The burden of Duchenne muscular dystrophy An international, cross-sectional study
The burden of Duchenne muscular dystrophy An international, cross-sectional study
复制标题
DOI：
10.1212/wnl.0000000000000669
复制
发表时间：
2014-08-05
期刊：
影响因子：
9.9
通讯作者：
Bushby, Katharine
中科院分区：
文献类型：
--
作者：
Landfeldt, Erik;Lindgren, Peter;Bushby, Katharine
目的：本研究的目的是估计疾病的总成本和经济负担的Duchenne型肌营养不良症（DMD）。方法：DMD患者从德国，意大利，英国和美国被确定通过在欧洲的翻译研究，评估和治疗神经肌肉疾病登记处，并邀请完成一份在线问卷与照顾者。收集有关医疗保健使用，生活质量，工作状况，非正式护理和家庭开支的数据，估计DMD的成本从社会和照顾者households.Results的角度：共770例患者（173德国，122意大利，191来自英国，284来自美国）完成了问卷。平均每位患者每年的直接疾病费用估计在23 920美元至54 270美元（2012年国际元）之间，比这些国家的人均卫生支出高出7至16倍。间接和非正式护理费用很高，各占总费用的18%至43%。总的社会负担估计为每例患者每年80，120美元至120，910美元，并随着疾病进展而显著增加。相应的家庭负担估计在58，440美元和71，900美元之间。结论：我们表明DMD与相当大的经济负担有关。我们的结果强调了罕见疾病（例如DMD）所伴随的许多不同成本以及受影响家庭所承受的相当大的经济负担。我们对罕见疾病以前未知的经济背景的描述，为干预计划和新疗法的卫生政策评估、患者及其家属的财政支持计划以及未来成本研究的设计提供了重要的情报输入。
Objective: The objective of this study was to estimate the total cost of illness and economic burden of Duchenne muscular dystrophy (DMD).Methods: Patients with DMD from Germany, Italy, United Kingdom, and United States were identified through Translational Research in Europe-Assessment & Treatment of Neuromuscular Diseases registries and invited to complete a questionnaire online together with a caregiver. Data on health care use, quality of life, work status, informal care, and household expenses were collected to estimate costs of DMD from the perspective of society and caregiver households.Results: A total of 770 patients (173 German, 122 Italian, 191 from the United Kingdom, and 284 from the United States) completed the questionnaire. Mean per-patient annual direct cost of illness was estimated at between $23,920 and $54,270 (2012 international dollars), 7 to 16 times higher than the mean per-capita health expenditure in these countries. Indirect and informal care costs were substantial, each constituting between 18% and 43% of total costs. The total societal burden was estimated at between $80,120 and $120,910 per patient and annum, and increased markedly with disease progression. The corresponding household burden was estimated at between $58,440 and $71,900.Conclusions: We show that DMD is associated with a substantial economic burden. Our results underscore the many different costs accompanying a rare condition such as DMD and the considerable economic burden carried by affected families. Our description of the previously unknown economic context of a rare disease serves as important intelligence input to health policy evaluations of intervention programs and novel therapies, financial support schemes for patients and their families, and the design of future cost studies.