Leukemic Transformation in Essential Thrombocythemia

Essential thrombocythemia patients develop acute myeloid leukemia (AML) at a rate of 1-4% during a median follow-up of 7-10 years. The risk increases with advanced age, anemia, platelet count ≥ 1000 × 10(9)/l, the presence of ≥ 2 somatic mutations and after the first decade of diagnosis. The use of alkylating agents and (32)radiophosphorus, particularly in higher doses, but not hydroxyurea and anagrelide, increases the risk. AML in essential thrombocythemia patients is frequently associated with unfavorable cytogenetics and poor prognosis. In young and fit patients, AML-type induction chemotherapy followed by allogeneic stem cell transplantation may offer the best chance of long-term disease control. In select elderly patients with poor performance status, hypomethylating agent such as azacytidine may prolong survival.