遗传性出血性毛细血管扩张症相关动脉性肺动脉高压患者生存状况及影响因素分析  

Survival status and prognostic factors analysis in patients with hereditary hemorrhagic telangiectasia‑associated pulmonary arterial hypertension

作　　者：俞洪锐 钱钰玲 夏婉莹 熊长明 韩慧君 姚桦 顾晴 何建国 Yu Hongrui;Qian Yuling;Xia Wanying;Xiong Changming;Han Huijun;Yao Hua;Gu Qing;He Jianguo(Pulmonary Vascular Disease Center,National Center for Cardiovascular Diseases,Fuwai Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100037,China;Department of Epidemiology and Biostatistics,Institute of Basic Medical Sciences,Chinese Academy of Medical Sciences&School of Basic Medicine,Peking Union Medical College,Beijing 100005,China;Department of Cardiology,Guangdong Cardiovascular Institute,Guangdong General Hospital,Guangdong Academy of Medical Sciences,Guangzhou 510080,China;Emergency and Critical Care Medicine Center,National Center for Cardiovascular Diseases,Fuwai Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100037,China)

机构地区：[1]中国医学科学院、北京协和医学院、国家心血管病中心、阜外医院呼吸与肺血管疾病诊治中心,北京100037 [2]中国医学科学院、基础医学研究所、北京协和医学院基础学院流行病与卫生统计学系,北京100005 [3]广东省医学科学院、广东省人民医院、广东省心血管病研究所心血管内科,广州510080 [4]中国医学科学院、北京协和医学院、国家心血管病中心、阜外医院急重症医学中心,北京100037

出　　处：《中华医学杂志》2024年第42期3910-3915,共6页National Medical Journal of China

基　　金：国家科技支撑计划(2006BAI01A07、2011BAI11B15);国家重点研发计划(2016YFC1304400)。

摘　　要：目的分析遗传性出血性毛细血管扩张症相关动脉性肺动脉高压(HHT‑PAH)患者的生存状况及影响因素。方法前瞻性纳入了2009年8月至2019年12月在中国医学科学院阜外医院和广东省人民医院诊断的HHT‑PAH患者,每6个月±2周对患者进行随访。采用多因素Cox风险回归模型分析全因死亡的影响因素;采用Kaplan‑Meier法绘制生存曲线;log‑rank检验比较有和无肝动静脉畸形患者生存时间差异。结果共纳入18例HHT‑PAH患者,女性14例,年龄[M(Q1,Q3)]为39(32,45)岁。中位随访时间为129(51,156)个月,生存时间为70(32,123)个月,随访期间12例患者死亡,1、3、5、10年生存率分别为100.0%、70.8%(95%CI:52.3%~96.0%)、53.1%(95%CI:34.0%~83.0%)、26.6%(95%CI:10.6%~66.4%)。有肝动静脉畸形HHT‑PAH患者中位生存时间短于无肝动静脉畸形患者[31.0(95%CI:16.6~45.4)比84.0(95%CI:54.4~113.6)个月,P=0.024]。Cox比例风险回归模型分析结果显示,肝动静脉畸形是HHT‑PAH患者全因死亡的危险因素(HR=4.493,95%CI:1.064~18.978)。结论HHT‑PAH患者的10年生存状况较差,肝动静脉畸形是HHT‑PAH患者死亡的危险因素。Objective To analyze the survival status and prognostic factors of patients with hereditary hemorrhagic telangiectasia‑associated pulmonary arterial hypertension(HHT‑PAH).Methods This prospective study included patients diagnosed with HHT‑PAH at the Fuwai Hospital of the Chinese Academy of Medical Sciences and Guangdong General Hospital from August 2009 to December 2019.Patients were followed up every 6 months±2 weeks,with all‑cause mortality as the study endpoint.Multivariate Cox proportional hazards regression mode was used to further screen and validate the prognostic factors.Survival curves were plotted using the Kaplan‑Meier method,and the log‑rank test was used to compare the survival time differences between patients with and without hepatic arteriovenous malformations.Results A total of 18 HHT‑PAH patients were included,with 14 females(77.8%)and a median age[M(Q1,Q3)]of 39(32,45)years.The median follow‑up time was 129(51,156)months.During the follow‑up period,12 patients(66.7%)died,with a median survival time of 70(32,123)months.The 1‑year,3‑year,5‑year,and 10‑year survival rates were 100.0%,70.8%(95%CI:52.3%-96.0%),53.1%(95%CI:34.0%-83.0%),and 26.6%(95%CI:10.6%-66.4%),respectively.HHT‑PAH patients with hepatic arteriovenous malformations had a shorter median survival time than those without hepatic arteriovenous malformations[31.0 months(95%CI:16.6-45.4 months)vs 84.0 months(95%CI:54.4-113.6 months),P=0.024].Cox proportional hazards regression analysis showed that hepatic arteriovenous malformation(HR=4.493,95%CI:1.064-18.978)was a risk factor for all‑cause mortality in HHT‑PAH patients.Conclusions The 10‑year survival rate of HHT‑PAH patients is poor.The presence of hepatic arteriovenous malformations is a risk factor for mortality in HHT‑PAH patients.

关 键 词：预后 遗传性出血性毛细血管扩张症 动脉性肺动脉高压 生存分析 随访研究 

分 类 号：R54[医药卫生—心血管疾病]