我国发作性睡病疾病患者疾病特征、治疗现状与经济负担调查  

Characteristics,current treatment status and economic burden in Chinese patients with narcolepsy

作　　者：王春萍 郑佳音 李薇 王新玲 朱晓晨 肖若薇 朱贺 韩晟[1,2] 史录文[1,2] WANG Chunping;ZHENG Jiaying;LI Wei;WANG Xinling;ZHU Xiaochen;XIAO Ruowei;ZHU He;HAN Sheng;SHI Luwen(International Research Center for Medicinal Administration,Peking University,Beijing 100191,China;Department of Pharmacy Administration&Clinical Pharmacy,School of Pharmacy,Peking University,Beijing 100191,China;Beijing Society of Rare Disease Clinical Care Accessibility,Beijing 100020,China)

机构地区：[1]北京大学医药管理国际研究中心,北京100191 [2]北京大学药事管理与临床药学系,北京100191 [3]北京罕见病诊疗与保障学会,北京100020

出　　处：《中国医院药学杂志》2024年第18期2165-2170,共6页Chinese Journal of Hospital Pharmacy

摘　　要：目的:评估中国发作性睡病患者的疾病特征、治疗现状与经济负担。方法:于2021年11月-2022年8月,通过中国罕见病联盟自由研发和搭建的中国罕见病综合云服务平台募集发作性睡病患者,基于自行设计问卷与发作性睡病日间过度嗜睡量表开展线上问卷调研。结果:共收集有效问卷280份,覆盖了我国29个省(直辖市、自治区),主要分布在东部。成人组平均年龄为30.8岁,儿童/青少年组为15.1岁。2组均发现日间嗜睡严重,每月猝倒出现频率大于10次的比例超过50%,绝大多数患者存在多种共病,常见(40%以上)为焦虑或抑郁。成人组与儿童/青少年组均存在50%以上患者误诊,误诊中位时间分别为5.0、1.0个月,最长可至282.1、48.4个月;患者从首诊到确诊平均需要辗转2家医院,最多可达8~10家医院。成人组因患发作性睡病病年均总成本为37 537.10元,间接成本(53.17%)是其主要驱动因素;儿童/青少年组年为54 109.12元,直接医疗成本(54.74%)是其主要驱动因素。2组人均总成本均高于2021年我国居民人均可支配收入(35 128元)。结论:我国发作性睡病患者疾病负担较重,需进一步采取有效措施提高医疗服务可及性,加强患者症状管理与加快安全、有效且经济药物研发与上市,以减轻患者与家庭的经济负担。OBJECTIVE To assess the clinical characteristics,current treatments and financial impact of patients with narcolepsy in China.METHODS From November 2021 to August 2022,a comprehensive survey was conducted through selfdesigned questionnaires and Narcolepsy Daytime Sleepiness Scale,targeting narcoleptics via the platform developed and built China Alliance for Rare Diseases.RESULTS A total of 280 valid questionnaires were collected,covering 29 provinces of China,primarily from eastern regions.The average age of adults group was 30.8 years and 15.1 years for children/adolescent group.Both groups exhibited severe daytime sleepiness with >50% experiencing frequent sudden falls(over 10 times a month).A large majority of participants had multiple comorbidities with dominating anxiety or depression(>40%).Over 50% of patients in both groups were initially misdiagnosed with a median misdiagnostic duration of 5.0 months for adults and 1.0 month for children/adolescent,extending up to 282.1 months and 48.4 months respectively.On average,patients visited 2 hospitals before receiving a correct diagnosis and some cases required visits to 8-10 hospitals.The annual average total cost for adults with narcolepsy was 37537.10 yuan,with indirect costs as a predominant factor(53.17%) and for children/adolescents,it was 54 109.12 yuan,primarily driven by direct medical costs(54.74%).The per capita total costs for both groups exceeded the national average disposable income per capita in 2021(35 128 yuan).CONCLUSION The economic burden of narcolepsy in China is substantial,highlighting the need for more effective measures to improve the accessibility of medical services,enhance the development and launch of effective and economic medicines and reduce the economic strain on rare disease patients and their families.

关 键 词：发作性睡病 临床特征 治疗现状 经济负担 

分 类 号：R956[医药卫生—药学]