125例中国年轻原发性血小板增多症患者的临床特点及预后分析  

Clinical Characteristics and Long-Term Outcome of 125 Chinese Young Patients with Essential Thrombocythemia

作　　者：付荣凤 刘晓帆[1] 刘葳[1] 黄月婷[1] 陈云飞[1] 李慧媛[1] 鞠满凯 杨仁池[1] 张磊 

机构地区：[1]中国医学科学院北京协和医学院血液病医院(血液学研究所),实验血液学国家重点实验室,天津300020

出　　处：《中国实验血液学杂志》2017年第3期837-842,共6页Journal of Experimental Hematology

基　　金：国家自然科学基金(81600099;81470302);天津市应用基础及前沿技术研究计划(15JCZDJC35800)

摘　　要：目的:探讨中国年轻原发性血小板增多症(ET)患者的临床特点及长期转归,建立年轻ET患者特异的血栓预测模型,为预后判断及治疗提供依据。方法:对1990年7月1日至2014年12月31日诊治的的125例年轻ET患者的病历资料进行回顾性分析。结果:在全部125例患者中,男37例,女88例,诊断时中位年龄32(18-40)岁。随访中18例(14.4%)出现严重血栓事件。多因素分析表明,JAK2 V617F(HR=8.895,P=0.001)、既往血栓史(HR=8.001,P<0.001)及WBC≥12.0×10~9/L(HR=5.225,P=0.002)为血栓事件的独立危险因素。年轻ET患者的血栓发生率及血栓危险因素不同于整体ET人群,因此建立了年轻ET特异的血栓预测模型,即根据JAK2V617F(2分)、既往血栓史(2分)和WBC≥12.0×10~9/L(1分),将年轻ET分为低危(0分)、中危(1-2分)和高危(≥3分),3组的无血栓生存有显著差异(χ~2=32.223,P<0.001)。使用抗血小板聚集药物可预防血栓形成(HR=0.081,P<0.001),但只有在中、高危患者中,减低血小板药物可降低血栓发生率[14.3%(5/35)vs 36.4%(12/33),χ~2=4.416,P=0.036]。7例患者(5.6%)进展为骨髓纤维化,其中1例进展为骨髓纤维化后又进展为急性白血病。仅诊断时WBC≥15.0×10~9/L是疾病进展危险因素(χ~2=5.434,P=0.020),抗血小板聚集药物及减低血小板药物均不能阻止疾病进展。结论:中国年轻ET患者的血栓发生率与血栓危险因素与整体ET患者不同,年轻ET患者特异的血栓预测模型对指导分层治疗有重要价值。Objective： To investigate the clinical （ 〈40 years ） with essential thrombocythemia （ET） , characteristics and long-term outcome of Chinese young patients and to develop a thrombosis predicting model specific for young patients with ET, so as to provide a new evidence for risk stratification and treatment. Methods： Medical records of 125 Chinese young patients with newly diagnosed of ET were retrospectively analyzed. Results The median age at diagnosis was 32 （ 18 -40） years old, with 37 males and 88 females. During follow-up, 18 patients （ 14.4% ） experienced major thrombotic events. JAK2 V617F （ HR = 8. 895, P = 0. 001 ）, history of thrombosis （ HR = 8. 001, P 〈 0. 001 ） and WBC /〉 12.0 x 109/L （ HR = 5. 225, P = 0. 002 ） were independent risk factors for thrombosis. The incidence of thrombosis and risk factors in young patients were different from that in general ET population, so a thrombosis predicting model specific for young patients with ET was developed. In this model, JAK2 V617F （score 2 ）, history of thrombosis （score 2） and WBC〉12.0 x 109/L （ score 1 ） were used to divide the patients into low risk （ score 0）, intermediate risk （ score 1 -2） and high risk （score 〉 3 ） groups. These 3 groups exhibited significantly different thrombosis-free survival （X2 = 32. 223, P 〈 0.001 ）. Antiplatelet treatment could prevent the occurrence of thrombosis （ HR --- 0. 081, P 〈 0. 001 ）, while cytoreductive agents significantly decreased the risk of thrombosis only in intermediate and high risk groups （ 14. 3% vs 36.4% , X2 =4. 416, P = 0. 036）. Seven patients （5.6%） evolved to myelofibrosis, and one of them finally progressed in to acute leukemia. The only risk factor for evolution was WBC〉15.0 x 109/L （ X2 =5. 434, P =0.020）.Neither antiplatelet treatment nor cytoreductive agents could prevent disease progression. Conclusion： The incidence of thrombosis and risk factors in young patients with ET are different from that in

关 键 词：血小板增多 原发性血小板增多症 血栓形成 危险分层 骨髓纤维化 

分 类 号：R558.3[医药卫生—血液循环系统疾病]