Comparative Effectiveness of N-said Treatment Versus No Treatment for PDA in Pret

N-said 治疗与不治疗对 Pret 患者 PDA 的疗效比较

• 批准号: 8970718
• 负责人: Jonathan Lee Slaughter
• 金额: $ 15.37万
• 依托单位: RESEARCH INST NATIONWIDE CHILDREN'S HOSP
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-12-15 至 2017-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8970718
• 关键词: Acute Kidney Failure; Adverse effects; Age; Anti-Inflammatory Agents; Anti-inflammatory; Benefits and Risks; Birth; Blood Circulation; Blood Vessels; Blood flow; Bronchopulmonary Dysplasia; Cerebrum; Cessation of life; Childhood; Chronic lung disease; Clinical; Clinical Treatment; Clinical Trials; Clinical Trials Design; Communities; Complication; Data; Data Analyses; Data Collection; Data Sources; Databases; Development; Diagnosis; Diagnostic; Disease; Doctor of Philosophy; Drug Utilization; Ductal; Ductus Arteriosus; Epidemiologist; Future; Goals; Health; Health Information System; Hospitals; Indomethacin; Infant; Inpatients; Institution; Intestinal Perforation; Investigation; Knowledge; Left; Length; Link; Lung; Measures; Mechanical ventilation; Mentors; Mentorship; Mesentery; Methods; Modeling; Natural experiment; Necrotizing Enterocolitis; Neonatal; Observational Study; Ohio; Outcome; Oxygen Therapy Care; Patent Ductus Arteriosus; Patient Care; Patient-Focused Outcomes; Patients; Pediatric Hospitals; Perinatal; Pharmaceutical Preparations; Pharmacotherapy; Positioning Attribute; Pregnancy; Premature Infant; Randomized Controlled Trials; Renal Blood Flow; Research; Research Personnel; Severity of illness; Site; Surveys; Techniques; Time; Training; Universities; Variant; administrative database; base; career; career development; comparative effectiveness; effectiveness research; fetal; fetal blood; improved; improved outcome; infant outcome; longitudinal analysis; mortality; neonatal care; neonate; premature; professor; prospective; respiratory; skills; treatment trial

DESCRIPTION (provided by applicant): Jonathan L. Slaughter, MD, MPH is an Assistant Professor in the Center for Perinatal Research at Nationwide Children's Hospital and The Ohio State University. His career goal is to improve neonatal care through rigorous comparative effectiveness research (CER) that will evaluate the risks and benefits of medications that, despite limited evidence, are commonly used in the treatment of prematurity-related disorders. The proposed four-year K08 mentored career development proposal will provide Dr. Slaughter with training in CER through 1) mentorship and didactic training in longitudinal analysis of large inpatient databases 2) training in advanced statistical techniques to investigate causal inference in observational data and 3) the use of database linkages to increase the robustness of outcome estimates. Additionally, he will gain a basic knowledge of clinical trial design, an important research skill for CER investigators. The objective of Dr. Slaughter's proposed research investigation is to utilize "real-world" patient data from multiple sites to determine the comparative effectiveness of non-steroidal anti-inflammatory drug (NSAID) treatment for patent ductus arteriosus (PDA) versus no treatment on mortality, development of bronchopulmonary dysplasia (BPD), and duration of respiratory support. The presence of a PDA in preterm infants is associated with increased mortality and higher rates of BPD. Ductal closure by administration of NSAIDs aims to reduce these complications. However, NSAIDs are not without side effects and evidence for their benefit in improving important patient-centered outcomes is lacking. Likely due to this absence of clear evidence, there is marked between-hospital practice variation for NSAID treatment of PDA. The primary data source will be the Pediatric Health Information System (PHIS), an administrative database inclusive of most large, U.S. children's hospitals that contains daily, patient-specific data on prescribed medications and respiratory support. The specific aims of the research plan are 1) to determine factors associated with between-hospital variation in NSAID treatment for PDA, 2) determine the comparative effectiveness of various NSAID treatments versus no treatment for PDA on mortality and respiratory outcomes, and 3) to validate Aim 1 and Aim 2 results within the subset of study infants represented in both PHIS and the Children's Hospital Neonatal Database (CHND). CHND is a smaller, clinical survey database with additional and more detailed, patient-specific diagnostic and procedural data. Two internationally renowned epidemiologists, Mark Klebanoff, MD, MPH and Thomas Newman MD, MPH, and Patricia Reagan, PhD, an economist with expertise in both longitudinal data analysis and econometric causal inference methods, will serve as mentors. Other consultants will provide cardiologic expertise (Vidu Garg, MD) and assist with merger of the PHIS and CHND databases (Matt Hall, PhD). In addition to preparing Dr. Slaughter to become an independent expert in neonatal comparative effectiveness research, we expect our findings to inform patient care for preterm infants with PDA and guide the development of prospective investigations.

描述（由申请人提供）：Jonathan L. Slaughter，医学博士、公共卫生硕士，是全国儿童医院和俄亥俄州立大学围产期研究中心的助理教授。他的职业目标是通过严格的比较有效性研究 (CER) 来改善新生儿护理，该研究将评估药物的风险和益处，尽管证据有限，但这些药物通常用于治疗早产相关疾病。拟议的为期四年的 K08 指导职业发展提案将为 Slaughter 博士提供 CER 培训，具体方式包括：1）对大型住院患者数据库进行纵向分析的指导和教学培训；2）高级统计技术培训，以调查观察数据中的因果推理；3）使用数据库链接来提高结果估计的稳健性。此外，他还将获得临床试验设计的基本知识，这是 CER 研究人员的一项重要研究技能。斯劳特博士提出的研究调查的目的是利用来自多个地点的“真实世界”患者数据来确定 非甾体抗炎药 (NSAID) 治疗动脉导管未闭 (PDA) 与不治疗对死亡率、支气管肺发育不良 (BPD) 发展和呼吸支持持续时间的疗效比较。早产儿 PDA 的存在与死亡率增加和 BPD 发生率升高相关。通过使用非甾体抗炎药封闭导管旨在减少这些并发症。然而，非甾体抗炎药并非没有副作用，而且缺乏证据表明它们有助于改善以患者为中心的重要结果。可能由于缺乏明确的证据，不同医院间非甾体抗炎药治疗 PDA 的做法存在明显差异。主要数据源将是儿科健康信息系统 (PHIS)，这是一个包含美国大多数大型儿童医院的管理数据库，其中包含有关处方药物和呼吸支持的每日患者特定数据。该研究计划的具体目标是 1) 确定与 NSAID 治疗 PDA 的医院间差异相关的因素，2) 确定各种 NSAID 治疗与不治疗 PDA 在死亡率和呼吸系统结局方面的比较效果，以及 3) 在 PHIS 和儿童医院新生儿数据库 (CHND) 中代表的研究婴儿子集中验证目标 1 和目标 2 结果。 CHND 是一个较小的临床调查数据库，包含额外且更详细的患者特定诊断和程序数据。两位国际知名流行病学家 Mark Klebanoff（医学博士、公共卫生硕士）和 Thomas Newman（医学博士、公共卫生硕士）以及帕特里夏·里根（Patricia Reagan）博士（一位精通纵向数据分析和计量经济学因果推理方法的经济学家）将担任导师。其他顾问将提供心脏病学专业知识（Vidu Garg，医学博士）并协助 PHIS 和 CHND 数据库的合并（Matt Hall，博士）。除了帮助 Slaughter 博士成为新生儿比较有效性研究的独立专家之外，我们希望我们的研究结果能为 PDA 早产儿的患者护理提供信息，并指导前瞻性研究的开展。