ECmo hemoSTAtic Transfusions In Children (ECSTATIC)

儿童 ECmo 止血输血 (ECSTATIC)

• 批准号: 10527446
• 负责人: Oliver L Karam
• 金额: $ 29.93万
• 依托单位: YALE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-09-01 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10527446
• 关键词: 18 year old; Benefits and Risks; Blinded; Blood Coagulation Disorders; Blood Platelets; Cannulations; Child; Child Support; Childhood; Clinical; Clinical Trials; Coagulation Process; Consent; Critically ill children; Data; Enrollment; Equilibrium; Etiology; Expert Opinion; Extracorporeal Membrane Oxygenation; Frequencies; Future; Goals; Guidelines; Hemodilution; Hemorrhage; Hemostatic Agents; Infant; Inflammatory; Informed Consent; Life; Methods; Morbidity - disease rate; Nature; Observational Study; Outcome; Patients; Pediatric Acute Lung Injury; Pilot Projects; Platelet Count measurement; Platelet Transfusion; Population; Premature Infant; Process; Publishing; Randomized; Randomized Controlled Trials; Reporting; Research Personnel; Risk; Running; Sepsis; Severities; Site; Testing; Thrombocytopenia; Thrombosis; Transfusion; adjudicate; adjudication; arm; base; cohort; design; improved outcome; innovation; mortality; open label; participant enrollment; patient population; pediatric patients; pilot trial; prevent; programs; prophylactic; randomized controlled design; thrombotic; thrombotic complications; treatment arm; trial comparing

PROJECT SUMMARY Due to coagulopathy and thrombocytopenia induced by hemodilution and the extracorporeal circuit itself, children supported by extracorporeal membrane oxygenation (ECMO) are at significant risk of bleeding. In order to prevent bleeding, pediatric intensivists often prescribe prophylactic platelet transfusions. However, in observational studies, prophylactic platelet transfusions to children on ECMO have been independently associated with increased thrombosis, mortality, and paradoxically, increased bleeding. Guidelines to direct platelet transfusions in this patient population are limited by the lack of evidence and therefore based on expert opinion alone. Given the significant associated risks, it is crucial to provide evidence to guide clinicians. The ECmo hemoSTAtic Transfusions In Children (ECSTATIC) pilot trial, endorsed by BloodNet, PediECMO, the Extracorporeal Life Support Organization (ELSO) and the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI), will be conducted in ten sites. Non-bleeding children 0-18 years of age will be randomized 1:1 to either a platelet transfusion threshold of 90 (liberal transfusion strategy) or 50 x109/L (restrictive transfusion strategy). They will be followed until they progress to severe bleeding or thrombosis, they are decannulated from ECMO or they reach 21 days. In this pilot, we will test the separation between the restrictive and liberal transfusion strategies, the feasibility of patient enrollment, and the ability of an adjudication committee to determine the severity of bleeding and thrombotic outcomes. The proposed pilot trial is innovative in that it is focused on children supported by ECMO, a population in whom transfusion strategies have never been tested previously; it involves the largest separation between the two arms of any platelet transfusion trial conducted in the past; and it involves two newly developed definitions of bleeding and thrombosis particularly applicable to children supported by ECMO. The R34 pilot trial will provide necessary and sufficient information to proceed with the definitive ECSTATIC RCT to evaluate the impact of a restrictive prophylactic platelet transfusion threshold on the clinical outcomes in children on ECMO. ECSTATIC has the potential to decrease mortality and morbidity of these extremely ill infants and children.

项目摘要 由于血液稀释和体外回路引起的凝血病和血小板减少症 本身，受体外膜氧合（ECMO）支持的儿童有重大风险 流血。为了防止出血，小儿浓缩主义者经常开处方预防性血小板 输血。然而，在观察性研究中，对ECMO的儿童进行预防性血小板输血 与血栓形成，死亡率和自相矛盾的增加有关 流血。该患者人群中直接血小板输血的指南受到限制 证据，因此仅基于专家意见。考虑到显着的相关风险，这是至关重要的 提供指导临床医生的证据。 由血液认可的儿童（狂喜）试验试验中的ECMO止血性输血， Pediecmo，体外生命支持组织（ELSO）和小儿急性肺损伤和 败血症调查人员（PALISI）将在十个地点进行。非流血儿童0-18岁将 将1：1随机分为90（自由输血策略）或50 x109/l的血小板输血阈值 （限制输血策略）。他们将被遵循，直到他们发展为严重的出血或血栓形成为止， 它们从ECMO中脱颖而出，或者达到21天。在此飞行员中，我们将测试 限制性和自由输血策略，患者入学的可行性以及 裁决委员会确定出血和血栓形成结果的严重程度。 拟议的试点试验具有创新性，因为它专注于Ecmo支持的儿童， 以前从未对输血策略进行过测试的人口；它涉及最大的 过去进行的任何血小板输血试验的两个臂之间的分离；这涉及两个 新开发的关于出血和血栓形成的定义，特别适用于支持儿童 ECMO。 R34试点试验将提供必要的足够信息，以进行确定性 狂喜的RCT评估限制性预防性血小板输血阈值对 ECMO儿童的临床结果。狂喜有可能降低死亡率和发病率 在这些生病的婴儿和儿童中。