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，与诊断后约5年的中位生存期相关。几种药物可用于这种疾病，包括皮质类固醇和其他免疫抑制剂，尽管它们的益处尚不清楚。此外，这些药物，特别是皮质类固醇，在其他人群中与显著的不良反应相关。目前的指南可用于帮助指导临床医生，但尚不清楚是否遵循这些指南，最常用的药物，或者哪些因素影响诊断时开始治疗的决定或处方药物的选择。本申请的广泛、长期目标是解决这些不确定性领域，从而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，拟定的队列研究将重点关注以下三个具体目标：1）量化糖皮质激素和糖皮质激素保留剂的使用以及在广泛的临床实践中为IPF患者开具的药物剂量; 2）确定哪些疾病和人口统计学因素与诊断IPF时开始糖皮质激素治疗相关;和3）确定与单独使用皮质类固醇相比，哪些疾病和人口统计学因素与开始使用皮质类固醇和皮质类固醇保留剂的联合治疗相关。拟定研究将使用英国的完全匿名病历研究数据库，评价约2100例IPF患者的队列。这将是对这种疾病的最大规模的研究。根据该数据库，将进行一项回顾性队列研究，并使用双变量和多变量分析进行分析，以评价患者特异性暴露变量与皮质类固醇或联合治疗使用之间的相关性。相关性：本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的肺部疾病，治疗通常无效，并伴有显著的副作用。这些结果将为进一步研究量化治疗风险以及更有效地平衡这些风险与治疗边际效益的方法奠定基础。

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.