Vanderbilt University School of Medicine, Nashville, Tennessee, USA.
Duke Clinical Research Institute, Durham, North Carolina, USA; Duke University Medical Center, Durham, North Carolina, USA.
Clin Ther. 2023 Apr;45(4):306-315. doi: 10.1016/j.clinthera.2023.03.003. Epub 2023 Mar 28.
Real-world studies have reported reduced mortality in patients with idiopathic pulmonary fibrosis (IPF) treated with antifibrotic therapy; however, the initiation or discontinuation of therapy during these studies may have introduced bias. This study investigated the effect of antifibrotic therapy on mortality and other outcomes in patients with IPF using causal inference methodology.
Data from a multicenter US registry of patients with IPF were used to assess the effect of antifibrotic therapy (nintedanib or pirfenidone) on death, death or lung transplant, respiratory-related hospitalization, and acute worsening of IPF (defined as any health care encounter deemed due to acute worsening of IPF). This study used the Gran method, which accounts for differences in patient characteristics and for treatment initiations and discontinuations during follow-up. The analysis cohort was limited to patients who started antifibrotic therapy on or after the day of enrollment or had never taken it.
Among the 499 patients analyzed, 352 (70.5%) received antifibrotic therapy. Estimated event rates of death at 1 year were 6.6% (95% CI, 6.1-7.1) for treated patients and 10.2% (95% CI, 9.5-10.9) for control patients. There was a numerical reduction in the risk of death (hazard ratio [HR], 0.53; 95% CI, 0.28-1.03; P = 0.060) but numerical increases in risks of respiratory-related hospitalization (HR, 1.88; 95% CI, 0.90-3.92; P = 0.091) and acute worsening of IPF (HR, 1.71; 95% CI, 0.36-8.09; P = 0.496) in treated versus control patients.
Analyses based on causal inference methodology suggest that patients with IPF who receive antifibrotic therapy have improved survival.
真实世界研究报告称，接受抗纤维化治疗的特发性肺纤维化（IPF）患者死亡率降低；然而，这些研究中治疗的开始或停止可能引入了偏倚。本研究使用因果推理方法研究抗纤维化治疗对 IPF 患者死亡率和其他结局的影响。
使用多中心美国 IPF 患者注册中心的数据评估抗纤维化治疗（尼达尼布或吡非尼酮）对死亡、死亡或肺移植、与呼吸相关的住院治疗以及 IPF 急性恶化（定义为任何被认为是由于 IPF 急性恶化而进行的医疗保健就诊）的影响。本研究使用 Gran 方法，该方法考虑了患者特征的差异以及随访期间治疗的开始和停止。分析队列仅限于在入组日或从未接受过抗纤维化治疗开始后开始接受抗纤维化治疗的患者。
在分析的 499 名患者中，352 名（70.5%）接受了抗纤维化治疗。治疗患者和对照患者 1 年死亡的估计发生率分别为 6.6%（95%CI，6.1-7.1）和 10.2%（95%CI，9.5-10.9）。死亡风险有降低的趋势（风险比[HR]，0.53；95%CI，0.28-1.03；P=0.060），但有增加的趋势，与呼吸相关的住院治疗（HR，1.88；95%CI，0.90-3.92；P=0.091）和 IPF 急性恶化（HR，1.71；95%CI，0.36-8.09；P=0.496）在治疗组和对照组之间。
基于因果推理方法的分析表明，接受抗纤维化治疗的 IPF 患者的生存率得到改善。
