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循环引起的凝血障碍和血小板减少症 体外膜肺氧合（ECMO）支持的儿童本身就有很大的风险 流血了为了防止出血，儿科重症监护医生经常开预防性血小板 输血然而，在观察性研究中，对ECMO儿童进行预防性血小板输注 与血栓形成、死亡率的增加独立相关， 流血了指导该患者人群进行血小板输注的指南受到缺乏以下因素的限制： 证据，因此仅基于专家意见。考虑到相关的重大风险， 提供证据来指导临床医生。 由BloodNet支持的ECmo儿童止血输血（ECSTATIC）试点试验， 儿科ECMO、体外生命支持组织（ELSO）和儿科急性肺损伤， 脓毒症研究者（PALISI）将在10个研究中心进行。0-18岁的非出血儿童将 按1：1随机分配至血小板输注阈值90（自由输注策略）或50 x109/L （限制性输血策略）。他们将被随访，直到他们进展到严重出血或血栓形成， 从体外膜肺氧合中拔管或存活21天。在本试点中，我们将测试 限制性和自由性输血策略、患者入组的可行性以及 裁定委员会确定出血和血栓形成结局的严重程度。 拟议的试点试验是创新的，因为它的重点是儿童支持的ECMO， 以前从未测试过输血策略的人群;它涉及最大的 过去进行的任何血小板输注试验的两组之间的分离;它涉及两个 新制定的出血和血栓形成定义，特别适用于 体外膜肺氧合 R34试点试验将提供必要和充分的信息，以继续进行确定性研究。 评估限制性预防性血小板输注阈值对 ECMO患儿的临床结局。狂喜有可能降低死亡率和发病率 这些重病的婴儿和儿童。