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

项目摘要 在题为“皮质类固醇在特发性肺纤维化急性加重中的应用： 和结果”我将确定目前的实践模式和结果与皮质类固醇治疗 用于重症患者特发性肺纤维化急性加重。每年，10-20%的患者 特发性肺纤维化（IPF）-一种进行性纤维化肺病-急性 急性加重（AE-IPF），通常导致住院和危重疾病。这些急性呼吸道疾病 恶化导致IPF患者的高发病率和死亡率;发生危重疾病的患者 住院死亡率为20-35%，加重后中位生存期为3-4个月。皮质类固醇 是AE-IPF患者的主要治疗方法，但证据不足，指南未做出 关于皮质类固醇的剂量、途径、时间和持续时间的建议。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。专家共识呼吁新证据来指导 AE-IPF管理。在这个建议中，我将通过以下方式填补这些巨大的知识空白：1）描述实践 一个大型国家队列中AE-IPF的皮质类固醇给药模式、给药途径、开始时间和持续时间，以及 2)使用比较有效性确定与皮质类固醇给药策略相关的结局 方法论本提案的完成将有助于为AE-IPF管理的未来研究提供信息， 最佳做法的证据基础。除了完成本课程所提供的直接体验式学习之外， 根据我的提议，我将在整个获奖期间参加教学法、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。与直接 我的赞助商的指导，波士顿大学医学中心肺中心的支持性环境 中心，和专业发展课程，我将成功地将这一建议的结果纳入一个 职业发展奖，我将专注于在AE-IPF中实施皮质类固醇使用的最佳实践。 该提案与国家心脏、肺和血液研究所的目标直接一致， 包括优化临床研究，以改善健康和减少疾病，并利用新的机会， 数据科学将在心脏、肺、血液和睡眠研究领域开辟新的前沿。这项研究经费将 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，并根据比较有效性方法的建议， 专家和两名特发性肺纤维化专家。

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.