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What is the standard treatment in essential thrombocythemia

  • Current Concepts of the Myeloproliferative Diseases
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Abstract

The treatment of patients with essential thrombocythemia (ET) should be based primarily on the expected risk of major thrombotic complications. Although the specific values chosen for separating different risk categories are in part arbitrary, the following recomendations can be made. Young asymptomatic subjects with platelet counts below 1,500×109/L are at lower risk and can be followed untreated. However, it should be emphasized that thrombotic events can also occur in a small percentage of these lower-risk cases. Low-dose aspirin (100–300 mg/day) should be given to patients with symptoms of microvascular occlusion, such as erythromelalgia or transient neurological attacks, and avoided in those with bleeding manifestations. The risk/benefit of low-dose aspirin in the primary prevention of thrombosis in asymptomatic patients remains uncertain. For high-risk patients (age >60 years, or platelet count >1,500×109/L, or previous thrombosis), hydroxyurea, plus aspirin in the case of thrombosis, is the treatment of choice because its efficacy in preventing thrombotic complications has been proven in a randomized clinical trial. However, the possible long-term leukemogenicity of this drug, as well as that of other effective cytoreductive agents such as busulphan and pipobroman, remains a major concern. Anagrelide and interferon could overcome this worry but their efficacy has been hitherto demonstrated only in lowering the platelet count. Controlled clinical studies showing a benefit in preventing thrombotic and hemorrhagic complications are urgently needed.

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References

  1. Fialkow PJ, Faguet GB, Jacobson RJ, et al. Evidence that essential thrombocythemia is a clonal disorder with origin in a multipotent cell.Blood. 1981;58:916–919.

    PubMed  CAS  Google Scholar 

  2. Harrison CN, Gale RE, Machin SJ, Linch DC. A large proportion of patients with a diagnosis of essential thrombocythemia do not have a clonal disorders and may be at lower risk of thrombotic complications.Blood. 1999;93:417–424.

    PubMed  CAS  Google Scholar 

  3. Murphy S. Therapeutic dilemmas: balancing the risk of bleeding, thrombosis, and leukemic transformation in myeloproliferative disorders (MPD).Thromb Haemostas. 1997;78:622–626.

    CAS  Google Scholar 

  4. Murphy S, Iland HJ. Thrombocytosis. In: Thrombosis and Hemorrhage (1st ed.) Loscalzo J and Schafer AL eds. Williams and Wilkins, Baltimore, 1994. pp. 597–612.

    Google Scholar 

  5. Pearson TC. Primary Thrombocythaemia: diagnosis and management.Br J Haematol. 1991;78:145–148.

    Article  PubMed  CAS  Google Scholar 

  6. Rozman C, Giralt M, Feliu E, et al. Life expectancy of patients with chronic nonleukemic myeloproliferative disorders.Cancer. 1991;67:2658–2663.

    Article  PubMed  CAS  Google Scholar 

  7. Murphy S, Iland HJ, Rosenthal D, et al. Essential thrombocythemia: an interim report from the Polycythemia Vera Study Group.Semin Hematol. 1986;23:177–182.

    PubMed  CAS  Google Scholar 

  8. 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.

    PubMed  CAS  Google Scholar 

  9. Dudley JM, Messinezy M, Eridani S, et al. Primary thrombocythemia: diagnostic criteria and a simple scoring system for positive diagnosis.Br J Haematol. 1989;71:331–335.

    PubMed  CAS  Google Scholar 

  10. Kutti J, Wadenvik H. Diagnostic and differential criteria of essential thrombocyhemia and reactive thrombocytosis.Leuk Lymphoma. 1996;22:41–45.

    PubMed  Google Scholar 

  11. Sawyer BM, Westwood NB, Pearson TC. Circulating megakaryocyte progenitor cells in patients with primary thrombocythemia and reactive thrombocytosis: results using a serum-deprived culture assay and positive detection technique.Eur J Hematol. 1994;53:108–113.

    Article  CAS  Google Scholar 

  12. Lengfelder E, Hochhaus A, Kronawitter U, et al. Should a platelet limit of 600×109/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,

    Article  PubMed  CAS  Google Scholar 

  13. Landolfi R, Rocca B, Patrono C. Bleeding and thrombosis in myeloproliferative disorders: mechanisms and treatment.Crit Review Oncol Hematol. 1995;20:203–222.

    Article  CAS  Google Scholar 

  14. Koudstaal PJ, Koudstaal A. Neurologic and visual symptoms in essential thrombocythemia: efficacy of low-dose aspirin.Semin Thromb Haemostas. 1997;23:365–370.

    Article  CAS  Google Scholar 

  15. Van Genderen PJJ, Michiels JJ. Erythromelalgia: a patognomonic microvascular thrombotic complication in essential thrombocythemia and polycythemia vera.Semin Thromb Haemostas. 1997;23:357–364.

    Article  Google Scholar 

  16. De Stefano V, Teofili L, Leone G, et al. Spontaneous erythroid colony formation as the clue to an underlying myeloproliferative disorder in patients with Budd-Chiari syndrome or portal vein thrombosis.Semin Thromb Haemostas. 1997; 23:411–418.

    Article  Google Scholar 

  17. Barbui T, Cortelazzo S, Viero P, et al. Thrombohaemorrhagic complications in 101 cases of myeloproliferative disorders: Relationship to platelet number and function.Eur J Cancer Clin Oncol. 1983;19:1593–1599.

    Article  PubMed  CAS  Google Scholar 

  18. Bellucci S, Janvier M, Tobelem G, et al. Essential thrombocythemia: Clinical evolutionary and biological data.Cancer. 1986;58:2440–2447.

    Article  PubMed  CAS  Google Scholar 

  19. Hehlmann R, Jahn M, Baumann B, et al. Essential thrombocythemia: Clinical characteristics and course of 61 cases.Cancer. 1988;61:2487–2496.

    Article  PubMed  CAS  Google Scholar 

  20. Fenaux P, Simon M, Caulier MT, et al. Clinical course of essential thrombocythemia in 147 cases.Cancer. 1990;66:549–556.

    Article  PubMed  CAS  Google Scholar 

  21. Colombi M, Radaelli F, Zocchi L, et al. Thrombotic and hemorrhagic complications in essential thrombocytemia.Cancer. 1991;67:29269–2930.

    Article  Google Scholar 

  22. Finazzi G, Budde U, Michiels JJ. Bleeding time and platelet function in essential thrombocythemia and other myeloproliferative syndromes.Leuk Lymphoma. 1996;22(suppl 1):71–78.

    PubMed  Google Scholar 

  23. Rinder HM, Schuster JE, Rinder CS, et al. Correlation of thrombosis with increased platelet turnover in thrombocytosis.Blood. 1998;91:1288–1293.

    PubMed  CAS  Google Scholar 

  24. Budde U, Scharf RE, Franke P, et al. Elevated platelet count as a cause of abnormal von Willebrand factor multimer distribution in plasma.Blood. 1993;82:1749–1757.

    PubMed  CAS  Google Scholar 

  25. Van Genderen PJJ, Michiels JJ, van der Poel-van de Luytgaarde SCPAM, et al. Acquired von Willebrand disease as a cause of recurrent mucocutaneous bleeding in primary thrombocythemia: relationship with platelet count.Ann Hematol. 1994;69:81–84.

    Article  PubMed  Google Scholar 

  26. Cortelazzo S, Viero P, Finazzi G, et al. Incidence and risk factors for thrombotic complications in a historical cohort of 100 patients with essential thrombocythemia.J Clin Oncol. 1990;8:556–562.

    PubMed  CAS  Google Scholar 

  27. Randi ML, Fabris F, Rossi C, et al. Sex and age as prognostic factors in essential thrombocythemia.Haematologica. 1992;77:402–404.

    PubMed  CAS  Google Scholar 

  28. McIntyre K, Hoagland H, Silverstein M, et al. Essential thrombocythemia in young adults.Mayo Clin Proc. 1991;66: 149–154.

    PubMed  CAS  Google Scholar 

  29. Mitus AJ, Barbui T, Shulman LN, et al. Hemostatic complications in young patients with essential thrombocythemia.Am J Med. 1990;88:371–375.

    Article  PubMed  CAS  Google Scholar 

  30. Kutti J. The management of thrombocytosis.Eur J Haematol. 1990;44:81–88.

    Article  PubMed  CAS  Google Scholar 

  31. Schafer AI. Essential thrombocythemia.Progr Hemostas Thromb. 1991;10:69–96.

    CAS  Google Scholar 

  32. Tefferi A, Silverstein MN, Hoagland HC. Primary thrombocythemia.Semin Oncol. 1995;22:334–340.

    PubMed  CAS  Google Scholar 

  33. Barbui T, Finazzi G, Dupuy E, et al. Treatment strategies in essential thrombocythemia.Leuk Lymphoma. 1996;22(suppl 1): 149–160.

    Article  PubMed  Google Scholar 

  34. Griesshammer M, Bangerter M, vanVliet HHDM, et al. Aspirin in essential thrombocythemia: status quo and quo vadis.Semin Thromb Haemostas. 1997;23:371–378.

    Article  CAS  Google Scholar 

  35. Antiplatelet Trialists’ Collaboration. Collaborative overview of randomised trials of antiplatelet therapy-I. Prevention of death, myocardial infarction, and stroke by prolonged antiplatelet therapy in various categories of patients.Br Med J. 1994;308: 81–106.

    Google Scholar 

  36. Kelly JP, Kaufman DW, Jurgelon JM, et al. Risk of aspirin-associated major upper-gastrointestinal bleeding with enteric-coated or buffered product.Lancet. 1996;348:1413–1416.

    Article  PubMed  CAS  Google Scholar 

  37. Pagliaro P, Arrigoni L, Muggiasca ML, et al. Primary thrombocyhthemia and pregnancy. Treatment and outcome in fifteen cases.Am J Hematol. 1996;53:6–10.

    Article  PubMed  CAS  Google Scholar 

  38. Griesshammer M, Heimpel H, Pearson TC. Essential thrombocythaemia and pregnancy.Leuk Lymphoma. 1996;22(suppl 1): 57–63.

    Article  PubMed  Google Scholar 

  39. Beressi AH, Tefferi A, Silverstein MN, et al. Outcome analysis of 34 pregnancies in women with essential thrombocythemia.Arch Intern Med. 1995;155:1217–1222.

    Article  PubMed  CAS  Google Scholar 

  40. Shpilberg O, Shimon I, Sofer O, et al. Transient normal platelet count and decreased requirement for interferon during pregnancy in essential thrombocythemia.Br J Haematol. 1996;92:491–493.

    Article  PubMed  CAS  Google Scholar 

  41. Lofvenberg E, Walhlin A. Management of polycythaemia vera, essential thrombocythaemia and myelofibrosis with hydroxyurea.Eur J Haematol. 1988;41:375–381.

    Article  PubMed  CAS  Google Scholar 

  42. Cortelazzo S, Finazzi G, Ruggeri M, et al. Hydroxyurea in the treatment of patients with essential thrombocythemia at high risk of thrombosis: a prospective randomized trial.N Engl J Med. 1995;332:1132–1136.

    Article  PubMed  CAS  Google Scholar 

  43. Daoud MS, Gibson LE, Pittelkow MR. Hydroxyurea dermopathy: a unique lichenoid eruption complicating long-term therapy with hydroxyurea.J Am Acad Dermatol. 1997;36:178–182.

    Article  PubMed  CAS  Google Scholar 

  44. Best P, Daoud MS, Pittelkow MR et al. Hydroxyurea-induced leg ulceration in 14 patients.Ann Intern Med. 1998;128:29–32.

    PubMed  CAS  Google Scholar 

  45. Nand S, Stock W, Godwin J, et al. Leukemogenic risk of hydroxyurea therapy in polycythemia vera, essential thrombocythemia and myeloid metaplasia with myelofibrosis.Am J Hematol. 1996;52:42–46.

    Article  PubMed  CAS  Google Scholar 

  46. Lfvenberg E, Nordenson I, Walhlin A. Cytogenetic abnormalities and leukemic transformation in hydroxyurea-treated patients with Philadelphia chromosome negative chronic myeloproliferative disease.Cancer Genet Cytogenet. 1990;49:57–67.

    Article  Google Scholar 

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

    Article  Google Scholar 

  48. Sterkers Y, Preudhomme C, Lai J-L, et al. Acute myeloid leukemia and myelodyslastic syndromes following essential thrombocythemia treated with hydroxyurea: high proportion of cases with 17p deletion.Blood. 1988;91:616–622.

    Google Scholar 

  49. Shibata K, Shimamoto Y, Suga K, et al. Essential Thrombocythemia terminating in acute leukemia with minimal myeloid differentiation. A brief review of recent literature.Acta Haematol. 1994;91:84–88.

    Article  PubMed  CAS  Google Scholar 

  50. Finazzi G, Ruggeri M, Rodeghiero F, Barbui T. Second malignancies in patients with essential thrombocythaemia treated with busulfan and hydroxyurea: long-term follow-up of a randomized clinical trial.Br J Haematol. 2000;110:577–583.

    Article  PubMed  CAS  Google Scholar 

  51. Van de Pette JEW, Prochazka AV, Pearson TC, et al. Primary thrombocythemia treated with busulfan.Br J Haematol. 1986; 62:229–237.

    Article  PubMed  Google Scholar 

  52. Berk P, Goldberg J, Donovan P, et al. Therapeutic recommendations in polycythemia vera based on polycythemia vera study group protocols.Semin Hematol. 1986;23:132–143.

    PubMed  CAS  Google Scholar 

  53. Haanen C, Mathe G for the EORTC. Treatment of polycythaemia vera by radioactive phosphorus or busulphan.Br J Cancer. 1981;44:75–78.

    Google Scholar 

  54. Lengfelder E, Griesshammer M, Hehlmann R. Interferon-alpha in the treatment of essential thrombocythemia.Leuk Lymphoma. 1996;22(suppl 1):135–142.

    Article  PubMed  Google Scholar 

  55. Tefferi A, Elliot MA, Solberg LA Jr, et al. New drugs in essential thrombocythemia and polycythemia vera.Blood Reviews. 1997;11:1–7.

    Article  PubMed  CAS  Google Scholar 

  56. Tefferi A, Silverstein MN, Petitt RM, et al. Anagrelide as a new platelet-lowering agent in essential thrombocythemia: mechanism of action, efficacy, toxicity, current indication.Semin Thromb Hemostas. 1997;23:379–384.

    Article  CAS  Google Scholar 

  57. Petitt RM, Silverstein MN, Petrone ME. Anagrelide for control of thrombocythemia in polycythemia and other myeloproliferative disorders.Semi Hematol. 1997;34:51–54.

    CAS  Google Scholar 

  58. Anagrelide Study Group. Anagrelide, a therapy for thrombocythemic states: experience in 577 patients.Am J Med. 1992; 92:69–76.

    Article  Google Scholar 

  59. Storen EC, Tefferi A. Long-term use of anagrelide in young patients with essential thrombocythemia.Blood. 2001;97:863–866.

    Article  PubMed  CAS  Google Scholar 

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  1. Divisione di Ematologia, Ospedali Riuniti, Bergamo, Italy

    Tiziano Barbui

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Barbui, T. What is the standard treatment in essential thrombocythemia. Int J Hematol 76 (Suppl 2), 311–317 (2002). https://doi.org/10.1007/BF03165140

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