Platelet production and platelet destruction: Assessing mechanisms of treatment effect in immune thrombocytopenia

Blood

Volumn 117, Issue 21, 2011, Pages 5723-5732

  Barsam, Sarah J ^a,b   Psaila, Bethan ^a,c   Forestier, Marc ^d   Page, Lemke K ^a   Sloane, Peter A ^a   Geyer, Julia T ^a   Villarica, Glynis O ^a   Ruisi, Mary M ^a   Gernsheimer, Terry B ^e   Beer, Juerg H ^d   Bussel, James B ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

^b UNIVERSITY COLLEGE LONDON HOSPITALS   (United Kingdom)

^c IMPERIAL COLLEGE SCHOOL OF MEDICINE   (United Kingdom)

^d Kantonsspital Baden   (Switzerland)

^e UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DANAZOL; ELTROMBOPAG; FOSTAMATINIB; GLYCOCALICIN; IMMUNOGLOBULIN G; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PREDNISONE; RHESUS D ANTIBODY;

ADOLESCENT; ADULT; AGED; ARTICLE; AUTOIMMUNE THROMBOCYTOPENIA; BONE MARROW; CELL DESTRUCTION; CELL FRACTIONATION; CELL STRUCTURE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; FEMALE; HUMAN; IMMATURE PLATELET FRACTION; INFANT; MALE; MEGAKARYOCYTE; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; SCHOOL CHILD; THERAPY EFFECT; THROMBOCYTE RELEASE REACTION; THROMBOCYTOPOIESIS; TREATMENT RESPONSE;

EID: 79957616016     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2010-11-321398     Document Type: Article

References (50)

1. Ballem PJ, Segal GM, Stratton JR, Gernsheimer T, Adamson JW, Slichter SJ. 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. (Pubitemid 17097513)
2. McMillan R. Autoantobodies and autoantigens in chronic immune thrombocytopenia purpura. Semin Hematol. 2000;37(3):239-248. (Pubitemid 30612902)
3. Nugent D, McMillan R, Nichol JL, et al. Pathogenesis of chronic immune thrombocytopenia: increased platelet destruction and /or decreased platelet production. Br J Haematol. 2009;146(6):585-596.
4. 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):887-894.
5. 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. (Pubitemid 38168649)
6. Bao W, Bussel JB, Heck S, et al. Improved regulatory T cell activity in patients with chronic imune thrombocytopenia treated with thrombopoietic agents. Blood. 2010;116(22):4639-4645.
7. Olsson B, Andersson PO, Jernås M, et al. T-cell-mediated cytotoxicity toward platelets in chronic idiopathic thrombocytopenic purpura. Nat Med. 2003;9(9):1123-1124. (Pubitemid 37173694)
8. Gernsheimer TB. The pathophysiology of ITP revisited: ineffective thrombopoiesis and the emerging role of thrombopoietin receptor agonists in the management of chronic immune thrombocytopenic purpura. Hematology Am Soc Hematol Educ Program. 2008:219-226.
9. 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(11):1630-1638. (Pubitemid 30367902)
10. Portielje JE,Westendorp RG, Kluin-Nelemans HC, Brand A. Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood. 2001;97(9):2549-2554.
11. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115(2):168-186.
12. 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(2):232-239. (Pubitemid 38534976)
13. Imbach P, Barandun S, d'Apuzzo V, et al. Highdose intravenous gammaglobulin for idiopathic thrombocytopenic purpura in childhood. Lancet. 1981;1(8232):1228-1231.
14. Fehr J, Hofmann V, Kappeler U. Transient reversal of thrombocytopenia in idiopathic thrombocytopenic purpura by high-dose intravenous gamma globulin. N Engl J Med. 1982;306(21):1254-1258. (Pubitemid 12100206)
15. Michel M, Kreidel F, Chapman ES, Zelmanovic D, Bussel JB. Prognostic relevance of large-platelet counts in patients with immune thrombocytopenic purpura. Haematologica. 2005;90(12):1715-1716. (Pubitemid 41815006)
16. Samuelsson A, Towers TL, Ravetch JV. Antiinflammatory activity of IVIG mediated through the inhibitory Fc receptor. Science. 2001;291(5503):484-486. (Pubitemid 32097072)
17. Nikolova KA, Tchorbanov AI, Djoumerska-Alexieva IK, Nikolova M, Vassilev TL. Intravenous immunoglobulin up-regulates the expression of the inhibitory FcgammaIIB receptor on B cells. Immunol Cell Biol. 2009;87(7):529-533.
18. Chow L, Aslam R, Speck ER, et al. A murine model of severe immune thrombocytopenia is induced by antibody and CD8+ T cell mediated responses that are differentially sensitive to therapy. Blood. 2010;115(21):1247-1253.
19. Lazarus AH, Crow AR. Mechanism of action of IVIG and anti-D in ITP. Transfus Apheresis Sci. 2003;28(3):249-255. (Pubitemid 36506499)
20. Cooper N, Heddle NM, Haas M, et al. Intravenous anti-D and IV immunoglobulin achieve acute platelet increases by different mechanisms: modulation of cytokine and platelet responses to intravenous anti-D by FcgammaRIIa and FcgammaRIIIa polymorphisms. Br J Haematol. 2004; 124(4):511-518. (Pubitemid 38230598)
21. Evangelista ML, Amadori S, Stasi R. Biologic aspects of thrombopoietin and the development of novel thrombopoietic agents for clinical use. Curr Drug Discov Technol. 2007;4(3):162-173. (Pubitemid 350171818)
22. Kuter DJ. New thrombopoietic growth factors. Blood. 2007;109(11):4607- 4616. (Pubitemid 46827750)
23. Boruchov DM, Gururangan S, Driscoll MC, Bussel JB. Multi-agent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura. Blood. 2007;110(10):3526-3531. (Pubitemid 350159616)
24. Cines DB, McKenzie SE, Siegel DL. Mechanisms of action of therapeutics in idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol. 2003; 25(suppl 1):S52-S56. (Pubitemid 37517920)
25. Gernsheimer T, Stratton J, Ballem P, Slichter S. Mechanisms of response to treatment in autoimmune thrombocytopenic purpura. N Engl J Med. 1989;320(15):974-980. (Pubitemid 19101615)
26. Fabris F, Scandellari R, Ruzzon E, Luigia Randi M, Luzzatto G, Girolami A. Platelet-associated autoantibodies as detected by a solid-phase modified antigen capture ELISA test (MACE) are a useful prognostic factor in idiopathic thrombocytopenic purpura. Blood. 2004;103(12):4562-4564. (Pubitemid 38745983)
27. Berchtold P, Wenger M. Autoantibodies against platelet glycoproteins in autoimmune thrombocytopenic purpura: their clinical significance and response to treatment. Blood. 1993;81(5):1246-1250. (Pubitemid 23067361)
28. McMillan R. Antiplatelet antibodies in chronic adult immune thrombocytopenia: assays and epitopes. J Pediatr Hematol Oncol. 2003;25(1):57-61.
29. Stoll D, Cines DB, Aster RH, Murphy S. Platelet kinetics in patients with idiopathic thrombocytopenic purpura and moderate thrombocytopenia. Blood. 1985;65(3):584-588. (Pubitemid 15131466)
30. Ault K, Knowles C. In vivo biotinylation demonstrates that reticulated platelets are the youngest platelets in circulation. Exp Hematol. 1995;23(9):996-1034.
31. Houwerzijl EJ, Blom NR, van der Want JJ, et al. Ultrastructural study shows morphological features of apoptosis and paraapoptosis in megakaryocytes from patients with idiopathic thrombocytopenic purpura. Blood. 2004;103(2):500-506. (Pubitemid 38140080)
32. Hirsch EO, Favre-Gilly J, Dameshek W. Thrombopathic thrombocytopenia: successful transfusion of blood platelets. Blood. 1950;5(6):568-580.
33. Beer JH, Buchi L, Steiner B. Glycocalicin: a new assay-the normal plasma levels and its potential usefulness in selected diseases. Blood. 1994;83(3):691-702. (Pubitemid 24043729)
34. Kurata Y, Hayahsi S, Kiyoi T, et al. Diagnostic value of tests for reticulated platelets, plasma glycocalicin, and thrombopoietin levels for discriminating between hyperdestructive and hypoplastic thrombocytopenia. Am J Clin Pathol. 2001;115(5):656-664. (Pubitemid 33051531)
35. Steinberg MH, Kelton JG, Coller BS. Plasma glycocalicin: an aid in the classification of thrombocytopenic disorders. N Engl J Med. 1987;317(17):1037- 1042. (Pubitemid 17146895)
36. Kienast J, Schmitz G. Flow cytometric analysis of thiazole orange uptake by platelets: a diagnostic aid in the evaluation of thrombocytopenic disorders. Blood. 1990;75(1):116-121. (Pubitemid 20031498)
37. Briggs C, Kunka S, Hart D, Ognuni S, Machin SJ. Assessment of immature platelet fraction (IPF) in peripheral thrombocytopenia. Br J Haematol. 2004;126:(1)93-99. (Pubitemid 38859488)
38. Walters J, Garrity P. Performance evaluation of the Sysmex XE-2100 Hematology Analyzer. Lab Hematol. 2000;6:83-92.
39. Pons I, Monteagudo M, Lucchetti G, et al. Correlation between immature platelet fraction and reticulated platelets: usefulness in the etiology diagnosis of thrombocytopenia. Eur J Haematol. 2010;85(2):158-163.
40. Nomura S, Dan K, Hotta T, Fujimura K, Ikeda Y. Effects of pegylated recombinant human megakaryocyte growth and development factor in patients with idiopathic thrombocytopenic purpura. Blood. 2002;100(2):728-730. (Pubitemid 34761148)
41. 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. (Pubitemid 350190754)
42. 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(9664):641-648.
43. Clarkson S, Bussel JB, Kimberley RP, Valinsky JE, Nachman RL, Unkeless JC. Treatment of refractory immune thrombocytopenic purpura with an anti-Fcgamma receptor antibody. N Engl J Med. 1986;314(19):1236-1239. (Pubitemid 16106411)
44. Nakar CT, Bussel JB. 3G8 and GMA161, anti Fcgamma receptor III inhibitory monoclonal antibodies in the treatment of chronic refractory ITP (summary of two pilot studies) [abstract]. Blood (ASH Annual Meeting Abstracts). 2009;114:2404.
45. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113(11):2386-2393.
46. Abe Y, Wada H, Tomatsu H, et al. A simple technique to determine thrombopoiesis level using immature platelet fraction (IPF). Thromb Res. 2006;118(4):463-469. (Pubitemid 44292797)
47. Cannavo I, Ferrero-Vacher C. Assessment of an immature platelet fraction (IPF%) in the diagnosis of thrombocytopenia. Ann Biol Clin (Paris). 2010;68(4):415-420.
48. Jung H, Jeon HK, Kim HJ, Kim SH. Immature platelet fraction: establishment of a reference interval and diagnostic measure of thrombopoiesis. Korean J Lab Med. 2010;30(5):451-459.
49. Cho YG, Lee JH, Kim DS, Lee HS, Choi SI. Clinical usefulness of the simple technique to diagnose thrombocytopenia using immature platelet fraction. Korean J Lab Med. 2007;27(1):1-6.
50. Chatterji AK, Lynch EC, Garg SK, Amorosi EL, Karpatkin S. Circulating large platelets. N Engl J Med. 1971;284(25):1440-1441.