70例成人阵发性睡眠性血红蛋白尿症临床分析(英文)  

Clinical Analysis of 70 Adult Patients with Paroxysmal Nocturnal Hemoglobinuria

作　　者：葛美丽[1] 李星鑫[1] 邵英起[1] 施均[1] 郑以州[1] 

机构地区：[1]中国医学科学院北京协和医学院血液学研究所血液病医院,天津300020

出　　处：《中国实验血液学杂志》2015年第3期774-778,共5页Journal of Experimental Hematology

基　　金：the grant No.81300388 from National Natural Science Foundation of China;grant No.14JCQNJC10700 from Tianjin Municipal Science and Technology Commission

摘　　要：目的:探讨我国PNH患者的临床特征。方法:对我院2000年1月至2009年12月期间诊治的70例PNH患者临床资料进行了回顾性分析,并总结其临床表现、实验室检查特点、并发症、生存率及预后影响因素。结果:70例PNH患者的中位发病年龄为37岁(18-73岁),其中男41例,女29例,临床表现包括乏力(87.1%),血红蛋白尿(44.3%),感染(22.9%),出血(37.1%)和腹痛(2.9%);56例患者(80%)FHb(游离血红蛋白)大于50mg/L,54例患者(77.1%)Hp(结合珠蛋白)小于0.5 g/L和49例患者(70.0%)LDH水平大于220 U/L。患者并发症包括:再发的腹痛(2.9%)、感染(30.0%)、血栓事件(8.6%)、进展为MDS/AML(5.7%)、结石(11.4%)及死亡(17.1%)。经Kaplan-Meier法计算出的10年总体生存率为72.2%。单因素和多因素分析显示:血栓事件、进展为骨髓增生异常综合征和/或急性髓系白血病(MDS/AML)和再发的感染为影响生存的不良预后因素。结论:本研究详细描述了我国成人PNH患者临床特征及预后因素,有助于更深入的认识此疾病,尤其是对预后因素的研究有助于评估本病的治疗效果。Objective： To investigate the clinical characteristics of Chinese patients with paroxysmal nocturnal hemoglobinuria （PNI-I）. Methods：The clinical data of 70 adult PNH cases in our hospital from January 2000 to December 2009 were analyzed retrospectively, and the clinical manifestation, laboratory examination, treatment, complications and prognostic factors influencing survival rate were assessed. Results： The nosopoietic median age of 70 cases（41 male cases and 29 female cases） was 37 （18 -73） years old. The clinical manifestation included fatigue （87.1%）, hemogolobinuria （44.3%）, infection （22.9%）, bleeding （37.1%）, and abdominal pain （2.9%）. FHb （ free hemoglobin） in 56 patients（ 80% ） was 〉 50 mg/L. Hp （haptoglobin） in 54 patients （ 77.1% ） was 〈 0.5 g/L, and LDH in 49 patients（70.0% ）was 〉220 U/L. The overall 10 year-survival rate after diagnosis was 72.2% estimated by Kaplan-Meier. The complications in this study were as follow： recurrent abdominal pain crisis（2.9% ）, infections （30.0%） ,thrombotic events（8.6% ） ,evolution to MDS/AML（5.7% ） ,calculus（ 11.4% ） and death（ 17.1% ）. Both univariate and multivariate analyses identified risk factors affecting survival, including development of thrombotic events, progression to myelodysplastic syndrome or acute myelogenous leukemia （MDS/AML） and recurrent infections. Conclusion：This larger number of cases for the first time allowed us to carry out a detailed analysis of prognostic factors for this rare disease. Evaluation of PNH prognostic factors may provide a basis to assess the current and future therapies of this disease.

关 键 词：阵发性睡眠性血红蛋白尿症 再生障碍性贫血 预后因素 

分 类 号：R556.64[医药卫生—血液循环系统疾病]