Skip to main content

Thank you for visiting You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

Acute Leukemias

Risk stratification for survival and leukemic transformation in essential thrombocythemia: a single institutional study of 605 patients


Unlike the case with thrombosis, prognostic models for survival and leukemic transformation (LT) in essential thrombocythemia (ET) are not available. Among 605 patients with ET seen at our institution and followed for a median of 84 months, 155 died and LT was documented in 20 patients (3.3%). In a multivariable analysis, hemoglobin level below normal (females<120 g/l; males<135 g/l) was identified as an independent risk factor for both inferior survival and LT. Additional risk factors for survival included age 60 years, leukocyte count15 × 109/l, smoking, diabetes mellitus and thrombosis. For LT, platelet count1000 × 109/l but not cytoreductive therapy was flagged as an additional independent risk factor. In fact, four of the 20 patients (20%) with LT were untreated previously. We used the above information to construct prognostic models that effectively discriminated among low-, intermediate- and high-risk groups with respective median survivals of 278, 200 and 111 months (P<0.0001), and LT rates of 0.4, 4.8 and 6.5% (P=0.0009) respectively. Presence of JAK2V617F did not impact either survival or LT and mutational frequency was similar among the different risk groups.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Rent or buy this article

Get just this article for as long as you need it


Prices may be subject to local taxes which are calculated during checkout

Figure 1


  1. Rozman C, Giralt M, Feliu E, Rubio D, Cortes MT . Life expectancy of patients with chronic nonleukemic myeloproliferative disorders. Cancer 1991; 67: 2658–2663.

    Article  CAS  Google Scholar 

  2. Mesa RA, Silverstein MN, Jacobsen SJ, Wollan PC, Tefferi A . Population-based incidence and survival figures in essential thrombocythemia and agnogenic myeloid metaplasia: an Olmsted County study, 1976–1995. Am J Hematol 1999; 61: 10–15.

    Article  CAS  Google Scholar 

  3. Cervantes F, Tassies D, Salgado C, Rovira M, Pereira A, Rozman C . Acute transformation in nonleukemic chronic myeloproliferative disorders: actuarial probability and main characteristics in a series of 218 patients. Acta Haematol 1991; 85: 124–127.

    Article  CAS  Google Scholar 

  4. Barbui T, Barosi G, Grossi A, Gugliotta L, Liberato LN, Marchetti M 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 the Italian Group for Bone Marrow Transplantation. Haematologica 2004; 89: 215–232.

    CAS  PubMed  Google Scholar 

  5. Harrison CN . Essential thrombocythaemia: challenges and evidence-based management. Br J Haematol 2005; 130: 153–165.

    Article  Google Scholar 

  6. Elliott MA, Tefferi A . Thrombosis and haemorrhage in polycythaemia vera and essential thrombocythaemia. Br J Haematol 2005; 128: 275–290.

    Article  CAS  Google Scholar 

  7. Cortelazzo S, Finazzi G, Ruggeri M, Vestri O, Galli M, Rodeghiero F et al. Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med 1995; 332: 1132–1136.

    Article  CAS  Google Scholar 

  8. Harrison CN, Campbell PJ, Buck G, Wheatley K, East CL, Bareford D et al. Hydroxyurea compared with anagrelide in high-risk essential thrombocythemia. N Engl J Med 2005; 353: 33–45.

    Article  CAS  Google Scholar 

  9. Passamonti F, Rumi E, Pungolino E, Malabarba L, Bertazzoni P, Valentini M et al. Life expectancy and prognostic factors for survival in patients with polycythemia vera and essential thrombocythemia. Am J Med 2004; 117: 755–761.

    Article  Google Scholar 

  10. Bernasconi P, Boni M, Cavigliano PM, Calatroni S, Brusamolino E, Passamonti F et al. Acute myeloid leukemia (AML) having evolved from essential thrombocythemia (ET): distinctive chromosome abnormalities in patients treated with pipobroman or hydroxyurea. Leukemia 2002; 16: 2078–2083.

    Article  CAS  Google Scholar 

  11. Dan K, Yamada T, Kimura Y, Usui N, Okamoto S, Sugihara T et al. Clinical features of polycythemia vera and essential thrombocythemia in Japan: retrospective analysis of a nationwide survey by the Japanese elderly leukemia and lymphoma study group. Int J Hematol 2006; 83: 443–449.

    Article  Google Scholar 

  12. Sterkers Y, Preudhomme C, Lai JL, Demory JL, Caulier MT, Wattel E et al. Acute myeloid leukemia and myelodysplastic syndromes following essential thrombocythemia treated with hydroxyurea: high proportion of cases with 17p deletion. Blood 1998; 91: 616–622.

    CAS  PubMed  Google Scholar 

  13. Wolanskyj AP, Schwager SM, McClure RF, Larson DR, Tefferi A . Essential thrombocythemia beyond the first decade: life expectancy, long-term complication rates, and prognostic factors. Mayo Clin Proc 2006; 81: 159–166.

    Article  Google Scholar 

  14. Chim CS, Kwong YL, Lie AK, Ma SK, Chan CC, Wong LG et al. Long-term outcome of 231 patients with essential thrombocythemia: prognostic factors for thrombosis, bleeding, myelofibrosis, and leukemia. Arch Intern Med 2005; 165: 2651–2658.

    Article  Google Scholar 

  15. Bazzan M, Tamponi G, Schinco P, Vaccarino A, Foli C, Gallone G et al. Thrombosis-free survival and life expectancy in 187 consecutive patients with essential thrombocythemia. Ann Hematol 1999; 78: 539–543.

    Article  CAS  Google Scholar 

  16. Kvasnicka HM, Thiele J . The impact of clinicopathological studies on staging and survival in essential thrombocythemia, chronic idiopathic myelofibrosis, and polycythemia rubra vera. Semin Thromb Hemost 2006; 32: 362–371.

    Article  Google Scholar 

  17. Fenaux P, Simon M, Caulier MT, Lai JL, Goudemand J, Bauters F . Clinical course of essential thrombocythemia in 147 cases. Cancer 1990; 66: 549–556.

    Article  CAS  Google Scholar 

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

    Article  CAS  Google Scholar 

  19. Kiladjian JJ, Rain JD, Bernard JF, Briere J, Chomienne C, Fenaux P . Long-term incidence of hematological evolution in three French prospective studies of hydroxyurea and pipobroman in polycythemia vera and essential thrombocythemia. Semin Thromb Hemost 2006; 32: 417–421.

    Article  CAS  Google Scholar 

  20. Thiele J, Kvasnicka HM . A critical reappraisal of the WHO classification of the chronic myeloproliferative disorders. Leuk Lymphoma 2006; 47: 381–396.

    Article  Google Scholar 

  21. Kokmen E, Ozsarfati Y, Beard CM, O'Brien PC, Rocca WA . Impact of referral bias on clinical and epidemiological studies of Alzheimer's disease. J Clin Epidemiol 1996; 49: 79–83.

    Article  CAS  Google Scholar 

  22. Vardiman JW, Brunning RD, Harris NL . WHO histological classification of chronic myeloproliferative diseases. In: Jaffe ES, Harris NL, Stein H, Vardiman JW (eds). World Health Organization Classification of Tumors: Tumours of the Haematopoietic and Lymphoid Tissues. Lyon, France: International Agency for Research on Cancer (IARC) Press, 2001, pp. 17–44.

    Google Scholar 

  23. Thiele J, Kvasnicka HM . Hematopathologic findings in chronic idiopathic myelofibrosis. Semin Oncol 2005; 32: 380–394.

    Article  Google Scholar 

  24. Bennett JM, Catovsky D, Daniel MT, Flandrin G, Galton DA, Gralnick HR et al. Proposals for the classification of the acute leukaemias. French-American-British (FAB) co-operative group. Br J Haematol 1976; 33: 451–458.

    Article  CAS  Google Scholar 

  25. Steensma DP, Dewald GW, Lasho TL, Powell HL, McClure RF, Levine RL et al. The JAK2 V617F activating tyrosine kinase mutation is an infrequent event in both ‘atypical’ myeloproliferative disorders and myelodysplastic syndromes. Blood 2005; 106: 1207–1209.

    Article  CAS  Google Scholar 

  26. Cabello AI, Collado R, Ruiz MA, Martinez J, Navarro I, Ferrer R et al. A retrospective analysis of myelodysplastic syndromes with thrombocytosis: reclassification of the cases by WHO proposals. Leuk Res 2005; 29: 365–370.

    Article  Google Scholar 

  27. Merlat A, Lai JL, Sterkers Y, Demory JL, Bauters F, Preudhomme C et al. Therapy-related myelodysplastic syndrome and acute myeloid leukemia with 17p deletion. A report on 25 cases. Leukemia 1999; 13: 250–257.

    Article  CAS  Google Scholar 

  28. Mauritzson N, Albin M, Rylander L, Billstrom R, Ahlgren T, Mikoczy Z et al. Pooled analysis of clinical and cytogenetic features in treatment-related and de novo adult acute myeloid leukemia and myelodysplastic syndromes based on a consecutive series of 761 patients analyzed 1976–1993 and on 5098 unselected cases reported in the literature 1974–2001. Leukemia 2002; 16: 2366–2378.

    Article  CAS  Google Scholar 

  29. Hernandez JA, Florensa L, Sole F, Bosch MA, Espinet B . Acute myeloid leukemia with 17p abnormality in untreated essential thrombocythemia. Leukemia 2001; 15: 1308.

    Article  CAS  Google Scholar 

  30. Tefferi A . Is hydroxyurea leukemogenic in essential thrombocythemia. Blood 1998; 92: 1459–1460.

    CAS  PubMed  Google Scholar 

  31. Lengfelder E, Hochhaus A, Kronawitter U, Hoche D, Queisser W, Jahn-Eder M et al. Should a platelet limit of 600 × 10(9)/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  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations


Corresponding author

Correspondence to A Tefferi.

Additional information

Supplementary Information accompanies the paper on the Leukemia website (

Supplementary information

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Gangat, N., Wolanskyj, A., McClure, R. et al. Risk stratification for survival and leukemic transformation in essential thrombocythemia: a single institutional study of 605 patients. Leukemia 21, 270–276 (2007).

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI:


  • thrombocythemia
  • survival
  • leukemia
  • JAK2V617F
  • risk
  • transformation

This article is cited by


Quick links