罕见肿瘤临床试验患者报告结局测量工具研究及应用现状  

Research and application status of patient-reported outcome measures in rare tumor clinical trials

作　　者：董晴 邢淑君 刘佳琪 张雪 王新宇 王书航 唐玉 李宁 Dong Qing;Xing Shujun;Liu Jiaqi;Zhang Xue;Wang Xinyu;Wang Shuhang;Tang Yu;Li Ning(Department of Clinical Trial Center,National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100021,China)

机构地区：[1]国家癌症中心、国家肿瘤临床医学研究中心、中国医学科学院北京协和医学院肿瘤医院药物临床试验研究中心,北京100021

出　　处：《中国肿瘤临床与康复》2024年第7期416-423,共8页Chinese Journal of Clinical Oncology and Rehabilitation

基　　金：中国医学科学院医学与健康科技创新工程(2021-I2M-1-045);中国癌症基金会北京希望马拉松专项基金护理课题(LC2021C04)。

摘　　要：罕见肿瘤已逐渐受到全社会关注,但因发病率低、患者数量少等,其研究开展存在困难。患者报告结局(PRO)指标关注来自患者及治疗相关的信息,为临床试验用药效果及上市考量提供全面的评价依据。文章主要综述了目前罕见肿瘤临床试验中应用的普适性及特异性PRO测量工具,介绍其特点与应用现状,以期为国内罕见肿瘤PRO的科学选用提供参考依据。Rare tumors have gradually garnered attention of the whole society,however due to the low incidence rate and the small patient populations,conducting research presents challenges.Patient-reported outcome(PRO)focus on information provided directly by patients and treatment-related aspects offering a comprehensive evaluation basis for assessing drug efficacy in clinical trials and considerations for market approval.This article mainly reviews the universal and specific PRO measurement tools used in clinical trials of rare tumors,introduces their characteristics and application status,in order to provide reference for the scientific selection of PRO for rare tumors in China.

关 键 词：罕见肿瘤 临床试验 患者报告结局 测量工具 

分 类 号：R730.5[医药卫生—肿瘤]