Department of Cardiology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Paediatric Intensive Care Unit, Bambino Gesù Children's Hospital, Scientific Institute for Research, Hospitalization and Healthcare, Rome, Italy.
Lancet Child Adolesc Health. 2024 Oct;8(10):773-780. doi: 10.1016/S2352-4642(24)00132-9.
Extracorporeal membrane oxygenation (ECMO) is a high-risk and low-volume life support with increasing clinical study. However, heterogenous outcome definitions impede data assimilation into evidence to guide practice. The Academic Research Consortium (ARC), an international collaborative forum committed to facilitating the creation of stakeholder-driven consensus nomenclature and outcomes for clinical trials of medical devices, supported the ECMO Core Elements Needed for Trials Regulation And quality of Life (ECMO-CENTRAL) ARC. The ECMO-CENTRAL ARC was assembled to develop definitions of paediatric ECMO adverse events for use in clinical trials and regulatory device evaluation. An initial candidate list of ECMO adverse events derived from the mechanical circulatory support ARC was supplemented with a review of ECMO-relevant adverse event definitions collated from literature published between Jan 1, 1988, and Feb 20, 2023. Distinct teams of international topic experts drafted separate adverse event definitions that were harmonised to existing literature when appropriate. Draft definitions were revised for paediatric ECMO relevance with input from patients, families, and an international expert panel of trialists, clinicians, statisticians, biomedical engineers, device developers, and regulatory agencies. ECMO-CENTRAL ARC was revised and disseminated across research societies and professional organisations. Up to three rounds of internet-based anonymous surveys were planned as a modified Delphi process. The expert panel defined 13 adverse event definitions: neurological, bleeding, device malfunction, acute kidney injury, haemolysis, infection, vascular access-associated injury, non-CNS thrombosis, hepatic dysfunction, right heart failure, left ventricular overload, lactic acidaemia, and hypoxaemia. Definitional structure varied. Among 165 expert panel members, 114 were eligible to vote and 111 voted. Consensus was achieved for all proposed definitions. Agreement ranged from 82% to 95%. ECMO-CENTRAL ARC paired rigorous development with methodical stakeholder involvement and dissemination to define paediatric ECMO adverse events. These definitions will facilitate new research and the assimilation of data across clinical trials and ECMO device evaluation in children.
体外膜肺氧合(ECMO)是一种高风险、低容量的生命支持方法,越来越多的临床研究正在开展。然而,异质的结局定义阻碍了数据整合到指导实践的证据中。学术研究联盟(ARC)是一个国际合作论坛,致力于促进为医疗器械临床试验制定由利益相关者驱动的共识命名法和结局,该联盟支持体外膜肺氧合核心要素临床试验监管和生活质量(ECMO-CENTRAL)ARC。ECMO-CENTRAL ARC 成立的目的是为儿科 ECMO 不良事件制定临床试验和监管设备评估用定义。最初从机械循环支持 ARC 中得出的 ECMO 不良事件候选清单,通过对 1988 年 1 月 1 日至 2023 年 2 月 20 日期间发表的文献中收集的与 ECMO 相关的不良事件定义进行回顾,进行了补充。国际专题专家小组分别起草了不良事件定义,在适当情况下,与现有文献进行了协调。在患者、家庭和国际试验专家小组、临床医生、统计学家、生物医学工程师、设备开发商和监管机构的共同参与下,根据儿科 ECMO 的相关性对定义进行了修订。对儿童 ECMO 相关性进行了修订,并在研究协会和专业组织中进行了传播。作为一项修改后的 Delphi 流程,计划进行多达三轮基于互联网的匿名调查。专家组定义了 13 种不良事件定义:神经、出血、设备故障、急性肾损伤、溶血、感染、血管通路相关损伤、非中枢神经系统血栓形成、肝功能障碍、右心衰竭、左心室过载、乳酸酸中毒和低氧血症。定义结构各不相同。在 165 名专家组成员中,有 114 名符合投票资格,其中 111 名投票。所有提议的定义均达成共识。同意率为 82%至 95%。ECMO-CENTRAL ARC 通过严格的开发、系统的利益相关者参与和传播,定义了儿科 ECMO 不良事件。这些定义将促进新的研究,并在儿童 ECMO 临床试验和设备评估中整合数据。