EMPHYSEMA: OUTCOMES AND TECHNOLOGY ASSESSMENT

该项目旨在改善肺气肿患者的预后。 诸如肺减容手术（LVRS）和肺移植（LTx）等治疗提供了生活质量的潜在改善，但它们带来了巨大的风险。 因此，关于LVRS和LTx的使用的决策需要权衡。 对这些权衡的合理分析需要有效测量患者在影响持续时间和生活质量的所有相关领域（包括发病率、功能状态、症状和满意度）的获益和损害。我们没有一个成功的治疗标准，特别是在生存时间不是唯一问题的情况下。 我们用来评估程序的工具可能无法完全捕捉最重要的健康和生活质量方面。 评估成果的新工具和模式是对现有工具和模式的补充。本项目的具体目的是：1）进一步开发评估肺气肿患者结局的方法，2）评估肺减容手术和肺移植对肺气肿患者结局的影响。将评估治疗对死亡率和生活质量的影响（以效用衡量）。 将测量患者人口统计学、共病、功能状态、症状和满意度，以及中间生理结局和资源利用。为了实现特定目标，将通过三种方式进行原始数据收集：仪器验证、横断面和（术前和术后）纵向研究。 肺气肿患者的功能状态、满意度、生理学和生活质量之间的关系模型将被开发并用于解释各种结局之间的关系。我们还将对接受LVRS或LTx的肺气肿患者的成功和不成功治疗制定操作定义，这些定义可解释功能状态、满意度、生理学和生活质量以及死亡率。将制定接受LVRS或LTx的肺气肿患者结局的预测规则。该研究计划促进评估和理解新技术和治疗干预在肺气肿患者中的作用的方法的发展。

This project seeks to improve outcomes in patients with emphysema. Therapies such as lung volume reduction surgery (LVRS) and lung transplantation (LTx) offer potential improvements in quality of life, but they impose substantial risks. Thus, decision making about the use of LVRS and LTx requires trade-offs. Rational analyses of these trade-offs require valid measurements of the benefits and harms to the patients in all relevant domains that effect duration and quality of life, including morbidity, functional status, symptoms, and satisfaction. We do not have an agreed upon standard of successful therapy, specifically in situations where duration of survival is not the only issue. The instruments we use to assess procedures may not fully capture the aspects of health and quality of life that are most important. New instruments and models for assessing outcomes are necessary to complement those that exist. The specific aims of this project are 1) to further develop methods to assess outcomes of patients with emphysema, and 2) to assess the effects of lung volume reduction surgery and lung transplant on outcomes of patients with emphysema. The effects of therapies on mortality and quality of life, measured as utilities, will be assessed. Patient demographics, co-morbidity, functional status, symptoms, and satisfaction, as well as intermediate physiologic outcomes, and utilization of resources will be measured. To achieve the specific aims, primary data collection will be performed in three ways: instrument validation, cross-sectional and (pre and post-operative) longitudinal studies. Models of the relationships among functional status, satisfaction, physiology, and quality of life in patients with emphysema will be developed and used to explain the relationships among the various outcomes. We will also develop operational definitions of successful and unsuccessful treatments among patients with emphysema undergoing LVRS or LTx that account for functional status, satisfaction, physiology, and quality of life, as well as mortality. Prediction rules for outcomes among patients with emphysema undergoing LVRS or LTx will be developed. This research plan promotes the development of methods for assessing and understanding the role of new technologies and therapeutic interventions in patients with emphysema.