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.

项目概要 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 就其本身而言，接受体外膜肺氧合（ECMO）支持的儿童面临着巨大的风险 流血。为了防止出血，儿科重症医生经常开预防性血小板 输血。然而，在观察性研究中，对使用 ECMO 的儿童进行预防性血小板输注 与血栓形成和死亡率增加独立相关，并且矛盾的是， 流血。由于缺乏相关资料，在该患者群体中直接输注血小板的指南受到限制。 证据，因此仅基于专家意见。考虑到重大的相关风险，至关重要 为指导临床医生提供证据。 ECmo 儿童止血输血 (ECSTATIC) 试点试验得到 BloodNet 的认可， PediECMO、体外生命支持组织 (ELSO) 和小儿急性肺损伤和 败血症调查员 (PALISI) 将在十个地点进行。 0-18岁不出血的儿童会 按 1:1 随机分配至血小板输注阈值 90（自由输血策略）或 50 x109/L （限制性输血策略）。他们将被跟踪，直到他们进展为严重出血或血栓形成， 他们从 ECMO 中拔管或达到 21 天。在此试点中，我们将测试之间的分离 限制性和自由性输血策略、患者入组的可行性以及 裁决委员会确定出血的严重程度和血栓形成的结果。 拟议的试点试验具有创新性，因为它重点关注 ECMO 支持的儿童。 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；这涉及到两个 新制定的出血和血栓形成定义特别适用于由以下人员支持的儿童 ECMO。 R34 试点试验将为进行最终决定提供必要且充分的信息 ECSTATIC RCT 评估限制性预防性血小板输注阈值对 ECMO 儿童的临床结果。 ECSTATIC 有降低死亡率和发病率的潜力 这些病情严重的婴儿和儿童。