摘要目的 观察真实世界中吡非尼酮治疗特发性肺纤维化(IPF)的疗效和安全性.方法 采用回顾性病例对照研究的方法连续入选北京朝阳医院接受吡非尼酮治疗的IPF患者47例(吡非尼酮组),其中男44例,女3例,年龄48~82岁,平均(66±9)岁;同期未接受吡非尼酮治疗的IPF患者47例(对照组),均为男性,年龄51~83岁,平均(67±8)岁.按照研究流程记录临床数据,观察吡非尼酮的疗效及不良反应,并进行定期随访.结果 随访6个月时吡非尼酮组与对照组FVC占预计值%较基线变化值分别为[(3.5±7.2)% 和(-2.3±6.7)%,t=2.166,P=0.041);DLCO占预计值%较基线变化值分别为[(1.1±6.1)%和(-4.7±6.2)%,t=2.519,P=0.018];随访12个月时FVC占预计值%较基线变化值分别为[(2.3±7.0)% 和(-3.3±6.2)%,t=2.292,P=0.030];DLCO占预计值%较基线变化值分别为[(-1.3±12.2)% 和(-5.3±9.8)%,t=1.047,P=0.303].吡非尼酮组中17例IPF患者规律随访,治疗后6个月与治疗前6个月相比,FVC占预计值%较基线变化值分别为[(3.9±7.8)% 和(-6.0±8.2)%,Z=-2.897,P=0.004];DLCO占预计值%的变化值分别为[(1.3±6.6)% 和(-7.0±13.3)%,Z=-2.151,P=0.031].分别以6个月内FVC占预计值%较基线下降≥5%或≥10%为界值进行分析,治疗后6个月患者FVC占预计值%较基线下降≥5%或≥10%的例数显著减少,差异有统计学意义(P=0.010、0.018).吡非尼酮治疗期间30例(63.8%)出现不良反应,所有患者均未中断治疗.其中14例出现胃肠道不良反应,11例出现皮肤不良反应.结论 在真实世界中随访12个月,IPF患者口服吡非尼酮能够一定程度地延缓肺功能下降,对吡非尼酮耐受良好.

abstractsObjective To evaluate the efficacy and safety of pirfenidone (PFD) in idiopathic pulmonary fibrosis (IPF) in real-life world.Methods 47 consecutive patients with IPF taking PFD for at least 12 months (PFD group) were included for analysis,with 47 patients with IPF who did not take PFD as controls.Data were collected from clinical charts to evaluate the lung function parameters and the adverse reactions of PFD.Results In the PFD group, the percent predicted forced vital capacity (FVC%) and diffusing capacity of carbon monoxide (DLCO%) increased by (3.5±7.2)% and (1.1±6.1)% after 6 months of PFD treatment compared with the baseline, while the mean decline of FVC% and DLCO% was (2.3±6.7)% and (4.7±6.2)% in the controls (t=2.166,P=0.041;t=2.519,P=0.018).After 12 months of treatment, FVC% increased by (2.3±7.0)% and DLCO% decreased by (1.3±12.2)% compared with the baseline,while the mean decline of FVC% and DLCO% was (3.3±6.2)% and (5.3± 9.8)% in the controls (t=2.292, P=0.030; t=1.047, P=0.303).In the PFD group, 17 patients regularly received pulmonary function test per 6 months.The mean decline in FVC% and DLCO% was (6.0 ±8.2)% and (7.0±13.3)% from 6 month before treatment to baseline when PFD was started,while the FVC% increased by (3.9±7.8)% and DLCO% increased by (1.3±6.6)% after 6 months of treatment compared with the baseline (Z=-2.897,P=0.004;Z=-2.151,P=0.031).The proportion of patients with more than 5% or 10% decline in FVC% decreased significantly after 6 months therapy (P=0.010 and 0.018, respectively).Adverse events were commonly seen in 30 patients while taking pirfenidone.The most common adverse events were gastrointestinal symptoms and skin reactions. Conclusions In real-life world, PFD therapy for 12 months showed good effectiveness on pulmonary functions in patients with mild to severe IPF.Adverse events were common but mild.