Practice guidelines for diagnosis and therapy of essential thrombocytaemia and thrombocytaemia associated with other myeloproliferative diseases

Česká verzia

Authors: M. Penka ¹; J. Schwarz ²; R. Pytlík ³; M. Doubek ⁴; Y. Brychtová ⁴; P. Ďulíček ⁵
Authors place of work: Oddělení klinické hematologie FN Brno, pracoviště Bohunice, přednosta prof. MUDr. Miroslav Penka, CSc. ¹; Ústav hematologie a krevní transfuze, Praha, ředitel prof. MUDr. Pavel Klener, DrSc. ²; I. interní klinika 1. lékařské fakulty UK a VFN, Praha, přednosta prof. MUDr. Pavel Klener, DrSc. ³; Interní hematoonkologická klinika Lékařské fakulty MU a FN Brno, pracoviště Bohunice, přednosta prof. MUDr. Jiří Vorlíček, CSc. ⁴; Oddělení klinické hematologie – II. interní klinika Lékařské fakulty UK a FN, Hradec Králové, přednosta prof. MUDr. Jaroslav Malý, CSc. ⁵
Published in the journal: Vnitř Lék 2005; 51(6): 741-751
Category: Guidelines

Summary

Essential thrombocytaemia (ET) is not a frequent disease (incidence rate is about 0.1–1.5 cases per 100,000 persons) but as new approaches and possibilities advancing the standard care but contrariwise requiring responsible and systematic approach occur both in relationship with diagnosis and with treatment, it is necessary to deal appropriately with this problem. There is a variety of aspects, which evoke controversy within the scope of diagnosis and differential diagnosis – the most important thing is whether the histological examination of bone marrow is required or not and how to organise care of patients. All this relates not only to essential thrombocytopenia but also to myeloproliferative diseases associated with other myeloproliferative diseases at all (MPO-T). Enclosed recommended practice guidelines could help to serve as a guide in problems given without limiting the space for disputation.

Key words:
myeloproliferation – (essential) thrombocytaemia – megakaryopoesis – anagrelide – interferon – hydroxyurea

Zdroje

1. Andrieux J, Demory JL, Dupriez B et al. Dysregulation and overexpression of HMGA2 in myelofibrosis with myeloid metaplasia. Genes Chormosomes Cancer 2004; 39: 82–87.

2. Barbui T, Barosi G, Brossi A et al. Practice guidelines for the therapy of essential thrombocythemia. A statement from the Italian Society of Hematology, the Italian Society of Experimental Hematology and Italian Group for Bone Marrow Transplantation. Haematologica 2004; 89: 215–232.

3. Castro-Malaspina H, Gay RE, Jhanwar SC et al. Characteristics of bonemarrow fibroblast colony-forming cells (CFU-F) and their progeny in patients with myeloproliferative disorders. Blood 1982; 59: 1046–1054.

4. Cortelazzo S, Finazzi G, Ruggeri M et al. Hydoxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med 1995; 332: 113.

5. Doubek M, Brychtová Y, Doubek R et al. Anagrelide in pregnancy: report of a case of essential thrombocythaemia. Ann Hematol 2004; 83: 726–727.

6. Elliott MA, Tefferi A Interferon-α therapy in polycythemia pera and essential phrombocythemia. Semin Thromb Hemost 1997; 23: 463.

7. Folman CC, Ooms M, Kuenen B et al. The role of thrombopoetin in post–operative thrombocytosis. Br J Haematol 2001; 114: 126–133.

8. Fruchtmann SM Treatment paradigms in the management of myeloproliferative disorders. Semin Hematol 2004; suppl. 3: 18–42.

9. Georgii A, Buhr T, Buesche G et al. Classification and staging of Ph–negative myeloproliferative disorders by histopathology from bone marrow biopsies. Leukemia Lymphoma 1996; 22( supl 1): 15–29.

10. Green A, Campbell P, Buck G et al. The Medical Research Council PT1 trial in essentials thrombocythemia. Blood 2004; 104: 5a–6a.

11. Harrison CN, Gale RE, Machin SK et al. A large proportion of patients with a diagnosis of Essential Thrombocythemia do not have a clonal disorder and may be at lower risk of thombotic complications. Blood 1999; 93: 417–424.

12. Horikawa Y, Matsumura I, Hashimoto K et al. Markedly reduced expression of platelet c-mpl receptor in essential thrombocythemia. Blood 1997; 90: 4031–4038.

13. Jaffe ES, Harris NL, Stein H et al. (Eds.): World health organization classification of tumours. Pathology and genetics of tumours of haematopoietic and lymphoid tissues. Lyon: IARC Press 2001.

14. Kralovics R, Buser AS, Teo SS et al. Comparison of molecular markers in a cohort of patients with chronic myeloproliferative disorders. Blood 2003; 102: 1869–1871.

15. Lengfelder E, Hochhaus A, Kronawitter U et al. Should a platelet limit of 600 x 109/l be used as a diagnostic criterion in essential thrombocythaemia? An analysis of the natural course including early stages. Br J Haematol 1998; 100: 15–23.

16. Lofvenberg E, Wahlin A Management of Polycythemia vera, Essential Thrombocythemia an myelofibrosis with hydroxyurea. Eur J Haematol 1998; 41: 375.

17. Liu E, Jelinek J, Pastore YD et al. Discrimation of polycythemias and thrombocytoses by novel, simple, accurate clonality assays and comparison with PRV-1 expresion and BFU-E response to erythrocytosis. Blood 2003; 101: 3294–3301.

18. Martyre MC, Le Bousse–Kerdiles MC, Romquin N et al. Elevated levels of basic fibroblast growth factor in megakaryocytes and platelets from patients with idiopathic myelofibrosis. Br J Haematol 1997; 1997: 441–448.

19. Mazur EM, Rosmarin AG, Sohl PA et al. Analysis of the mechanism of anagrelid-induced thrombocytopenia in humans. Blood 1992; 79: 1931.

20. Messa RA, Tefferi A, Jacobsen SJ et al. Population-based incidence and survival figures in Essential Thrombocythemia and agnogenicmyeloid meta-plasia: an Olmsted County study. Am J Hematol 1999; 61: 10–15.

21. Michiels JJ Diagnostic criteria of the myeloproliferative disorders (MPD): essential thrombocythaemia, polycythaemia vera and chronic megakaryocytic granulocytic metaplasia. Netherlands J Med 1997; 51: 57–64.

22. Michiels JJ, Juvonen E Proposal for revised diagnostic criteria of essential thrombocythemia and polycythemia vera by the Thrombocythemia Vera Study Group. Semin Thromb Hemost 1997; 23: 339–347.

23. Michiels JJ, Kutti J, Stark P et al. Diagnosis, pathogenesis and treatment of the myeloproliferative disorders essential thrombocythemia, polycythemia vera and essential megakaryocytic granulocytic

metaplasia and myelofibrosis. Neth J Med 1999; 54: 46–62.

24. Michiels JJ, Barbui T, Finazzi G et al. Diagnosis and treatment of Polycythemia Verand possible future study designsof the PVSG. Leuk Lymphoma 2000, 36: 239–253.

25. Michiels JJ, Thiele J Clinical and pathological criteria for thed diagnosis of essential thrombocythemia, polycythemia vera and idiopathic myelofibrosis (agnogenic myeloid metaplasia). Int J Hematol 2002; 76: 133–145.

26. Murphy S, Peterson P, Iland H et al. Experience of the Polycythemia Vera Study Group with essential thrombocythemia: a final report on diagnostic criteria, survival, and leukemic transition by treatment. Semin Hematol 1997; 34: 29–39.

27. Oehler L, Jaeger E, Eser A et al. Imatinib mesylate inhibits autonomous erythropoesis in patients with polycythemia vera in vitro. Blood 2003; 102: 2240–2242.

28. Penka M, Buliková A, Matýšková M et al. Hematologie I. Neonkologická hematologie. Praha: Grada 2001.

29. Petrides PE Anagrelide: decade of clinical experinces with its use for the treatment of primary thrombocythemia. Expert Opin Pharmacother 2004; 5: 1781–1798.

30. Petitt RM, Silverstein MN, Petrone ME Anagrelide for controle of thrombocythemia in polycythemia and other myeloproliferative disorders. Semin Hematol 1997; 34: 51.

31. Prchal JF, Axelrad AA Letter: bone marrow responses in polycythemia vera. N Engl J Med 1974; 290: 1382.

32. Pytlík R, Cmunt E, Kleibl Z et al. Úloha anagrelidu v léčbě esenciální trombocytémie. Trans Hematol dnes 2004; 10: 154–160.

33. Rameshwar P, Denny TN, Stein D et al. Monocytes adhesion in patients with bone marrow fibrosis is required for the production of fibrogenic cytokines. Potential role for interleukin-1 and TGF-β. J Immunol 1994; 153: 2819–2830.

34. Reeder TL, Bailey RJ, Dewald GW et al. Both B and T lymphocytes may be clonally involved in myelofibrosis with myeloid metaplasia. Blood 2003; 101: 1981–1983.

35. Roder S, Steimle C, Meinhardt G et al. STAT3 is constitutively active in some patients with polycythemia rubra vera. Exp Hematol 2001; 29: 694–702.

36. Sacchi S, Vinci G, Gugliotta L et al. Diagnosis of essential thrombocythemia at platelet counts between 400 and 600 × 109/l. Haematologica 2000; 85: 492–495.

37. Schafer AI Thrombocytosis. N Engl J Med 2004; 350: 1211–1219.

38. Schwarz J, Penka M Trombocytózy a trombocytemie. Vnitř Lék 2005; 51: v tisku.

39. Skoda RC. Chronic myeloproliferative disorders: molecular markers and pathogenesis. Hematol J 2004; 5: S122–S125.

40. Spinelli O, Rota B, Finazzi G et al. Quantitative analysis of PRV-l gene expression in chronic myeloproliferative disorders: positive correlation with polycytemia vera diagnosis and leukocyte

alkaline phospahtase expression. Blood 2002; 100: 3145a.

41. Spivak JL Diagnosis of the myeloproliferative disorders: resolving phenotypic mimicry. Semin Hematol 2003; 40: 1–5.

42. Thiele J, Kvasnicka HM, Diehl V et al. Clinickopathological diagnosis and differential criteria of thrombocythemias in various myeloproliferative disorders by histopathology, histochemistry and immunostaining from bone marrow biopsies. Leukemia Lymphoma 1999; 33: 207–218.

43. Thiele J, Kvasnicka HM, Graeff AS et al. Follow-up examinations including sequential bone marrow biopsies in essential thrombocythemia (ET): A retrospective clinicopathological study of 120 patients. Am J Hematol 2002; 70: 283–291.

44. Thiele J, Kvasnicka HM, Werden C et al. Idiopathic primary osteo-myelofibrosis: a clinico-pathological study on 208 patients with special emphasis on evolution of disease features, differentiation from essential thrombocythemia and variables of prognostic impact. Leukemia Lymphoma 1996; 22: 303–317.

45. Thiele J, Kvasnicka HM, Zankovich R et al. Clinical and morphological criteria for the diagnosis of prefibrotic idiopathic (primary) myelofibrosis. Ann Hematol 2001; 80: 160–165.

46. Tefferi A, Solberg LA, Silverstein MN A clinical update on Polycythemia Vera and Essential Thrombocythemia. Am J Med 2000; 109: 141–149.

47. Weinfeld A, Swolin B, Westin J Acute leukaemia after hydroxyurea therapy in Polycythaemia Vera and allied disorders: prospective study of efficacy and leukemogenicity with therapeutic implications. Eur J Haematol 1994; 52: 134–139.

48. Wright CA, Tefferi A A single institutional experience with 43 pregnancies in Essential Thrombocythemia. Eur J Haematol 2001; 66: 152.