Respiratory Diseases and Lung Transplantation Unit, Azienda Ospedaliera Universitaria Senese (AOUS) - Department of Medical and Surgical Sciences & Neurosciences, University of Siena, Viale Bracci, 16, 53100, Siena, Italy.
Diagnostic Imaging Unit, Azienda Ospedaliera Universitaria Senese (AOUS) - Department of Medical and Surgical Sciences & Neurosciences, University of Siena, Siena, Italy.
Lung. 2019 Apr;197(2):147-153. doi: 10.1007/s00408-019-00203-w. Epub 2019 Feb 13.
Familial pulmonary fibrosis (FPF) is defined as an idiopathic diffuse parenchymal lung disease affecting two or more members of the same primary biological family. The aim of this study was to compare disease progression and tolerance to pirfenidone in a population of FPF patients who presented with radiological and/or histological evidence of UIP, and a group of idiopathic pulmonary fibrosis (IPF) patients.
Seventy-three patients (19 with FPF and 54 with IPF) were enrolled and data were collected retrospectively at 6, 12 and 24 months follow-up.
FPF patients were statistically younger and more frequently females. A significantly greater decline in FVC and DLCO was recorded in FPF than in IPF patients at 24 months follow-up. At the 6-min walking test, walked distance declined significantly in FPF patients than IPF at 24 months. No statistically significant differences in drug tolerance or side effects were recorded between groups.
Different rate of progression was observed in patients with IPF and FPF on therapy with pirfenidone; our findings may not be due to lack of effectiveness of therapy, but to the different natural history and evolution of these two conditions. Pirfenidone was well tolerated by FPF and IPF patients. Specific unbiased randomized clinical trials on larger populations to validate our preliminary exploratory results are needed.
家族性肺纤维化（FPF）定义为影响同一原发性生物学家族两个或多个成员的特发性弥漫性实质性肺疾病。本研究的目的是比较影像学和/或组织学证据为 UIP 的 FPF 患者和特发性肺纤维化（IPF）患者群体对吡非尼酮的疾病进展和耐受性。
共纳入 73 例患者（19 例 FPF 和 54 例 IPF），回顾性收集 6、12 和 24 个月随访的数据。
FPF 患者在统计学上更年轻，女性更常见。在 24 个月的随访中，与 IPF 患者相比，FPF 患者的 FVC 和 DLCO 下降更为明显。在 6 分钟步行试验中，与 IPF 相比，FPF 患者在 24 个月时的步行距离明显下降。两组间药物耐受性或副作用无统计学差异。
在接受吡非尼酮治疗的 IPF 和 FPF 患者中观察到不同的进展速度；我们的发现可能不是由于治疗缺乏有效性，而是由于这两种情况的不同自然史和演变。吡非尼酮在 FPF 和 IPF 患者中均具有良好的耐受性。需要进行特定的、无偏的、针对更大人群的随机临床试验来验证我们初步的探索性结果。
