Oliva Fabrizio, Comin-Colet Josep, Fedele Francesco, Fruhwald Friedrich, Gustafsson Finn, Kivikko Matti, Borbély Attila, Pölzl Gerhard, Tschöpe Carsten
ASST Grande Ospedale Metropolitano Niguarda - Cardiologia 1, Milan, Italy.
Heart Failure Program, Department of Cardiology, Bellvitge University Hospital and Bellvitge Biomedical Research Institute (IDIBELL), University of Barcelona, Hospitalet de Llobregat (Barcelona), Barcelona, Spain.
Eur Heart J Suppl. 2018 Dec;20(Suppl I):I11-I20. doi: 10.1093/eurheartj/suy040. Epub 2018 Nov 2.
Inotropes may be an appropriate treatment for patients with advanced heart failure (AdHF) who remain highly symptomatic despite optimized standard therapies. Objectives for inotrope use in these situations include relief of symptoms and improvement of quality of life, and reduction in unplanned hospitalizations and the costs associated with such episodes. All of these goals must be attained without compromising survival. Encouraging findings with intermittent cycles of intravenous levosimendan have emerged from a range of exploratory studies and from three larger controlled trials (LevoRep, LION-HEART, and LAICA) which offered some evidence of clinical advantage. In these settings, however, obtaining statistically robust data may prove elusive due to the difficulties of endpoint assessment in a complex medical condition with varying presentation and trajectory. Adoption of a composite clinical endpoint evaluated in a hierarchical manner may offer a workable solution to this problem. Such an instrument can explore the proposition that repetitive administration of levosimendan early in the period after discharge from an acute episode of worsening heart failure may be associated with greater subsequent clinical stability vis-à-vis standard therapy. The use of this methodology to develop a 'stability score' for each patient means that all participants in such a trial contribute to the overall outcome analysis through one or more of the hierarchical endpoints; this has helpful practical implications for the number of patients needed and the length of follow-up required to generate endpoint data. The LeoDOR study (NCT03437226), outlined in this review, has been designed to explore this new approach to outcome assessment in AdHF.
对于尽管接受了优化的标准治疗但仍有高度症状的晚期心力衰竭(AdHF)患者,正性肌力药物可能是一种合适的治疗方法。在这些情况下使用正性肌力药物的目标包括缓解症状、改善生活质量、减少计划外住院以及与此类发作相关的费用。所有这些目标都必须在不影响生存的情况下实现。一系列探索性研究以及三项较大的对照试验(LevoRep、LION-HEART和LAICA)得出了关于静脉注射左西孟旦间歇性给药的令人鼓舞的结果,这些试验提供了一些临床优势的证据。然而,在这些情况下,由于在表现和病程各异的复杂医疗状况中进行终点评估存在困难,可能难以获得统计学上可靠的数据。采用以分层方式评估的综合临床终点可能为这个问题提供一个可行的解决方案。这样一种工具可以探讨这样一个命题,即在心衰恶化急性发作出院后的早期重复给予左西孟旦可能比标准治疗带来更大的后续临床稳定性。使用这种方法为每个患者制定一个“稳定性评分”意味着此类试验中的所有参与者都通过一个或多个分层终点为总体结果分析做出贡献;这对于生成终点数据所需的患者数量和随访时间具有有益的实际意义。本综述中概述的LeoDOR研究(NCT03437226)旨在探索这种评估AdHF结局的新方法。