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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&lt;120 g/l; males&lt;135 g/l) was identified as an independent risk factor for both inferior survival and LT. Additional risk factors for survival included age &gt; or =60 years, leukocyte count&gt; or =15 x 10(9)/l, smoking, diabetes mellitus and thrombosis. For LT, platelet count&gt; or =1000 x 10(9)/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&lt;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.

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