiving Lenalidomide. 2006. Available from: http://www.amgentrials.com/trial_details.cfm?tid= 11353&local=0 [Last accessed May 17 2008]