Gangat Naseema, Tefferi Ayalew
Mayo Clinic, Division of Hematology, 200 First Street SW, Rochester, MN 55905, USA.
Expert Opin Pharmacother. 2008 Jul;9(10):1679-85. doi: 10.1517/14656566.9.10.1679.
The natural history of essential thrombocythemia is characterized by an increased incidence of thrombotic and hemorrhagic events and, in the long-term, a tendency for disease transformation to myelofibrosis or acute leukemia. Advanced age and a prior history of thrombosis are the major predictors of thrombotic complications.
The aim of this study was to outline the current evidence and the authors' opinion regarding the clinical management of patients with essential thrombocythemia.
The study reviewed the pertinent literature addressing the management options for essential thrombocythemia patients.
RESULTS/CONCLUSIONS: Cytoreductive agents can reduce the rate of thrombotic events, but do not affect the overall survival or rate of disease transformation. Thus, a risk-adapted strategy is recommended for the management of patients with essential thrombocythemia. High-risk patients with essential thrombocythemia should be treated with cytoreductive therapy and low-dose aspirin. Hydroxycarbamide is the agent of choice in most patients: however, interferon-alpha is a reasonable alternative in young patients, pregnancy or those intolerant of hydroxycarbamide. Low-risk patients can be safely observed. The management of intermediate-risk patients needs to be individualized: however, low-dose aspirin can be used after excluding acquired von Willebrand's disease. In the future we await the role of molecularly targeted therapy with JAK2 inhibitors in high-risk essential thrombocythemia patients.
原发性血小板增多症的自然病史特点为血栓形成和出血事件的发生率增加，从长远来看，疾病有向骨髓纤维化或急性白血病转变的趋势。高龄和既往有血栓形成史是血栓形成并发症的主要预测因素。
本研究旨在概述目前关于原发性血小板增多症患者临床管理的证据及作者的观点。
本研究回顾了有关原发性血小板增多症患者管理选择的相关文献。
结果/结论：细胞减灭剂可降低血栓形成事件的发生率，但不影响总体生存率或疾病转化率。因此，建议对原发性血小板增多症患者采用风险适应性策略进行管理。原发性血小板增多症的高危患者应接受细胞减灭疗法和小剂量阿司匹林治疗。羟基脲是大多数患者的首选药物；然而，α干扰素是年轻患者、孕妇或不耐受羟基脲患者的合理替代药物。低危患者可安全观察。中危患者的管理需要个体化；然而，在排除获得性血管性血友病后可使用小剂量阿司匹林。未来，我们期待JAK2抑制剂分子靶向治疗在高危原发性血小板增多症患者中的作用。
