Hematology· Topic 6.6 Myeloproliferative and myelodysplastic neoplasms· 6.6.1

Essential thrombocythemia

Essential thrombocythemia is a clonal myeloproliferative neoplasm driven by Janus kinase 2 (JAK2), calreticulin (CALR), or MPL mutations that produces a sustained platelet count above 450x10^9/L. Diagnosis rests on marrow megakaryocytic hyperplasia after excluding reactive thrombocytosis and other myeloproliferative neoplasms. High-risk patients are treated with low-dose aspirin plus hydroxyurea cytoreduction.

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What this article covers

  • Overview

    What it is: essential thrombocythemia (ET) is a clonal myeloproliferative neoplasm (MPN) of the hematopoietic stem cell, defined by megakaryocytic hyperplasia in the bone marrow and a sustained…

  • Epidemiology

    Typical patient: median age at diagnosis is 60 years, with a second, smaller peak among women under 40; women are affected somewhat more often than men overall.

  • Risk factors

    Age 60 years or older: the single strongest predictor of thrombosis and the age threshold used to stratify treatment intensity.

  • Pathophysiology

    The driver mutations: JAK2 V617F occurs in about 50-60% of patients, CALR exon 9 insertion-deletion mutations in about 20-35%, and MPL exon 10 mutations in about 5%; up to 10% of patients carry none of the three ("triple-negative" ET).

  • Clinical features

    Asymptomatic finding: about half of patients have no symptoms; the disease is discovered on a CBC obtained for another reason.

  • Differential diagnosis

    Reactive (secondary) thrombocytosis: infection, inflammation, iron deficiency, or recent surgery elevates the platelet count, but the marrow lacks megakaryocytic clustering or atypia, and the count falls once the trigger resolves.

  • Diagnosis

    What establishes the diagnosis: a sustained platelet count above 450x10^9/L together with a bone marrow biopsy showing megakaryocytic hyperplasia with large, mature, hyperlobulated megakaryocytes…

  • Management

    First, risk-stratify: classify every patient as low risk (age under 60 years, no history of thrombosis) or high risk (age 60 years or older, and/or a history of arterial or venous thrombosis), which determines whether cytoreduction is added.

  • Complications

    Arterial thrombosis: stroke, transient ischemic attack, myocardial infarction, and digital or limb ischemia, more frequent in JAK2-positive disease.

  • Prognosis

    Overall survival: most patients with ET have a life expectancy close to an age-matched general population, better than PV or PMF.

  • Prevention

    Aspirin for thrombosis prevention: low-dose aspirin 81 mg PO once daily, continued indefinitely in patients who can tolerate it, is the mainstay of thrombosis prevention across risk groups.

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