Factors associated with corticosteroid therapy in idiopathic pulmonary fibrosis

描述(由申请人提供)：特发性肺纤维化(IPF)是特发性间质性肺炎最常见的形式，与确诊后中位生存期约5年有关。可以使用几种药物来治疗这种疾病，包括皮质类固醇和其他免疫抑制剂，尽管它们的疗效尚不清楚。此外，这些药物，特别是皮质类固醇，在其他人群中也与显著的不良反应有关。目前的指南可以帮助指导临床医生，但不知道是否遵循了这些指南，最常用的药物是什么，或者哪些因素影响了在诊断时开始治疗的决定或处方药物的选择。这项应用的广泛、长期目标是解决这些不确定的领域，因此更好地确定如何改进治疗策略，以最大限度地减少患者暴露于现有治疗的潜在毒性。因此，拟议的队列研究将集中于以下三个具体目标：1)在广泛的临床实践中量化皮质类固醇和非皮质类固醇类药物的使用以及给IPF患者开出的药物的剂量；2)确定哪些疾病和人口因素与IPF诊断时开始皮质类固醇治疗有关；以及3)确定哪些疾病和人口因素与开始使用皮质类固醇和非皮质类固醇类药物联合治疗与单独使用皮质类固醇相比较。这项拟议的研究将使用来自英国的完全匿名的医疗记录研究数据库来评估大约2100名IPF患者的队列。这将是此类疾病中规模最大的研究。从这个数据库中，将进行一项回溯性队列研究，并使用双变量和多变量分析来评估患者特定的暴露变量与皮质类固醇或联合治疗的使用之间的关联。相关性：这项研究旨在了解哪些因素影响医生在特发性肺纤维化治疗中的决策。特发性肺纤维化是一种进行性和致命性的肺部疾病，治疗通常无效，并与显著的副作用相关。这些结果将为进一步研究量化治疗的风险以及如何更有效地平衡这些风险与治疗的边际效益奠定基础。

DESCRIPTION (provided by applicant): Idiopathic Pulmonary Fibrosis (IPF) is the most common form of Idiopathic Interstitial Pneumonia and is associated with a median survival of approximately 5 years from the time of diagnosis. Several medications may be used for this disease, including corticosteroids and other immunosuppressive agents, although their benefit is unclear. Further, these agents, particularly corticosteroids, have been associated with significant adverse effects, in other populations. Current guidelines are available to help guide clinicians, but it is not known if these guidelines are followed, which medications are most commonly used, or which factors impact the decision to initiate therapy at the time of diagnosis or the choice of agents prescribed. The broad, longterm objectives of this application are to address these areas of uncertainty and therefore better determine how treatment strategies could be refined to minimize the exposure of patients to the potential toxicities of existing therapy. The proposed cohort study will therefore focus on the following three specific aims: 1) Quantifying the use of corticosteroids and corticosteroid-sparing agents and the doses of medications prescribed to patients with IPF in broad-based clinical practice; 2) Determining which disease and demographic factors are associated with the initiation of corticosteroid therapy at the time of diagnosis with IPF; and 3) Determining which disease and demographic factors are associated with the initiation.of combination therapy using corticosteroids and corticosteroid sparing agents compared to the use of corticosteroids alone. The proposed study will use a fully anonymized medical record research database from the United Kingdom to evaluate a cohort of approximately 2100 patients with IPF. This would be the largest study of its kind in this disease. From this database, a retrospective cohort study will be conducted and analyzed using bivariable and multivariable analyses to evaluate the association between patientspecific exposure variables and the use of corticosteroids or combination therapy. Relevance: This study aims to understand which factors influence physician decision-making in the treatment of Idiopathic Pulmonary Fibrosis, a progressive and fatal lung disease for which treatments are Often ineffective and associated with significant side effects. The results will lay the foundation for further research quantifying the risks of therapy and the ways in which these risks can be balanced more effectively against the marginal benefits of treatment.