Corticosteroids for Acute Exacerbations of Idiopathic Pulmonary Fibrosis: Patterns and Outcomes

项目总结 在题为“糖皮质激素在特发性肺纤维化急性加重中的应用：实践”的提案中 和结果“我将决定目前与皮质类固醇治疗相关的实践模式和结果 用于重症患者特发性肺纤维化的急性加重。每年，10%-20%的患者 特发性肺纤维化(IPF)是一种进行性的纤维化性肺部疾病，急性发作 加重(AE-IPF)，通常导致入院和危重疾病。这些急性呼吸道疾病 病情恶化导致IPF患者的高发病率和死亡率；发展为危重疾病的患者 住院死亡率为20-35%，病情加重后中位生存期为3-4个月。皮质类固醇 是基于薄弱证据的AE-IPF患者的主要治疗方法，指南没有制定 关于皮质类固醇的剂量、路线、时机和持续时间的建议。由于缺乏明确的指导方针 和证据，使用模式可能是高度可变的。专家们一致认为需要新的证据来指导 AE-IPF管理。在这个提案中，我将通过1)描述实践来填补这些巨大的知识空白 皮质类固醇剂量、途径、启动时机和持续时间在一个大型的全国性队列中的模式 2)使用比较有效性确定与皮质类固醇给药策略相关的结果 方法论。这项建议的完成将有助于为AE-IPF管理的未来研究提供参考，并建立 最佳实践的证据基础。除了完成本课程提供的直接体验式学习之外 提议，我将参加整个奖项的教学、科学研讨会和研究会议。 期间，为了提高我在临床研究设计和方法学方面的能力。此外，我将获得 使用大型数据库进行数据收集和统计分析所需的技能。有了直接 我的赞助商的指导，波士顿大学医学院肺中心的支持环境 中心和专业发展课程，我将成功地将这项提议的结果纳入一个 在职业发展奖中，我将专注于在AE-IPF中实施皮质类固醇使用的最佳实践。 这项提议直接与国家心肺和血液研究所的目标保持一致 包括优化临床研究以改善健康和减少疾病，并利用 数据科学开辟了心、肺、血液和睡眠研究的新领域。这笔研究经费将是 在两名具有重症监护专业知识的卫生服务调查员的直接指导下进行 流行病学和结果研究以及在比较有效性方法论的建议下 特发性肺纤维化专家和两名专家。

PROJECT SUMMARY In the proposal entitled, “Corticosteroids in Acute Exacerbations of Idiopathic Pulmonary Fibrosis: Practices and Outcomes” I will determine current practice patterns and outcomes associated with corticosteroid therapy for acute exacerbations of idiopathic pulmonary fibrosis in critically ill patients. Every year, 10-20% of patients with idiopathic pulmonary fibrosis (IPF) – a progressive, fibrosing lung disease – experience acute exacerbations (AE-IPF), often leading to hospital admission and critical illness. These acute respiratory deteriorations result in high morbidity and mortality for IPF patients; patients who develop critical illness have in-hospital mortality rates of 20-35% and a median survival post-exacerbation of 3-4 months. Corticosteroids are a mainstay of therapy for patients with AE-IPF based on weak evidence, and guidelines do not make recommendations as to dosing, route, timing, and duration of corticosteroids. Given lack of clear guidelines and evidence, use patterns are likely highly variable. Expert consensus has called for new evidence to guide AE-IPF management. In this proposal, I will fill these large knowledge gaps by 1) characterizing practice patterns of corticosteroid dosing, route, initiation timing, and duration for AE-IPF in a large, national cohort and 2) determining outcomes associated with dosing strategies for corticosteroids using comparative effectiveness methodology. Completion of this proposal will help inform future studies for AE-IPF management and build an evidence base for best practices. In addition to the direct experiential learning provided by completion of this proposal, I will partake in didactics, scientific seminars, and research conferences throughout this award period, in order to advance my competency in clinical research design and methodology. Further, I will obtain the skills necessary for data collection and statistical analysis using large databases. With the direct mentorship of my sponsors, the supportive environment of The Pulmonary Center at Boston University Medical Center, and professional development courses, I will successfully incorporate the results of this proposal into a Career Development Award where I will focus on implementing best practices for corticosteroid use in AE-IPF. This proposal is directly aligned with the goals of the National Heart, Lung, and Blood Institute objectives which include optimizing clinical research to improve health and reduce disease and leveraging new opportunities in data science to open new frontiers in heart, lung, blood, and sleep research. This research grant will be performed under the direct mentorship of two health services investigators with expertise in critical care epidemiology and outcomes research and under the advice of a comparative effectiveness methodological expert and two experts in idiopathic pulmonary fibrosis.