■选定的不符合心脏移植资格的晚期心力衰竭患者可以从左心室辅助装置治疗中受益,作为“目的地治疗”。有证据表明目的地治疗的疗效;然而,由于缺乏经济证据,目前尚未在英国国家卫生局内委托。
■对于不适合进行心脏移植(目的地治疗)的晚期心力衰竭患者,左心室辅助装置的临床和成本效益与医疗管理相比如何?
■对左心室辅助装置作为目的地治疗的临床和成本效益的证据进行了系统审查,包括:在可行的情况下,一项网络荟萃分析,以间接评估当前可用的左心室辅助设备与医疗管理相比的相对有效性.对于系统审查,搜索的数据源(截至2022年1月11日)是CochraneCENTRAL,MEDLINE和EMBASE通过Ovid进行初步研究,以及Epidemonikos和Cochrane系统评价数据库,用于相关系统评价。还搜索了试用登记簿,以及来自特定干预措施登记册的数据和报告。经济研究在EconLit中被确定,CEA注册表和NHS经济评估数据库(NHSEED)。通过检查纳入研究的参考列表来补充搜索。从英国国家卫生服务/个人社会服务的角度,开发了一种经济模型(马尔可夫)来估算左心室辅助设备与医疗管理相比的成本效益。进行确定性和概率敏感性分析以探索不确定性。在可能的情况下,所有分析都集中在目前唯一可用的左心室辅助装置(HeartMate3TM,雅培,芝加哥,IL,美国)在英国。
■临床有效性综述包括134项研究(240篇)。没有直接比较HeartMate3和医疗管理的研究(一项随机试验正在进行中)。当前可用的左心室辅助装置与早期装置相比并相对于医疗管理提高了患者存活率并降低了中风率和并发症。例如,使用HeartMate3装置24个月时的生存率为77%,而使用HeartMateII时的生存率为59%(MOMENTUM3试验)。间接比较表明,与医疗管理相比,死亡率降低[相对死亡风险0.25(95%置信区间0.13至0.47);24个月;本研究]。成本效益审查包括5项成本分析和14项经济评估,涵盖不同世代的设备,并具有不同的观点。与医疗管理相比,报告的每质量调整生命年的增量成本较后几代设备更低[低至46,207英镑(2019年价格;英国观点;时间期限至少5年)]。从英国国家卫生服务/个人社会服务的角度来看,与医疗管理相比,经济评估使用了不同的方法来获得当前左心室辅助设备的相对效果。所有这些都给出了类似的增量成本效益比,即每获得质量调整后的寿命年-寿命期53,496-58,244英镑。模型输出对与医疗管理相关的参数估计敏感。根据心力衰竭的严重程度,探索性亚组分析的结果没有实质性差异。
■没有直接证据将HeartMate3的临床有效性与医疗管理进行比较。间接比较是基于来自异质性研究的有限数据,这些研究涉及心力衰竭的严重程度(机构间登记机构间登记机构机械辅助循环支持评分分布)和可能的生存。此外,英国晚期心力衰竭的医疗管理成本尚不清楚.
■使用英国适用的成本效益标准,对于不符合心脏移植条件的晚期心力衰竭患者,左心室辅助装置与医疗管理相比可能不具成本效益.如果可用,持续评估HeartMate3与医疗管理相比的数据可用于更新成本效益估计值.需要对英国的医疗管理成本进行审计,以进一步减少经济评估中的不确定性。
■本研究注册为PROSPEROCRD42020158987。
■该奖项由美国国家卫生与护理研究所(NIHR)卫生技术评估计划(NIHR奖参考:NIHR128996)资助,并在《卫生技术评估》中全文发表。28号38.有关更多奖项信息,请参阅NIHR资助和奖励网站。
由于年龄和合并症,大多数晚期心力衰竭患者不适合进行心脏移植,但选定的患者可以从左心室辅助装置中受益。用于此类患者的左心室辅助设备治疗被称为“目的地治疗”。这是一种长期疗法,涉及植入电池供电的泵来支持患者的心脏。该项目的目的是收集和评估左心室辅助装置用于目的地治疗时有效性的研究证据。并从英国国家卫生服务/个人社会服务的角度评估与医疗管理相比的物有所值。这项研究发现,目前可用的左心室辅助设备与早期设备相比,与医疗管理相比,可以提高患者的生存率,并减少中风率和并发症。然而,由于缺乏直接将当前设备与单独药物治疗进行比较的研究,因此证据存在不确定性.目前正在进行的临床试验正在对此进行评估。这也意味着对于左心室辅助设备是否可以提供目前为英国国家卫生局确定的物有所值的不确定性。
UNASSIGNED: Selected patients with advanced heart failure ineligible for heart transplantation could benefit from left ventricular assist device therapy as \'destination therapy\'. There is evidence of the efficacy of destination therapy; however, it is not currently commissioned within the United Kingdom National Health Service due to the lack of economic evidence.
UNASSIGNED: What is the clinical and cost-effectiveness of a left ventricular assist device compared to medical management for patients with advanced heart failure ineligible for heart transplantation (destination therapy)?
UNASSIGNED: A systematic review of evidence on the clinical and cost-effectiveness of left ventricular assist devices as destination therapy was undertaken including, where feasible, a network meta-analysis to provide an indirect estimate of the relative effectiveness of currently available left ventricular assist devices compared to medical management. For the systematic reviews, data sources searched (up to 11 January 2022) were Cochrane CENTRAL, MEDLINE and EMBASE via Ovid for primary studies, and Epistemonikos and Cochrane Database of Systematic Reviews for relevant systematic reviews. Trial registers were also searched, along with data and reports from intervention-specific registries. Economic studies were identified in EconLit, CEA registry and the NHS Economic Evaluation Database (NHS EED). The searches were supplemented by checking reference lists of included studies. An economic model (Markov) was developed to estimate the cost-effectiveness of left ventricular assist devices compared to medical management from the United Kingdom National Health Service/personal social service perspective. Deterministic and probabilistic sensitivity analyses were conducted to explore uncertainties. Where possible, all analyses focused on the only currently available left ventricular assist device (HeartMate 3TM, Abbott, Chicago, IL, USA) in the United Kingdom.
UNASSIGNED: The clinical effectiveness review included 134 studies (240 articles). There were no studies directly comparing HeartMate 3 and medical management (a randomised trial is ongoing). The currently available left ventricular assist device improves patient survival and reduces stroke rates and complications compared to earlier devices and relative to medical management. For example, survival at 24 months is 77% with the HeartMate 3 device compared to 59% with the HeartMate II (MOMENTUM 3 trial). An indirect comparison demonstrated a reduction in mortality compared to medical management [relative risk of death 0.25 (95% confidence interval 0.13 to 0.47); 24 months; this study]. The cost-effectiveness review included 5 cost analyses and 14 economic evaluations covering different generations of devices and with different perspectives. The reported incremental costs per quality-adjusted life-year gained compared to medical management were lower for later generations of devices [as low as £46,207 (2019 prices; United Kingdom perspective; time horizon at least 5 years)]. The economic evaluation used different approaches to obtain the relative effects of current left ventricular assist devices compared to medical management from the United Kingdom National Health Service/personal social service perspective. All gave similar incremental cost-effectiveness ratios of £53,496-58,244 per quality-adjusted life-year gained - lifetime horizon. Model outputs were sensitive to parameter estimates relating to medical management. The findings did not materially differ on exploratory subgroup analyses based on the severity of heart failure.
UNASSIGNED: There was no direct evidence comparing the clinical effectiveness of HeartMate 3 to medical management. Indirect comparisons made were based on limited data from heterogeneous studies regarding the severity of heart failure (Interagency Registry for Mechanically Assisted Circulatory Support score distribution) and possible for survival only. Furthermore, the cost of medical management of advanced heart failure in the United Kingdom is not clear.
UNASSIGNED: Using cost-effectiveness criteria applied in the United Kingdom, left ventricular assist devices compared to medical management for patients with advanced heart failure ineligible for heart transplant may not be cost-effective. When available, data from the ongoing evaluation of HeartMate 3 compared to medical management can be used to update cost-effectiveness estimates. An audit of the costs of medical management in the United Kingdom is required to further decrease uncertainty in the economic evaluation.
UNASSIGNED: This study is registered as PROSPERO CRD42020158987.
UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: NIHR128996) and is published in full in Health Technology Assessment; Vol. 28, No. 38. See the NIHR Funding and Awards website for further award information.
The majority of patients with advanced heart failure would be unsuitable for heart transplantation due to their age and comorbidities but selected patients could benefit from a left ventricular assist device. Left ventricular assist device therapy for such patients is known as ‘destination therapy’. This is a long-term therapy that involves implanting a battery-powered pump to support the patient’s heart. The purpose of this project was to collect and assess the research evidence on the effectiveness of left ventricular assist devices when used for destination therapy, and to estimate value for money compared to medical management from the United Kingdom National Health Service/personal social service perspective. This research identified that the currently available left ventricular assist device improves patient survival as well as reducing stroke rates and complications compared to earlier devices and relative to medical management. However, there is uncertainty in the evidence due to the absence of studies directly comparing the current device to medical therapy alone. An ongoing clinical trial is currently assessing this. It also means there is uncertainty about whether left ventricular assist devices could provide value for money as determined currently for the United Kingdom National Health Service.