Validation of a pediatric thrombosis risk assessment model and characterization of bleeding in hospitalized children through the Children's Hospital Acquired Thrombosis (CHAT) Consortium

项目摘要/摘要 静脉血栓栓塞症(VTE)是一种严重的危及生命的疾病，其发病率在 儿科住院患者减少70%-200%。这导致了发病率、死亡率和更长的时间 住院治疗，被认为是由于更有效地照顾患有严重和危及生命的儿童 精神错乱。不幸的是，目前还不清楚哪些儿童患医院获得性(HA)VTE的风险最高 由于许多研究都是单中心、病例数量少，如何预防这些事件。本研究 解决使用风险评估模型识别HA-VTE高危儿童的迫切需要 (公羊)，但出血的风险很低。这一队列将被用作评估 为实现HA-VTE的长期目标预防性抗凝的有效性和安全性 儿童的预防。本研究的总体目标是：(1)前瞻性地验证儿科HA-VTE 由Jaffray博士的多中心儿童医院获得性血栓形成(CHAT)联盟创建的RAM 使用病例对照研究设计和电子病历审查(EMR)；(2)评估发病率和 通过EMR复习确定住院儿童抗凝前后出血的特征；以及 通过组织焦点小组和半 使用定性主题分析策略对住院医疗队进行结构化访谈。要实现 这些研究的目的是，Jaffray博士的直接职业目标是获得新的知识、技能和 使她能够(1)分析来自多中心研究的大数据集的数据，并构建和 通过多元分析验证风险模型；(2)培养定性研究技能，以设计和分析 焦点小组和半结构化访谈；以及(3)获得计划和进行随机调查的知识 对照试验。实现这些职业目标将支持她成为一名 独立资助，在确定有效和安全的儿童VTE预防策略方面发挥带头作用 通过多中心随机对照试验。Jaffray博士将通过以下方式实现她的研究和培训目标 在她的主要导师盖伊·杨博士(儿科血栓临床研究)和共同导师的支持下， Neil Goldenberg博士(儿科临床试验研究)、Ernest Amankwah博士(生物统计学)、Neil Zakai博士(成人 血栓形成临床研究)和伊丽莎白·伯纳博士(定性研究)。杰弗里博士的职业规划 洛杉矶儿童医院出色的研究环境和 南加州大学，为初级教职员工提供大量的教学和培训 专门为促进向研究职业独立过渡而设计的机会。通过完成 她的研究和培训目标，Jaffray博士将采取必要的第一步来识别高危儿童 HA-VTE可以安全地从预防策略中受益，从而减少儿科 VTE。

PROJECT SUMMARY/ABSTRACT Venous thromboembolism (VTE), a serious and life-threatening condition, has increased in incidence for hospitalized pediatric patients by 70-200%. This has led to increased morbidity, mortality and longer hospitalizations, and is thought to be due to more effective care of children with serious and life-threatening disorders. Unfortunately, it is unclear which children are at the highest risk of hospital acquired (HA) VTE and how to prevent these events due to many studies being single center with small case numbers. This study addresses the critical need to identify children at a high risk of HA-VTE using a risk assessment model (RAM), but a low risk of bleeding. This cohort will be used as the target population for trials evaluating the efficacy and safety of prophylactic anticoagulation in order to progress towards the long term goal of HA-VTE prevention in children. The overall objectives of this study are to (1) prospectively validate a pediatric HA-VTE RAM created through Dr. Jaffray’s multi-center Children’s Hospital Acquired Thrombosis (CHAT) Consortium using a case-control study design and electronic medical record review (EMR); (2) evaluate the incidence and characterization of bleeding in hospitalized children on and off anticoagulation through EMR review; and (3) explore barriers to provider engagement with RAM implementation by conducting focus groups and semi- structured interviews of inpatient medical teams using qualitative thematic analysis strategies. To achieve these research aims, Dr. Jaffray has the immediate career goals to obtain new knowledge, skills, and experience that will allow her to (1) analyze data from large datasets from multicenter studies and build and validate risk models through multivariate analysis; (2) develop qualitative research skills to design and analyze focus groups and semi-structured interviews; and (3) acquire the knowledge to plan and conduct randomized controlled trials. Achieving these career goals will support her long term career goal of becoming an independently funded, leader in the effort to determine efficacious and safe VTE prevention strategies in children through multicenter randomized controlled trials. Dr. Jaffray will accomplish her research and training aims with the support of her primary mentor, Dr. Guy Young (pediatric thrombosis clinical research), and co-mentors, Dr. Neil Goldenberg (pediatric clinical trial research), Dr. Ernest Amankwah (biostatics), Dr. Neil Zakai (adult thrombosis clinical research) and Dr. Elizabeth Burner (qualitative research). Dr. Jaffray’s plan for career development is enhanced by the outstanding research environment at Children’s Hospital Los Angeles and The University of Southern California, which provides junior faculty members with numerous didactic and training opportunities designed specifically to facilitate the transition to research career independence. By completing her research and training aims, Dr. Jaffray will take the first necessary step in identifying children at high risk of HA- VTE who may safely benefit from prevention strategies and subsequently reduce the incidence of pediatric VTE.