BACKGROUND Pragmatic use of the anti-fibrotic medications pirfenidone and nintedanib for idiopathic pulmonary fibrosis (IPF) in the United States (US) has not been studied and may be different from international settings due to structural differences between health care systems. This study examined the relationship between patient- and site-level characteristics and anti-fibrotic (a) use and (b) selection. METHODS Data from the Pulmonary Fibrosis Foundation Patient Registry was used to perform univariable and multivariable regressions with generalized linear mixed models. A random effects model examined registry site variation. RESULTS 703 of 1218 (57.7%) patients were taking a single anti-fibrotic of which 312 (44.4%) were taking nintedanib and 391 (55.6%) were taking pirfenidone. Up to 25% of patients using an anti-fibrotic may have been excluded from clinical trial participation due to having too severe disease as measured by diffusion limitation for carbon monoxide. Age (OR = 0.974, p = 0.0086) and diffusion capacity of the lungs for carbon monoxide (per 10% increase in percent-predicted; OR = 0.896, p = 0.0007) was negatively associated with anti-fibrotic use while time (in log of days) since diagnosis (OR = 1.138, p < 0.0001), recent patient clinical trial participation (OR = 1.569, p = 0.0433) and oxygen use (OR = 1.604, p = 0.0027) was positively associated with anti-fibrotic use. Time (log of days) since diagnosis (OR = 1.075, p = 0.0477), history of coronary artery disease (OR = 1.796, p = 0.0030), presence of pulmonary hypertension (OR = 2.139, p = 0.0376), patient clinical trial participation in the prior 12 months (OR = 2.485, p = 0.0002), diffusion capacity of the lungs for carbon monoxide (per 10% increase in percent-predicted; OR = 1.138, p = 0.0184), anticoagulant use (OR = 2.507, p = 0.0028), and enrollment at a registry site in the Midwest region (OR = 1.600, p = 0.0446) were associated with pirfenidone use. Anti-fibrotic use varied by registry site. Rates of discontinuation were modest and nearly identical for the two medications with side effects being the most common reason given for discontinuation. Twenty-three percent (23%, 274) of persons with IPF were using or had recently used an immunomodulatory agent. CONCLUSIONS This analysis provides a detailed characterization of IPF treatment patterns in the US; many users of anti-fibrotic medications may not have qualified for inclusion in clinical trials. More research is needed to understand variations in medical decision-making for use and selection of anti-fibrotic medication.

中文翻译：

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在美国，使用吡非尼酮和nintedanib相关的患者和部位特征；肺纤维化基金会患者登记处登记的特发性肺纤维化患者的分析。

背景技术在美国（US）中，尚未研究抗纤维化药物吡非尼酮和任他尼布在特发性肺纤维化（IPF）中的实用性，并且由于医疗保健系统之间的结构差异，可能与国际环境有所不同。这项研究检查了患者和部位水平特征与抗纤维化之间的关系（a）使用和（b）选择。方法采用来自肺纤维化基金会患者登记处的数据对广义线性混合模型进行单变量和多变量回归。随机效应模型检查了注册表站点的变异。结果1218名患者中有703名患者（57.7％）正在接受单一抗纤维化药物治疗，其中312名（44.4％）正在使用nintedanib和391名（55.6％）正在使用吡非尼酮。由于一氧化碳扩散限制所导致的疾病过于严重，多达25％的使用抗纤维化药物的患者可能已被排除在临床试验之外。年龄（OR = 0.974，p = 0.0086）和肺对一氧化碳的扩散能力（预测的百分比每增加10％； OR = 0.896，p = 0.0007）与抗纤维化药物的使用时间呈负相关（以对数表示）天数）自诊断以来（OR = 1.138，p <0.0001），最近的患者临床试验参与（OR = 1.569，p = 0.0433）和吸氧量（OR = 1.604，p = 0.0027）与抗纤维化使用正相关。自诊断以来的时间（天数）（OR = 1.075，p = 0.0477），冠心病病史（OR = 1.796，p = 0.0030），存在肺动脉高压（OR = 2.139，p = 0.0376），前12个月的患者临床试验参与（OR = 2.485，p = 0.0002），肺对一氧化碳的扩散能力（预测的百分数增加10％; OR = 1.138，p = 0.0184），抗凝药的使用（OR = 2.507，p = 0.0028），以及在中西部地区注册中心的注册（OR = 1.600，p = 0.0446）与吡非尼酮的使用相关。抗纤维化的使用因注册表站点而异。两种药物的停药率均中等，几乎相同，而副作用是停药的最常见原因。IPF患者中有23％（23％，274）正在使用或最近使用过免疫调节剂。结论该分析提供了美国IPF治疗模式的详细特征。许多抗纤维化药物的使用者可能没有资格纳入临床试验。需要更多的研究来了解使用和选择抗纤维化药物的医疗决策的差异。