Comparative Effectiveness of Medications Used in Congenital Heart Surgery

描述（由申请人提供）：本申请涉及挑战领域（04）临床研究和特定挑战主题04- hl -114：使用现有数据集计划儿科心脏病学有效性试验。在接受先天性心脏手术的儿童中使用的大多数治疗方法都没有证据。治疗决定通常基于小型观察性研究、成人数据推断或临床医生经验。我们建议链接两个丰富的数据源：胸外科先天性心脏外科学会（STS-CHS）数据库，其中包含接受心脏手术的儿童的详细临床、诊断、手术和结局数据；儿童健康信息系统（PHIS）数据库，其中包含儿童医院药物使用的详细信息。相关数据将包括来自29个中心的45,000多名患者的信息，这些信息将用于评估围手术期治疗的当前变化，并评估围手术期使用的药物的相对有效性和安全性。该应用程序将重点研究3个治疗领域的药物：手术期间使用的抗纤溶药物，术后环境中的后负荷减少，以及围手术期抗生素预防。我们的总体假设是，对现有大型儿科数据源的链接和分析可以解决许多当前的知识空白，并在3个特定领域提供可靠的数据：1)确定当前的实践模式和先天性心脏手术儿童围手术期护理的变化；2)开展观察性比较有效性研究，提供有关疗效和安全性、样本量、事件发生率和药物效应的数据，用于规划后续试验；4)建立愿意参与后续研究的站点网络。我们的研究团队在分析和链接大规模临床和管理数据集以及开展儿科试验方面拥有丰富的经验，并且非常适合进行拟议的研究。

DESCRIPTION (provided by applicant): This application addresses Challenge Area (04) Clinical Research and specific Challenge Topic 04-HL-114: Using existing datasets to plan effectiveness trials in pediatric cardiology. Most of the treatments used in children undergoing congenital heart surgery are not evidence-based. Treatment decisions are instead usually based on small observational studies, extrapolation from adult data, or clinician experience. We propose to link two rich data sources: The Society of Thoracic Surgeons Congenital Heart Surgery (STS-CHS) Database, which contains detailed clinical, diagnostic, operative, and outcomes data for children undergoing heart surgery, and the Pediatric Health Information System (PHIS) Database, which contains detailed information on medication utilization in children's hospitals. The linked data will include information on over 45,000 patients from 29 centers, which will be utilized to assess current variations in peri-operative treatment, and evaluate comparative effectiveness and safety of medications used in the peri-operative setting. This application will focus on investigating medications in 3 therapeutic areas: anti-fibrinolytic agents used during surgery, afterload reduction in the post-operative setting, and peri-operative antibiotic prophylaxis. Our overall hypothesis is that the linking and analysis of existing large pediatric data sources can address many of these current knowledge gaps and provide robust data in 3 specific areas: 1) identifying current practice patterns and variation in peri-operative care of children undergoing congenital heart surgery; 2) performing observational comparative effectiveness studies which will provide data on efficacy and safety, as well as sample sizes, event rates, and drug effect to be used in planning subsequent trials; 4) establishing a network of sites willing to participate in subsequent studies. Our research team has extensive experience in the analysis and linkage of large-scale clinical and administrative datasets and in conducting pediatric trials, and is uniquely suited to conduct the proposed study. PUBLIC HEALTH RELEVANCE: Congenital heart defects are the most common birth defects and cause a large health burden resulting in >100,00 years of life lost each year and an estimated 6 billion dollars annually in acute care costs alone. However, most medications used to treat children with congenital heart disease have not been tested in them. We propose to link two rich data sources: The Society of Thoracic Surgeons Congenital Heart Surgery Database and the Pediatric Health Information System Database to evaluate the comparative effectiveness of medications used in children undergoing heart surgery and to plan future studies to benefit children with heart disease.