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Expert Opinion on Emerging Drugs Volume 13, 2008 - Issue 2
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Review

Emerging drugs for idiopathic thrombocytopenic purpura in adults

Xiaofang Li Shandong University, Qilu Hospital, Department of Haematology, Jinan, Shandong, 250012, P R China +86 0531 82169879; +86 0531 86927544; [email protected]
&
Ming Hou Shandong University, Qilu Hospital, Department of Haematology, Jinan, Shandong, 250012, P R China +86 0531 82169879; +86 0531 86927544; [email protected]
Pages 237-254 | Published online: 08 Jun 2008

Bibliography

  • Levine SP. Thrombocytopenia. In: Greer JP, Foerster J, Lukens JN, et al., editors, Wintrobe's clinical haematology. 11th edition. Philadelphia: Lippincott Williams & Wilkins. Reprinted by Jinan, PRC: Shandong Science & Technology Press; 2004. p. 1529-72
  • Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med 2002;346:995-1008
  • 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
  • Provan D, Newland A, Bolton-Maggs P, et al. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol 2003;120:574-96
  • Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood 1999;94:909-13
  • Neylon AJ, Saunders PWG, Howard MR, et al. Clinically significant newly presenting autoimmune thrombocytopenic purpura in adults: a prospective study of a population-based cohort of 245 patients. Br J Haematol 2003;122:966-74
  • 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
  • McMillan R. Autoantibodies and autoantigens in chronic immune thrombocytopenic purpura. Semin Hematol 2000;37:239-48
  • Coopamah M, Garvey MB, Freedman J, Semple JW. Cellular immune mechanisms in autoimmune thrombocytopenic purpura: an update. Transfus Med Rev 2003;17:69-80
  • Andersson PO, Wadenvik H. Chronic idiopathic thrombocytopenic purpura (ITP): molecular mechanisms and implications for therapy. Expert Rev Mol Med 2004;6:1-17
  • Kuwana M, Kaburaki J, Ikeda Y. Autoreactive T cells to platelet GPIIb-IIIa in immune thrombocytopenic purpura. Role in production of anti-platelet autoantibody. J Clin Invest 1998;102:1393-402
  • Olsson B, Andersson PO, Jernas M, et al. T-cell-mediated cytotoxicity toward platelets in chronic idiopathic thrombocytopenic purpura. Nat Med 2003;9:1123-4
  • Zhang F, Chu X, Wang L, et al. Cell-mediated lysis of autologous platelets in chronic idiopathic thrombocytopenic purpura. Eur J Haematol 2006;76:427-31
  • Catani L, Fagiolia ME, Tazzari PL, et al. Dendritic cells of immune thrombocytopenic purpura (ITP) show increased capacity to present apoptotic platelets to T lymphocytes. Exp Hematol 2006;34:879-87
  • Semple JW, Milev Y, Cosgrave D, et al. Differences in serum cytokine levels in acute and chronic autoimmune thrombocytopenic purpura: relationship to platelet phenotype and antiplatelet T-cell reactivity. Blood 1996;87:4245-54
  • Solanilla A, Pasquet JM, Viallard JF, et al. Platelet-associated CD154 in immune thrombocytopenic purpura. Blood 2005;105:215-8
  • Hou M, Andersson PO, Stockelberg D, et al. Plasma thrombopoietin levels in thrombocytopenic states: implication for a regulatory role of bone marrow megakaryocytes. Br J Haematol 1998;101:420-4
  • Heyns AP, Badenhorst PN, Lotter MG, et al. Platelet turnover and kinetics in immune thrombocytopenic purpura: results with autologous 111In-labeled platelets and homologous 51Cr-labeled platelets differ. Blood 1986;67:86-92
  • Chang M, Nakagawa PA, Williams SA, et al. Immune thrombocytopenic purpura (ITP) plasma and purified ITP monoclonal autoantibodies inhibit megakaryocytopoiesis in vitro. Blood 2003;102:887-95
  • McMillan R, Wang L, Tomer A, et al. Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP. Blood 2004;103:1364-9
  • Li SG, Wang L, Zhao CH, et al. CD8+ T cells suppress autologous megakaryocyte apoptosis in idiopathic thrombocytopenic purpura. Br J Haematol 2007;139:605-11
  • Andemariam B, Psaila B, Bussel JB. Novel thrombopoietic agents. Hematology Am Soc Hematol Educ Program 2007;2007:106-13
  • Nomura S, Matsuzaki T, Ozaki Y, et al. Clinical significance of HLA-DRB1*0410 in Japanese patients with idiopathic thrombocytopenic purpura. Blood 1998;91:3616-22
  • van Sorge NM, van der Pol WL, van de Winkel JG. FcgammaR polymorphisms: implications for function, disease susceptibility and immunotherapy. Tissue Antigens 2003;61:189-202
  • Takahashi T, Yujiri T, Shinohara K, et al. Molecular mimicry by Helicobacter pylori CagA protein may be involved in the pathogenesis of H. pylori associated chronic idiopathic thrombocytopenic purpura. Br J Haematol 2004;124:91-6
  • Kuwana M, Kaburaki J, Kitasato H, et al. Immunodominant epitopes on glycoprotein IIb-IIIa recognized by autoreactive T cells in patients with immune thrombocytopenic purpura. Blood 2001;98:130-9
  • Franchini M, Cruciani M, Mengoli C, et al. Effect of Helicobacter pylori eradication on platelet count in idiopathic thrombocytopenic purpura: a systematic review and meta-analysis. J Antimicrob Chemother 2007;60:237-46
  • Portielje JEA, Westendorp RGJ, Kluin-Nelemans HC, Brand A. Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood 2001;97:2549-54
  • Cortelazzo S, Finazzi G, Buelli M, et al. High risk of severe bleeding in aged patients with chronic idiopathic thrombocytopenic purpura. Blood 1991;77:31-3
  • Cines DB, Bussel JB. How I treat idiopathic thrombocytopenic purpura (ITP). Blood 2005;106:2244-51
  • George JN. Management of patients with refractory immune thrombocytopenic purpura. J Thromb Haemos 2006;8:1664-72
  • Vesely SK, Perdue JJ, Rizvi MA, et al. Management of adult patients with persistent idiopathic thrombocytopenic purpura following splenectomy: a systematic review. Ann Intern Med 2004;140:112-20
  • 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;146:25-33
  • Kuwana M, Ikeda Y. Helicobacter pylori and immune thrombocytopenic purpura: unsolved questions and controversies. Int J Hematol 2006;84:309-15
  • Veneri D, De Matteis G, Solero P, et al. Analysis of B- and T-cell clonality and HLA class II alleles in patients with idiopathic thrombocytopenic purpura: correlation with Helicobacter pylori infection and response to eradication treatment. Platelets 2005;16:307-11
  • Maeda S, Ogura K, Yoshida H, et al. Major virulence factors, VacA and CagA, are commonly positive in Helicobacter pylori isolates in Japan. Gut 1998;42:338-43
  • Stasi R, Stipa E, Masi M, et al. Long-Term observation of 208 adults with chronic idiopathic thrombocytopenic purpura. Am J Med 1995;98:436-42
  • Sailer T, Lechner K, Panzer S, et al. The course of severe autoimmune thrombocytopenia in patients not undergoing splenectomy. Haematologica 2006;91:1041-5
  • Cohen YC, Djulbegovic B, Shamai-Lubovitz O, Mozes B. The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med 2000;160:1630-8
  • Mathias SD, Bussel JB, George JN, et al. A disease-specific measure of health-related quality of life in adults with chronic immune thrombocytopenic purpura: psychometric testing in an open-label clinical trial. Clin Ther 2007;29:950-62
  • Silverman GJ, Weissman S. Rituximab therapy and autoimmune disorders: prospects for anti-B cell therapy. Arthritis Rheum 2003;48:1484-92
  • Cartron G, Watier H, Golay J, Solal-Celigny P. From the bench to the bedside: ways to improve rituximab efficacy. Blood 2004;104:2635-42
  • 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
  • Cooper N, Evangelista ML, Amadori S, Stasi R. Should rituximab be used before or after splenectomy in patients with immune thrombocytopenic purpura? Curr Opin Hematol 2007;14:642-6
  • Frampton JE, Wagstaff AJ. Alemtuzumab. Drugs 2003;63:1229-43
  • Lim SH, Hale G, Marcus RE, et al. CAMPATH-1 monoclonal antibody therapy in severe refractory autoimmune thrombocytopenic purpura. Br J Haematol 1993;84:542-4
  • Liossis SNC, Sfikakis PP. Costimulation blockade in the treatment of rheumatic diseases. Biodrugs 2004;18:95-102
  • Kuwana M, Nomura S, Fujimura K, et al. Effect of a single injection of humanized anti-CD154 monoclonal antibody on the platelet-specific autoimmune response in patients with immune thrombocytopenic purpura. Blood 2004;103:1229-36
  • Kuter DJ. New thrombopoietic growth factors. Blood 2007;109:4607-16
  • Bussel JB, Kuter DJ, George JN, et al. AMG531, a thrombopoiesis-stimulating protein, for chronic ITP. N Engl J Med 2006;355:1672-81
  • 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
  • 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
  • Kaushansky K. Small molecule mimics of hematopoietic growth factors: improving on Mother Nature? Leukemia 2001;15:673-4
  • Jenkins J, Williams D, Deng Y, et al. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood 2007;109:4739-41
  • Bussel JB, Cheng G, Saleh MN, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007;357:2237-47
  • Repeated exposure to eltrombopag in adults with idiopathic thrombocytopenic purpura (REPEAT). NCT00424177. Available from: http://www.clinicaltrials.gov/ct2/show/NCT00424177?term=NCT00424177&rank=1
  • RAISE. randomized placebo-controlled idiopathic thrombocytopenic purpura (ITP) study with eltrombopag. NCT00370331. Available from: http://www.clinicaltrials.gov/ct2/show/NCT00370331?term=NCT00370331&rank=
  • Advances in the treatment of immune thrombocytopenic purpura: report from the 2006 American Society of Hematology Meeting. Available from: http://professional.cancerconsultants.com/conference_ash_2006.aspx?id=38854
  • Study of AKR-501 tablets taken orally once daily for 28 days in patients with chronic ITP. NCT00441090. Available from: http://www.clinicaltrials.gov/ct2/show/NCT00441090?term=NCT00441090&rank=1
  • Ligand presents positive LGD-4665 Phase I clinical trial results at the American Society of Hematology Annual Meeting (ASH). Available from: http://investors.ligand.com/releasedetail.cfm?ReleaseID=280413
  • Clarkson SB, Bussel JB, Kimberly RP, et al. Treatment of refractory immune thrombocytopenic purpura with an anti-Fcγ-receptor antibody. N Engl J Med 1986;314:1236-9
  • Medarex and Aventis Behring announce positive clinical trial results for new autoimmune disease drug. Available from: http://www.medarex.com/cgi-local/item.pl/20001204-555458
  • Rigel R788 raises platelet counts in immune thrombocytopenic purpura (ITP) patients in Phase 2 study. Available from: http://ir.rigel.com/phoenix.zhtml?c=120936&p=irol-newsArticle&ID=1075637&highlight
  • SuppreMol receives orphan drug designation for SM101. Available from: http://www.biospace.com/news_story.aspx?NewsEntityId=69497
  • Protalex reports clinical progress of PRTX-100 in rheumatoid arthritis and idiopathic thrombocytopenic purpura. Available from: http://prtx.client.shareholder.com/releasedetail.cfm?ReleaseID=251389
  • Pierce LR, Jain N. Risks associated with the use of intravenous immunoglobulin. Trans Med Rev 2003;17:241-51
  • Sorensen R. Expert opinion regarding clinical and other outcome considerations in the formulary review of immune globulin. J Manag Care Pharm 2007;13:278-83
  • CSL Behring announces FDA approval of Privigen™-First proline-stabilized 10 percent liquid IVIg. Available from: http://www.cslbehring.com/s1/cs/enco/1151517263302/news/1182280875254/prdetail.htm
  • Kallenberg CGM. A 10% ready-to-use intravenous human immunoglobulin offers potential economic advantages over a lyophilized product in the treatment of primary immunodeficiency. Clin Exp Immunol 2007;150:437-41
  • Crow AR, Song S, Siragam V, Lazarus AH. Mechanisms of action of intravenous immunoglobulin in the treatment of immune thrombocytopenia. Pediatr Blood Cancer 2006;47:710-3
  • Siragam V, Crow AR, Brinc D, et al. Intravenous immunoglobulin ameliorates ITP via activating Fcγ receptors on dendritic cells. Nat Med 2006;12:688-92
  • Zaja F, Vianelli N, Battista M, et al. Earlier administration of Rituximab allows higher rate of long-lasting response in adult patients with autoimmune thrombocytopenia. Exp Hematol 2006;34:571-2
  • Provan D, Butler T, Evangelista ML, et al. Activity and safety profile of low-dose rituximab for the treatment of autoimmune cytopenias in adults. Haematologica 2007;92:1695-8
  • Stasi R, Stipa E, Forte V, et al. Variable patterns of response to rituximab treatment in adults with chronic idiopathic thrombocytopenic purpura. Blood 2002;99:3872-3
  • Stasi R, Del Poeta G, Stipa E, et al. Response to B-cell depleting therapy with rituximab reverts the abnormalities of T-cell subsets in patients with idiopathic thrombocytopenic purpura. Blood 2007;110:2924-30
  • Perrotta AL. Re-treatment of chronic idiopathic thrombocytopenic purpura with rituximab: literature review. Clin Appl Thromb Hemost 2006;12:97-100
  • Kuter DJ, Begley CG. Recombinant human thrombopoietin: Basic biology and evaluation of clinical studies. Blood 2002;100:3457-69
  • Zhang XL, Peng J, Sun JZ, et al. Modulation of immune response with cytotoxic T-lymphocyte-associated antigen 4 immunoglobulin-induced anergic T cells in chronic idiopathic thrombocytopenic purpura. J Thromb Haemost 2008;6:158-65
  • Sutherland AP, Mackay F, Mackay CR. Targeting BAFF: immunomodulation for autoimmune diseases and lymphomas. Pharmacol Ther 2006;112:774-86
  • Dillon SR, Gross JA, Ansell SM, Novak AJ. An APRIL to remember: novel TNF ligands as therapeutic targets. Nat Rev Drug Discov 2006;5:235-46
  • Emmerich F, Bal G, Barakat A, et al. High-level serum B-cell activating factor and promoter polymorphisms in patients with idiopathic thrombocytopenic purpura. Br J Haematol 2007;136:309-14
  • Yu HM, Liu YF, Hou M. BAFF – an essential survival factor for B cells: links to genesis of ITP and may be of therapeutic target. Med Hypotheses 2008;70:40-2
  • Sakaguchi S. Naturally arising Foxp3-expressing CD25+CD4+ regulatory T cells in immunological tolerance to self and non-self. Nat Immunol 2005;6:345-52
  • Liu B, Zhao H, Poon MC, et al. Abnormality of CD4+CD25+ regulatory T cells in idiopathic thrombocytopenic purpura. Eur J Haematol 2007;78:139-43
  • Sakakura M, Wada H, Tawara I, et al. Reduced Cd4+Cd25+ T cells in patients with idiopathic thrombocytopenic purpura. Thromb Res 2007;120:187-93
  • Zhang AJ, Qu YY, Zhang BJ, et al. The different effects of indirubin on effector and CD4+CD25+ regulatory T cells in mice: potential implication for the treatment of autoimmune diseases. J Mol Med 2007;85:1263-70
  • Available from: http://www.clinicaltrials.gov; http://www.pdsa.org/itp-clinical-trials/index.html
  • Freedman J, Garvey MB, Elinder G, Blanchette VS. Directions for research in autoimmune thrombocytopenic purpura (ITP). Acta Paediatr Suppl 1998;424:82-4
  • Guo CS, Chu XX, Shi Y, et al. Correction of Th1-dominant cytokine profiles by high-dose dexamethasone in patients with chronic idiopathic thrombocytopenic purpura. J Clin Immunol 2007;27:557-62
  • Cheng Y, Wong RS, Soo YO, et al. Initial treatment of immune thrombocytopenicpurpura with high-dose dexamethasone. N Engl J Med 2003;349:831-6
  • Mazzucconi MG, Fazi P, Bernasconi S, et al. Therapy with high-dosedexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood 2007;109:1401-7
  • Lazarus AH, Crow AR. Mechanism of action of IVIG and anti-D in ITP. Transfus Apher Sci 2003;28:249-55
  • Gaines AR. Disseminated intravascular coagulation associated with acute hemoglobinemia or hemoglobinuria following Rh(0)(D) immune globulin intravenous administration for immune thrombocytopenic purpura. Blood 2005;106:1532-7
  • Ahn YS, Rocha R, Mylvaganam R, et al. Long-term danazol therapy in autoimmune thrombocytopenia: unmaintained remission and age-dependent response in women. Ann Int Med 1989;111:723-9
  • Szczepanik AB, Sikorska A, Slomkowski M, Konopka L. The use of vinca alkaloids in preparation for splenectomy of corticosteroid refractory chronic immune thrombocytopenic purpura patients. Int J Lab Hematol 2007;29:347-51
  • Peng J, Liu C, Liu D, et al. Effects of B7-blocking agent and/or CsA on induction of platelet-specific T-cell anergy in chronic autoimmune thrombocytopenic purpura. Blood 2003;101:2721-6
  • Hou M, Peng J, Shi Y, et al. Mycophenolate mofetil (MMF) for the treatment of steroid-resistant idiopathic thrombocytopenic purpura. Eur J Haematol 2003;70:353-7
  • Zhao YQ, Wang QY, Zhai M, et al. A multi-center clinical trial of recombinant human thrombopoietin in chronic refractory idiopathic thrombocytopenic purpura. Zhonghua Nei Ke Za Zhi (Chin) 2004;43:608-10
  • 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;100:728-30
  • Goodnough LT, Shander AS. Recombinant factor VIIa: safety and efficacy. Curr Opin Hematol 2007;14:504-9

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