Clinical prognostic factors and outcomes of essential thrombocythemia when transformed to myelodysplastic syndromes and acute myeloid leukemia

Leuk Res. 2016 Mar;42:52-8. doi: 10.1016/j.leukres.2016.01.015. Epub 2016 Feb 2.


Transformation of essential thrombocythemia (ET) to myelodysplastic syndromes or acute myeloid leukemia is infrequent, comprising 1-5% of cases with dismal clinical outcome. Studies on prognosis in ET patients with leukemic transformation are limited. The large cohort included 40 patients (1990-2014) with ET transformation (median age of 59 years, M:F of 1:1). Median time from ET diagnosis to transformation was 76 months (26-481) with median follow-up time of 15 years. Advanced age, myelofibrosis (grade 2-3), and leukocytosis at the time of transformation were associated with inferior OS from transformation (p<0.05). Given rarity of the clinical scenario, multicenter efforts are encouraged.

Keywords: Acute myeloid leukemia (AML); Essential thrombocythemia (ET); Myelodysplastic syndromes (MDS); Myelofibrosis; Prognostic factors; Transformation.

MeSH terms

  • Adult
  • Age Factors
  • Aged
  • Cell Transformation, Neoplastic / genetics
  • Cell Transformation, Neoplastic / pathology*
  • Female
  • Humans
  • Kaplan-Meier Estimate
  • Leukemia, Myeloid, Acute / mortality
  • Leukemia, Myeloid, Acute / pathology*
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic Syndromes / pathology*
  • Prognosis
  • Proportional Hazards Models
  • Retrospective Studies
  • Risk Factors
  • Thrombocythemia, Essential / genetics
  • Thrombocythemia, Essential / pathology*
  • Young Adult