Mechanical circulatory support: Measures of adjustment and quality of life

 描述(由申请人提供)：迫切需要开发更好的方法来评估对机械循环支持(MCS)和与健康相关的生活质量(HRQOL)的调整，因为这种疗法对患者生活的影响是深远的。MCS设备被植入到晚期心力衰竭患者的手术中，帮助心脏将血液泵到全身。出院后，患者管理和排除MCS设备故障，同时尽其所能恢复日常生活活动(例如，家庭责任、工作和娱乐)。MCS的好处包括更长的存活期(与晚期心力衰竭的药物治疗相比)、减少心力衰竭症状和改善身体功能。与设备相关的并发症(包括出血、感染、设备故障和中风)是MCS植入的风险。随着MCS技术的发展，存活率正在提高，不良事件的风险正在降低。然而，未来对这项昂贵技术的可取性的建议不仅取决于生存能力和不良事件的风险，还取决于其他结果，如对MCS和HRQOL的调整，这些结果的定义不太明确。重要的是，目前可用的HRQOL工具是通用的或专为心力衰竭或其他疾病设计的，没有解决MCS对患者日常生活的许多令人担忧的问题和独特的负担。目前，每年有50,000至100,000名患者被诊断为晚期心力衰竭。考虑到心脏移植的可获得性有限，晚期心力衰竭患者可能会被提供MCS作为一种永久性植入(即，目的地治疗)。我们建议开发一种MCS特有的测量系统，我们将其称为机械循环支持：调整和生活质量(MCS A-QOL)。我们的研究设计将重点放在我们的MCS A-QOL测量系统的开发和心理测量测试以及对我们的概念模型的评估上，然后探索患者和提供者对我们的测量(通过移动应用程序)的可用性。随着测量系统的发展，我们的研究将推动晚期心力衰竭患者的MCS领域的发展。使用我们的有效和响应性的MCS A-QOL测量将大大有助于更清楚地定义与药物治疗或其他策略相比，这一不断发展的技术的好处和风险。因此，随着MCS A-QOL被纳入我们对风险和收益的理解中，“生存”将变得更有意义。

 DESCRIPTION (provided by applicant): There is a critical need to develop better measures to assess adjustment to mechanical circulatory support (MCS) and health-related quality of life (HRQOL) because the impact of this therapy on patients' lives is profound. MCS devices are implanted surgically in patients with advanced heart failure and help the heart pump blood throughout the body. After discharge from the hospital, patients manage and troubleshoot their MCS devices while returning, as best they can, to usual activities of daily living (e.g., home and family responsibilities, work, and recreation). Benefits of MCS include longer survival (as compared to medical management of advanced heart failure), reduced heart failure symptoms, and improved physical function. Device-related complications (including bleeding, infection, device malfunction, and stroke) are risks of MCS implantation. As MCS technology evolves, survival is improving and the risk of adverse events is decreasing. However, future recommendations on the advisability of this expensive technology will depend not only on survivability and the risk of adverse events, but also on other outcomes, such as adjustment to MCS and HRQOL, which are less well defined. Importantly, currently available HRQOL instruments, which are generic or designed for heart failure or other illnesses, do not address many of the issues of concern and unique burdens of MCS on patients' daily lives. Currently, 50,000 to 100,ooo patients are diagnosed with advanced heart failure annually. Given the limited availability of heart transplantation, advanced heart failure patients may be offered MCS as a permanent implant (i.e., destination therapy). We propose to develop an MCS-specific measurement system, which we will refer to as Mechanical Circulatory Support: Adjustment and Quality of Life (MCS A-QOL). Our study design will focus on development and psychometric testing of our MCS A-QOL measurement system and evaluation of our conceptual model, followed by an exploration of usability of our measures (via a mobile app) by patients and providers. With development of a measurement system, our research will advance the field of MCS in patients with advanced heart failure. Use of our valid and responsive MCS A-QOL measures will contribute significantly to more clearly defining the benefits and risks of this evolving technology as compared with medical therapy or other strategies. Thus, "survival" will become more meaningful, as MCS A-QOL is incorporated into our understanding of risks and benefits.