The effects of lung transplantation on lung disease related disabilities

描述（由申请人提供）：健康相关生活质量（HRQL）是肺移植中一个关键的、以患者为中心的结果。虽然移植后HRQL可能会改善，但现有数据表明，HRQL仍远低于一般人群。在残疾的理论模型中，功能限制领域导致残疾，两者都是HRQL的前体，因此也是HRQL的“上游”决定因素。迄今为止，HRQL的决定因素以及移植后HRLQ限制中持续性限制和相关残疾的作用尚未阐明。该研究的中心假设是，终末期肺病患者将遭受功能限制、残疾和较差的HRQL，并且移植后，肺和非肺器官功能障碍（部分与维持移植物功能所需的治疗相关）将导致残余功能限制、持续残疾和持续降低的HRQL。提出了一项前瞻性队列研究，以评估肺移植前后终末期肺病患者的功能限制、残疾和HRQL。移植前，受试者将完成一项评估功能限制、残疾和HRQL的结构化调查。此外，还将进行肺功能、运动能力（6分钟步行测试）和下肢力量测试。与移植的疾病适应证、疾病严重程度和合并症相关的补充数据将从医疗记录中提取。关键措施将在肺移植后3、6和12个月重复。基线数据将用于：阐明终末期肺病对等待肺移植患者的功能限制、残疾和HRQL的影响。纵向数据将用于评估肺移植对功能限制、残疾和HRQL的影响，并确定可能影响HRQL的持续或新的限制或残疾的来源。我们打算使用重复测量分析对30名受试者进行研究，并允许足够的研究能力来检测关键结果测量的0.5 SD变化（与临床有意义的差异一致）。

DESCRIPTION (provided by applicant): Health related quality of life (HRQL) is a key, patient-centered outcome in lung transplantation. While HRQL may improve following transplantation, available data indicate that HRQL remains well below that of the general population. In theoretical models of disablement, the domain of functional limitation leads to disability and both are precursors to and, therefore, "upstream" determinants of HRQL. To date, the determinants of HRQL and the role of persistent limitations and associated disability in the HRLQ limitations following transplant have not been elucidated. The central study hypotheses are that patients with end-stage lung disease will suffer from functional limitations, disability, and poor HRQL and that following transplantation, both pulmonary and non-pulmonary organ dysfunction, associated in part with therapies required to preserve graft function, will account for residual functional limitations, ongoing disability, and persistently reduced HRQL. A prospective cohort study is proposed to assess functional limitations, disability, and HRQL in patients with end-stage lung disease before and after lung transplantation. Prior to transplantation, subjects will complete a structured survey assessing functional limitations, disability, HRQL. In addition, tests of lung function, exercise capacity (6 minute walk test), and lower extremity strength will be performed. Supplemental data related to the disease indication for transplantation, disease severity and comorbidities will be extracted from medical records. Key measures will be repeated at 3, 6, and 12 months after lung transplantation. Baseline data will be used as follows: to elucidate the impact of end- stage lung disease on functional limitation, disability, and HRQL on patients awaiting lung transplantation. Longitudinal data will be used as follows: to assess the impact of lung transplantation on measures of functional limitation, disability, and HRQL and to identify sources of persistent or new limitations or disabilities that may affect HRQL. We intend to study 30 subjects using repeated measures analysis and allowing for sufficient study power to detect an 0.5 SD change (consistent with a clinically meaningful difference) in key outcome measures. PUBLIC HEALTH RELEVANCE: Characterizing how lung transplantation enables or does not assist patients with end-stage lung disease to return to participating in activities they rate as meaningful (valued life activities) should provide valuable information to patients, their families, and health care providers coping with post- transplantation management. Moreover, elucidating the causes of functional limitations and disability following transplant may identify new areas for intervention that may lead to improved outcomes, in particular HRQL.