Sustaining quality of life of the aged: Heart transplant or mechanical support?

 描述（由申请人提供）： 本拟议研究的目的是比较接受心脏移植（HT）或机械循环支持（MCS）作为永久植入物（即，目的地治疗[DT]）和他们的照顾者，穷人的风险因素， HRQOL、不良事件和症状负担以及质量调整生命年（QOL）。我们将使用一个理论框架来模拟疾病、治疗、不良事件和症状对HRQOL的影响，以指导我们提出的研究。60-80岁的晚期HF患者是本拟定研究的适当目标人群，因为他们更频繁地接受HT和MCS器械植入，尽管临床结局较差的风险更大，但他们的生存率可接受。我们提出的研究可能有助于更好地以患者为中心的老年晚期HF患者及其护理人员的护理，通过告知决策和指导策略，以提高术后HRQOL。我们拟定的研究还将提供关键信息，以支持范式转变，从仅在HT评价后进行DT MCS植入，到同时考虑两种治疗，因为MCS后2年生存率接近HT后2年生存率的基准，HRQOL具有更高的优先级。采用前瞻性纵向设计，我们拟定的比较有效性研究将比较接受HT或DT MCS的患者及其护理人员从基线至术后2年的HRQOL结局。本拟定研究的主要目的是确定接受DT MCS的老年晚期HF患者与接受HT的患者相比，从基线至术后2年，总体HRQOL（主要）和HRQOL领域（身体、精神和社会）的变化是否非劣效性。第二个目标是（1）。确定接受DT MCS的老年晚期HF患者的护理人员与接受HT的老年晚期HF患者的护理人员相比，从基线到手术后2年，总体HRQOL和领域的变化是否非劣效;（2.- 3.）第三章在手术后2年，与老年HT患者及其护理人员相比，确定与老年DT MCS患者及其护理人员总体HRQOL较差相关的风险因素;（4.）确定与接受HT的患者相比，接受DT MCS的老年晚期HF患者在术后1年和2年时是否具有非劣效的无不良事件和症状率;以及（5）与在术后2年时接受HT的患者相比，评价接受DT MCS的老年晚期HF患者中QALs的分布。

 DESCRIPTION (provided by applicant): The purpose of this proposed study is to compare health-related quality of life (HRQOL) outcomes in older (60-80 years) advanced heart failure (HF) patients who undergo heart transplantation (HT) or mechanical circulatory support (MCS) as a permanent implant (i.e., destination therapy [DT]) and their caregivers, risk factors for poor HRQOL, adverse event and symptom burden, and quality-adjusted life years (QALYs). We will use a theoretical framework which models the influence of disease, treatment, adverse events, and symptoms on HRQOL to guide our proposed study. Advanced HF patients, 60-80 years of age, are an appropriate target group for this proposed study because they are receiving HTs and implant of MCS devices more frequently, and despite a greater risk for poor clinical outcomes, they have acceptable rates of survival. Our proposed study may contribute to better patient-centered care of older advanced HF patients and their caregivers, by informing decision making and guiding strategies to enhance post-operative HRQOL. Our proposed study will also provide critical information to support a paradigm shift, from DT MCS implant only after evaluation for HT, to concurrent consideration of both therapies, given that as 2-year survival after MCS approaches the benchmark 2-year survival after HT, HRQOL assumes a much higher priority. Using a prospective, longitudinal design, our proposed comparative effectiveness research will compare HRQOL outcomes in patients who receive HT or DT MCS and their caregivers, from baseline to 2 years post-operatively. The primary aim of this proposed study is to determine whether older advanced HF patients who undergo DT MCS, as compared to patients who undergo HT, experience non-inferior change in overall HRQOL (primary), and domains of HRQOL (physical, mental, and social) from baseline through 2 years after surgery. Secondary Aims are (1.) to determine whether caregivers of older advanced HF patients who undergo DT MCS, compared to caregivers of older advanced HF patients who undergo HT, experience non-inferior change in overall HRQOL and domains from baseline through 2 years after surgery; (2.-3.) to identify risk factors related to poorer overall HRQOL in older DT MCS patients and their caregivers, as compared to older HT patients and their caregivers, at 2 years after surgery; (4.) to determine whether older advanced HF patients who undergo DT MCS, as compared to patients who undergo HT, have non-inferior rates of freedom from adverse events & symptoms at 1 and 2 years after surgery; and (5) to evaluate the distribution of QALYs in older advanced HF patients who undergo DT MCS, as compared to patients who undergo HT at 2 years after surgery.