a Ospedale di Circolo , ASST Sette Laghi, Hematology , Varese , Italy.
b Department of Medicine and Surgery , Universita degli Studi dell'Insubria , Varese , Italy.
Expert Rev Hematol. 2019 Mar;12(3):159-171. doi: 10.1080/17474086.2019.1585239. Epub 2019 Mar 13.
Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm characterized by thrombocytosis, increased risk of thrombotic/hemorrhagic events and clonal evolution into blast phase or myelofibrosis. Areas covered: The authors will discuss biology, diagnosis, prognosis, therapy, and outcome of ET. An accurate molecular-morphologic assessment is necessary in order to properly establish diagnosis and prognosis of ET. Stratification for thrombosis prediction is essential, and IPSET-t model is widely applied. The current treatment strategy is directed to lower the rate of vascular events using cytoreduction in patients at high risk. Prophylactic low dose aspirin indication is more uncertain. To date, therapies for patients who are resistant or intolerant to first-line treatments are scarce. Overall, life expectancy indicates an indolent disease, but IPSET model helps in predicting survival at the time of diagnosis. Expert opinion: Challenging for the future will be to share criteria for ET diagnosis with the community. New insights into the molecular pathogenesis of the disease will improve the prediction of clonal evolution and outcome, and lead to the use of disease-modifying treatments.
原发性骨髓纤维化（ET）是一种慢性骨髓增生性肿瘤，其特征是血小板增多、血栓形成/出血事件风险增加以及向白血病期或骨髓纤维化期演变。
作者将讨论 ET 的生物学、诊断、预后、治疗和结局。为了正确确定 ET 的诊断和预后，需要进行准确的分子形态评估。血栓形成预测的分层至关重要，IPSET-t 模型得到广泛应用。目前的治疗策略是针对高危患者进行细胞减少以降低血管事件的发生率。预防性低剂量阿司匹林的适应证更不确定。迄今为止，对于对一线治疗耐药或不耐受的患者，治疗方法有限。总的来说，预期寿命表明这种疾病是惰性的，但 IPSET 模型有助于在诊断时预测生存。
未来的挑战将是与社区共享 ET 诊断标准。对疾病分子发病机制的新认识将改善对克隆演变和结局的预测，并导致使用疾病修饰治疗。
