MASSIVE TRANSFUSION EPIDEMIOLOGY AND OUTCOMES IN CHILDREN (MATIC) STUDY

摘要/项目摘要 危及生命的出血发生在儿童中的许多病因，如术中和术后出血， 胃肠道出血、弥散性血管内凝血和创伤性损伤。流行病学， 目前尚未对常用的治疗方法以及与严重出血相关的结局进行系统研究， 这是我们在这一高危人群中知识的一个重大空白。损害控制复苏 (DCR)已经开发出减少成年创伤患者出血死亡的原理。一 DCR中的一个重要概念称为止血复苏，其被定义为高比率的 血浆和血小板与红细胞的比例（> 1：2单位），以及少量血浆不足（红细胞总单位-血浆总单位 单位），两者都在复苏的早期开始。实施止血复苏，大量输血 已经在许多成人和儿科三级护理中心开发了MTP方案。尽管缺乏 支持儿童止血复苏的证据，正在推广到儿科实践， 创伤性和非创伤性病因学。儿童大量输血的流行病学和结局 （MATIC）研究是一项多中心、前瞻性、观察性研究，将收集 孩子们在参与中心。这项研究的目的是确定流行病学，使用的治疗方法， 所有病因需要MTP激活的儿童的结局。我们将前瞻性地收集所有MTP的数据 来自35个学术儿科三级护理中心的约502名患者的连续样本中的激活 在22个月的时间内将其集中到基于网络的数据库中。我们将确定患者的具体因素， 医院间变异性与MTP实践和结果相关。我们的主要假设是 发病率和死亡率结果将因患者疾病类别而异（手术vs创伤vs内科）。我们 还旨在确定早期和持续的止血复苏是否与增加生存率有关， 需要MTP激活的创伤性损伤儿童。我们收集的初步数据表明， 研究的可行性和数据收集方法的有效性。我们还限制了对 止血复苏对创伤性损伤儿童结局的影响，以减少研究中的异质性 人口本研究是创新性的，因为没有儿科多中心数据发表在 流行病学，治疗和儿童在这一人群中的结果。我们的统计分析也是创新的。 我们将开发一个复杂的模型，该模型将倾向分数与时间，比率和赤字相结合 血浆和血小板的RBC，以确定“止血复苏”是否与改善 成果。这项研究的完成将提供初步数据，为大型比较研究提供支持。 有效性研究，以确定哪些DCR原则会影响有或无创伤的儿童的结局 严重出血危及生命。高质量的试验对于改善临床实践至关重要，因为 这一人群的死亡率为35%至78%。

Abstract/Project Summary Life-threatening bleeding occurs from many etiologies in children, such as intra and post-operative bleeding, gastrointestinal bleeding, disseminated intravascular coagulation, and traumatic injury. The epidemiology, therapies commonly used, and outcomes related to severe hemorrhage has not been systematically studied in children, and is a major gap in our knowledge in this high-risk population. Damage Control Resuscitation (DCR) principles have been developed to reduce death from hemorrhage in adult trauma patients. One important concept within DCR is called hemostatic resuscitation, which has been defined as a high ratio of plasma and platelets to RBCs (> 1:2 units), as well as a small plasma deficit (RBC total units - plasma total units), both starting very early in the resuscitation. To implement hemostatic resuscitation, Massive Transfusion protocols (MTP) have been developed in many adult and pediatric tertiary care centers. Despite a paucity of evidence supporting hemostatic resuscitation in children, it is being generalized to pediatric practice for both traumatic and non-traumatic etiologies. The MAssive Transfusion epidemiology and outcomes In Children (MATIC) study is a multicenter, prospective, observational study that will collect data on all MTP activations on children at participating centers. The goal of this study is to determine the epidemiology, therapies used, and outcomes for children requiring MTP activation from all etiologies. We will prospectively collect data on all MTP activations in a consecutive sample of approximately 502 patients from 35 academic pediatric tertiary care centers into a web-based database over a 22-month period. We will determine if patient specific factors and inter-hospital variability are associated with MTP practice and outcomes. Our primary hypothesis is that morbidity and mortality outcomes will vary by patient illness category (operative vs traumatic vs medical). We also aim to determine if early and sustained hemostatic resuscitation is associated with increased survival in children with traumatic injury requiring MTP activation. We have collected preliminary data indicating the feasibility of the study and validity of the data collection methods. We have also limited the analysis of hemostatic resuscitation on outcomes to children with traumatic injury to reduce heterogeneity in the study population. This study is innovative due to the absence of pediatric multicenter data published on the epidemiology, therapies, and outcomes for children in this population. Our statistical analysis is also innovative. We will develop a sophisticated model that will incorporate propensity scores with the timing, ratio, and deficits of plasma and platelets to RBCs to determine if “hemostatic resuscitation” is associated with improved outcomes. Completion of this study will provide preliminary data that will provide support for large comparative effectiveness studies to determine which DCR principles affect outcomes in children with or without traumatic injury with severe life-threatening bleeding. High quality trials are essential to improve clinical practice since the mortality in this population ranges from 35 to 78%.