Manchester Medical School, The University of Manchester, Manchester, UK.
North West Interstitial Lung Disease Unit, University Hospital of South Manchester, Wythenshawe Hospital, Manchester, UK.
Adv Ther. 2021 Jan;38(1):268-277. doi: 10.1007/s12325-020-01523-7. Epub 2020 Oct 24.
Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive and irreversible lung disease. Licensed treatment options for IPF are pirfenidone and nintedanib. The aim of this study was to assess the impact of antifibrotic therapy in patients with IPF with preserved lung function based upon a forced vital capacity (FVC) above 80%.
This is a retrospective single-centre cohort study, performed as part of a service evaluation, between January 2007 and September 2018. Patient demographic, treatment and lung function profiles were collected using electronic patient records. A linear mixed model and Kaplan-Meier estimator were utilised to assess changes in FVC and survival over 36 months.
A total of 161 patients were included in this study. Mean age was 72 ± 4. Twenty-four (14.9%) received pirfenidone, 86 (53.4%) received nintedanib and 18 (11.2%) received both antifibrotics provided by a compassionate use program (CUP), as the National Institute of Heath and Clinical excellence (NICE) criteria for antifibrotics in the UK is restricted to an FVC 50-80%. Thirty-three (20.5%) patients did not receive treatment. Patients without antifibrotic therapy had a statistically higher baseline FVC compared to other groups: 3.55 l (100%) vs 2.85 l (89.7%) pirfenidone (p = 0.012), vs 2.99 l (93.5%) nintedanib (p = 0.04) and 3.10 l (92.7%) (p = 0.07) for both antifibrotics. FVC decline over 1 year was similar in groups receiving pirfenidone, nintedanib or no treatment [3.72% (158.1 ml) untreated vs 2.77% (139 ml) pirfenidone vs 2.96% (131 ml) nintedanib]; however, it was significantly greater in patients who received both antifibrotics [6.36% (233 ml), p = 0.01]. Use of antifibrotics was associated with a higher median survival post diagnosis; 3.5, 3 and 3.75 years respectively in pirfenidone, nintedanib and both antifibrotic cohorts, compared to the untreated cohort (2.5 years).
One in five untreated patients with an average FVC of 100% die within a median of 2.5 years. Antifibrotic therapy was associated with a higher median survival of 3-3.75 years despite treatment groups having lower baseline lung function.
特发性肺纤维化（IPF）是一种慢性、进行性和不可逆转的肺部疾病。IPF 的许可治疗选择是吡非尼酮和尼达尼布。本研究旨在评估基于用力肺活量（FVC）大于 80%的保留肺功能的 IPF 患者接受抗纤维化治疗的影响。
这是一项回顾性单中心队列研究，作为服务评估的一部分，于 2007 年 1 月至 2018 年 9 月进行。使用电子病历收集患者的人口统计学、治疗和肺功能概况。线性混合模型和 Kaplan-Meier 估计器用于评估 36 个月内 FVC 和生存的变化。
本研究共纳入 161 例患者。平均年龄为 72±4 岁。24 例（14.9%）接受吡非尼酮治疗，86 例（53.4%）接受尼达尼布治疗，18 例（11.2%）接受由同情使用计划（CUP）提供的两种抗纤维化药物治疗，因为英国国家卫生与临床卓越研究所（NICE）抗纤维化药物的标准仅限于 FVC 50-80%。33 例（20.5%）患者未接受治疗。未接受抗纤维化治疗的患者的基线 FVC 明显高于其他组：3.55 l（100%）与吡非尼酮的 2.85 l（89.7%）相比（p=0.012），与尼达尼布的 2.99 l（93.5%）相比（p=0.04），与两种抗纤维化药物的 3.10 l（92.7%）相比（p=0.07）。接受吡非尼酮、尼达尼布或未治疗的患者在 1 年内的 FVC 下降相似[未治疗组为 3.72%（158.1ml），吡非尼酮组为 2.77%（139ml），尼达尼布组为 2.96%（131ml）]；然而，接受两种抗纤维化药物的患者的 FVC 下降明显更大[6.36%（233ml），p=0.01]。抗纤维化药物的使用与更高的中位生存期相关；在吡非尼酮、尼达尼布和两种抗纤维化药物组中，分别为 3、3 和 3.75 年，而未治疗组为 2.5 年。
在平均 FVC 为 100%的未治疗患者中，五分之一的患者在中位时间 2.5 年内死亡。尽管治疗组的基础肺功能较低，但抗纤维化治疗与中位生存时间 3-3.75 年相关。
