Treatment options for chronic refractory idiopathic thrombocytopenic purpura in adults: Focus on romiplostim and eltrombopag

Pharmacotherapy

Volumn 30, Issue 7, 2010, Pages 666-683

  Chouhan, Jyoti D ^a,b   Herrington, Jon D ^a,b,c  

^a SCOTT AND WHITE MEMORIAL HOSPITAL   (United States)

^b UNIVERSITY OF TEXAS AT AUSTIN   (United States)

^c TEXAS A AND M HEALTH SCIENCE CENTER   (United States)

Author keywords

AMG 531; Eltrombopag; Idiopathic thrombocytopenic purpura; ITP; Romiplostim; SB497115; Thrombopoietin; TPO

Indexed keywords

AZATHIOPRINE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; DANAZOL; DAPSONE; ELTROMBOPAG; ETOPOSIDE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PLACEBO; PREDNISONE; PROCARBAZINE; RITUXIMAB; ROMIPLOSTIM; VINCRISTINE; BENZOIC ACID DERIVATIVE; FC RECEPTOR; HYBRID PROTEIN; HYDRAZINE DERIVATIVE; MONOCLONAL ANTIBODY; PYRAZOLE DERIVATIVE; THROMBOPOIETIN;

ARTERY THROMBOSIS; ARTHRALGIA; CLINICAL TRIAL; DEEP VEIN THROMBOSIS; DIARRHEA; DIZZINESS; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG WITHDRAWAL; ECCHYMOSIS; EPISTAXIS; FATIGUE; HEART INFARCTION; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; INJECTION SITE CONTUSION; INSOMNIA; ISCHEMIC HEART DISEASE; LIMB PAIN; MYALGIA; ORAL BLISTER; PETECHIA; REVIEW; RHINOPHARYNGITIS; SIDE EFFECT; SPLENECTOMY; THROMBOCYTE COUNT; THROMBOCYTOPENIA; THROMBOPHLEBITIS; TRANSIENT ISCHEMIC ATTACK; TREATMENT RESPONSE; ADULT; CHRONIC DISEASE; DRUG EFFECTS; IMMUNOLOGY; PHASE 3 CLINICAL TRIAL (TOPIC); PURPURA, THROMBOCYTOPENIC, IDIOPATHIC; THROMBOCYTOPOIESIS;

ADULT; ANTIBODIES, MONOCLONAL; ANTIBODIES, MONOCLONAL, MURINE-DERIVED; BENZOATES; CHRONIC DISEASE; CLINICAL TRIALS, PHASE III AS TOPIC; HUMANS; HYDRAZINES; PLATELET COUNT; PURPURA, THROMBOCYTOPENIC, IDIOPATHIC; PYRAZOLES; RECEPTORS, FC; RECOMBINANT FUSION PROTEINS; THROMBOPOIESIS; THROMBOPOIETIN;

EID: 77954096636     PISSN: 02770008     EISSN: None     Source Type: Journal    
DOI: 10.1592/phco.30.7.666     Document Type: Review

References (38)

1. George JN, Woolf SH, Raskob GE, et al. Diagnosis and treatment of idiopathic thrombocytopenic purpura: recommendations of the American Society of Hematology. Ann Intern Med 1997;126:319-26.
2. Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med 2002;346;995-1008.
3. Bussel J, Cines D. Immune thrombocytopenic purpura, neonatal alloimmune thrombocytopenia and post-transfusion purpura. In: Hoffman R, Benz EJ, Shattil SS, Furie B, Cohen HJ, eds. Hematology: basic principles and practice, 4th ed. Philadelphia: Elsevier Inc., 2005:2269-85.
4. Kuter DJ. New thrombopoietic growth factors. Blood 2007;109:4607-16.
5. Cines DB, Bussel JB. How I treat idiopathic thrombocytopenic purpura. Blood 2005;106:2244-51.
6. Quiquandon I, Fenaux P, Caulier MT, Pagniez D, Huart JJ, Bauters F. Reevaluation of the role of azathioprine in the treatment of adult chronic idiopathic thrombocytopenic purpura: a report on 53 cases. Br J Haematol 1990;74:223-8. (Pubitemid 20041099)
7. Figueroa M, Ghesen J, Hammond D, et al. Combination chemotherapy in refractory immune thrombocytopenic purpura. N Engl J Med 1993;328;1226-9.
8. McMillan R. Long-term outcomes after treatment for refractory immune thrombocytopenic purpura. N Engl J Med 2001;344: 1402-3.
9. Reiner A, Gernsheimer T, Slichter SJ. Pulse cyclophosphamide therapy for refractory autoimmune thrombocytopenic purpura. Blood 1995;85:351-8.
10. Kappers-Klunne MC, van't Veer MB. Cyclosporin A for the treatment of patients with chronic idiopathic thrombocytopenic purpura refractory to corticosteroids or splenectomy. Br J Haematol 2001;114:121-5.
11. Emilia G, Morselli M, Luppi M, et al. Long-term salvage therapy with cyclosporin A in refractory idiopathic thrombocytopenic purpura. Blood 2002;99:1482-5.
12. Maloisel F, Andres E, Zimmer J, et al. Danazol therapy in patients with chronic idiopathic thrombocytopenic purpura: long-term results. Am J Med 2004;116:590-4.
13. Godeau B, Durand JM, Roudot-Thoraval F, et al. Dapsone for chronic autoimmune thrombocytopenic purpura: a report of 66 cases. Br J Haematol 1997;97:336-9.
14. Provan D, Moss AJ, Newland AC, Bussel JB. Efficacy of mycophenolate mofetil as single-agent therapy for refractory immune thrombocytopenic purpura. Am J Hematol 2006;81:19-25.
15. Cooper N, Stasi R, Cunningham-Rundles S, et al. The efficacy and safety of B-cell depletion with anti-CD20 monoclonal antibody in adults with chronic immune thrombocytopenic purpura. Br J Haematol 2004;125:232-9.
16. U.S. Food and Drug Administration. FDA approves first bone marrow stimulator to treat immune-related low platelet counts, August 2008. Available from www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2008/ucm116938.htm. Accessed August 25, 2008.
17. U.S. Food and Drug Administration. Eltrombopag. Available from www.fda.gov/AboutFDA/CentersOffices/CDER/ucm129214.htm. Accessed January 15, 2009.
18. Bussel JB, Kuter DJ, Geroge JN, et al. AMG 531, a thrombopoiesis- stimulating protein, for chronic ITP. N Engl J Med 2006;355:1672-81.
19. 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.
20. 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 2009;113:2161-71.
21. Amgen, Inc. Nplate (romiplostim ) package insert. Thousand Oaks, CA; 2008.
22. PDR Staff, eds. Red book: pharmacy's fundamental reference, 113th ed. Montvale, NJ: Thomson Healthcare, 2009.
23. Amgen, Inc. A randomized, controlled, open-label study evaluating the efficacy and tolerability of AMG 531 versus medical standard of care as chronic therapy for non-splenectomized subjects with immune (idiopathic) thrombocytopenic purpura. ClinicalTrials.gov identifier NCT00415532. Available from www.clinicaltrials.gov. Accessed September 17, 2008.
24. Amgen, Inc. A randomized, double-blind, placebo-controlled phase 1/2 study to determine the safety and efficacy of AMG 531 in thrombocytopenic pediatric subjects with chronic immune (idiopathic) thrombocytopenic purpura. ClinicalTrials.gov identifier NCT00515203. Available from www.clinicaltrials. gov. Accessed September 17, 2008.
25. Amgen, Inc. An open label, sequential, dose escalation study to evaluate the safety and efficacy of romiplostim in thrombocytopenic subjects with low or intermediate-1 risk myelodysplastic syndrome (MDS). ClinicalTrials.gov identifier NCT00303472. Available from www.clinicaltrials.gov. Accessed September 17, 2008.
26. Amgen, Inc. A randomized, double blind, placebo controlled study evaluating the efficacy and safety of romiplostim treatment of thrombocytopenia in subjects with low or intermediate-1 risk myelodysplastic syndrome (MDS). ClinicalTrials.gov identifier NCT00614523. Available from www.clinicaltrials. gov. Accessed September 17, 2008.
27. Amgen, Inc. Phase I/II study of AMG 531 in patients with advanced malignancy receiving treatment with carboplatin. ClinicalTrials.gov identifier NCT00147225. Available from www.clinicaltrials.gov. Accessed September 23, 2009.
28. Amgen, Inc. Study of AMG 531 to evaluate the safety and efficacy in patients with non-Hodgkin's lymphoma. ClinicalTrials.gov identifier NCT00299182. Available from www.clinicaltrials.gov. Accessed September 23, 2009.
29. Jenkins JM, Williams D, Deng Y, et al. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood 2007;109:4739-41.
30. Bussel JB, Cheng G, Saleh M, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007;357:2237-47.
31. Cheng G, Saleh MN, Bussel JB, et al. Oral eltrombopag for the long-term treatment of patients with chronic idiopathic thrombocytopenic purpura: results of a phase III, double-blind, placebo-controlled study (RAISE) [abstract]. Presented at the American Society of Hematology annual meeting, San Francisco, CA, December 6-9, 2008.
32. Cancer Therapy Evaluation Program. Common terminology criteria for adverse events, version 3.0. Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Department of Health and Human Services, August 9, 2006. Available from http://ctep.cancer.gov/ protocolDevelopment/electronic-applications/docs/ctcae-index. pdf. Accessed November 13, 2009.
33. GlaxoSmithKline. Promacta (eltrombopag) package insert. Research Triangle Park, NJ; 2008.
34. Kellu A, Jagiello-Gruszfeld AJ, Bondarenko I, et al. Hepatobiliary (HB) safety of eltrombopag administered after carboplatin + paclitaxel (carb + pac) in patients (pts) with solid tumors [abstract]. J Clin Oncol 2009;27(May 20 suppl):320667. Available from http://www.asco.org/ASCOv2/Meetings/Abstracts? &vmview=abst-detail-view&confID=65&abstractID=35164. Accessed June 12, 2009.
35. GlaxoSmithKline. EXTEND (eltrombopag extended dosing study): an extension study of eltrombopag olamine in adults with idiopathic thrombocytopenic purpura (ITP), previously enrolled in an eltrombopag study. ClinicalTrials.gov identifier NCT00351468. Available from www.clinicaltrials.gov. Accessed September 17, 2008.
36. GlaxoSmithKline. An open-label repeat dosing study of eltrombopag olamine in adult subjects with chronic idiopathic thrombocytopenic purpura (ITP). ClinicalTrials.gov identifier NCT00424177. Available from www.clinicaltrials. gov. Accessed September 17, 2008.
37. GlaxoSmithKline. A double-blind, randomized, multicenter, placebo-controlled, parallel group, dose ranging study to assess the efficacy, safety, and pharmacokinetics of an oral thrombopoietin receptor agonist administered at 50, 75, and 100 mg to cancer patients receiving multiple cycles of chemotherapy. ClinicalTrials.gov identifier NCT00102726. Available from www.clinicaltrials.gov. Accessed September 17, 2008.
38. GlaxoSmithKline. Randomised, double-blind, placebocontrolled, multi-centre study to evaluate the safety and efficacy of eltrombopag to reduce the need for platelet transfusion in thrombocytopenic subjects with chronic liver disease undergoing elective invasive procedures. ClinicalTrials.gov identifier NCT00678587. Available from www.clinicaltrials.gov. Accessed September 17, 2008.