School of Health and Related Research, University of Sheffield, Sheffield, UK.
Economics, University of Sheffield, Sheffield, UK.
BMJ Open. 2019 Mar 9;9(3):e023685. doi: 10.1136/bmjopen-2018-023685.
Preference-based measures (PBMs) of health-related quality of life (HRQoL) are used to generate quality-adjusted life years, which are necessary for cost-effectiveness evaluations of health interventions via cost-utility analysis. These measures of health can be generic (ie, pandiagnostic) or condition specific. No condition-specific PBM of HRQoL in Duchenne muscular dystrophy (DMD) exists, yet there are concerns that standard generic measures lack the specificity to assess aspects of HRQoL that are especially important to people with DMD. This study has been designed to produce a condition-specific PBM of HRQoL in DMD.
This mixed-methods study proceeds through three stages. In the first stage (concept elicitation), semistructured interviews will be conducted with boys and men diagnosed with DMD, and analysed with framework to produce a draft health state descriptive system for HRQoL in DMD. In the second stage (refining the descriptive system), patients, clinicians and primary caregivers of people with DMD will assess the face validity of the descriptive system. This will be followed by a quantitative survey on a larger sample of patients, which will be analysed with psychometric analyses to produce a refined descriptive system. In the third stage (valuation and econometric modelling), an online discrete choice experiment with duration will be administered to a general public sample to generate utility values for the new measure.
This study has received ethical approval from the National Health Service (REC reference: 18/SW/0055). The primary output of this research will be a condition-specific PBM (or 'bolt-on' to an existing generic PBM) in people with DMD and an associated value set. Results will be disseminated through international conferences and open-access journals.
健康相关生活质量（HRQoL）的偏好测量（PBM）用于生成质量调整生命年，这是通过成本效用分析对健康干预措施进行成本效益评估所必需的。这些健康衡量标准可以是通用的（即泛诊断）或特定于疾病的。杜氏肌营养不良症（DMD）中不存在特定于疾病的 HRQoL PBM，但人们担心标准通用措施缺乏特异性，无法评估对 DMD 患者特别重要的 HRQoL 方面。本研究旨在制定 DMD 中特定于疾病的 HRQoL PBM。
这项混合方法研究分三个阶段进行。在第一阶段（概念发掘）中，将对确诊患有 DMD 的男孩和男性进行半结构化访谈，并使用框架进行分析，以生成 DMD 中 HRQoL 的草案健康状态描述系统。在第二阶段（描述系统的精炼）中，DMD 患者、临床医生和患者的主要照顾者将评估描述系统的表面有效性。之后，将对更大的患者样本进行定量调查，并使用心理测量分析对其进行分析，以生成精炼的描述系统。在第三阶段（评估和计量经济学建模）中，将对一般公众样本进行在线离散选择实验（持续时间），以生成新测量方法的效用值。
这项研究已获得国家卫生服务机构（REC 参考号：18/SW/0055）的伦理批准。这项研究的主要成果将是针对 DMD 患者的特定于疾病的 PBM（或“附加”到现有的通用 PBM）以及相关的价值集。研究结果将通过国际会议和开放获取期刊进行传播。
