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

通过儿童医院获得性血栓 (CHAT) 联盟验证儿科血栓风险评估模型和住院儿童出血特征

• 批准号: 10730124
• 负责人: Julie Ann Jaffray
• 金额: $ 17.11万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-08-01 至 2027-07-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10730124
• 关键词: Validation; pediatric; thrombosis; risk; assessment

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.

项目概要/摘要 静脉血栓栓塞 (VTE) 是一种严重且危及生命的疾病，其发病率有所增加 住院儿童患者减少了 70-200%。这导致了发病率、死亡率的增加和更长的时间 住院治疗，被认为是由于对患有严重和危及生命的儿童进行了更有效的护理 失调。不幸的是，目前尚不清楚哪些儿童发生医院获得性 (HA) VTE 的风险最高， 由于许多研究都是单中心、病例数少，如何预防这些事件。这项研究 满足使用风险评估模型识别 HA-VTE 高风险儿童的迫切需求 （RAM），但出血风险较低。该队列将用作评估试验的目标人群 预防性抗凝的有效性和安全性，以实现 HA-VTE 的长期目标 儿童预防。本研究的总体目标是 (1) 前瞻性验证儿科 HA-VTE RAM 通过 Jaffray 博士的多中心儿童医院获得性血栓 (CHAT) 联盟创建 使用病例对照研究设计和电子病历审查（EMR）； (2) 评估发生率 通过电子病历 (EMR) 审查了解住院儿童接受和停止抗凝治疗时的出血特征；和（3） 通过开展焦点小组和半专题讨论，探索供应商参与 RAM 实施的障碍 使用定性主题分析策略对住院医疗团队进行结构化访谈。达到 这些研究目标，贾弗雷博士的近期职业目标是获得新的知识、技能和 经验将使她能够（1）分析来自多中心研究的大型数据集的数据，并构建和 通过多变量分析验证风险模型； (2) 培养设计和分析的定性研究技能 焦点小组和半结构化访谈； (3) 获得计划和实施随机化的知识 对照试验。实现这些职业目标将支持她成为一名 独立资助，在确定有效且安全的儿童 VTE 预防策略方面处于领先地位 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： 她的主要导师 Guy Young 博士（儿科血栓临床研究）和共同导师的支持， 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 Elizabeth Burner 博士（定性研究）。 Jaffray 博士的职业规划 洛杉矶儿童医院和 The Children’s Hospital 出色的研究环境促进了发展 南加州大学，为初级教师提供大量的教学和培训 专门为促进研究职业独立过渡而设计的机会。通过完成 根据她的研究和培训目标，贾弗雷博士将采取必要的第一步来识别高风险儿童 HA-VTE 可以安全地受益于预防策略，从而减少儿科的发生率 静脉血栓栓塞。