Abstract
BackgroundPleuroparenchymal fibroelastosis (PPFE) has a variable disease course with dismal prognosis in the majority of patients with no validated drug therapy. This study is to evaluate the effect of nintedanib in patients with idiopathic and secondary PPFE. Patients admitted to a tertiary care center (2010–2019) were included into this retrospective analysis if they had a multidisciplinary diagnosis of PPFE, had been followed-up for 3 months or more, and had lung function tests and chest CTs available for review. Changes in pulmonary function tests were assessed using non-parametric tests and linear mixed effect model. Lung volumes were measured with lobar segmentation using chest CT.ResultsOut of 21 patients with PPFE, nine had received nintedanib, six had received another treatment and another six patients were monitored without drug therapy. Annual FVC (% of predicted) relative decline was − 13.6 ± 13.4%/year before nintedanib and − 1.6 ± 6.02%/year during nintedanib treatment (p = 0.014), whereas no significant change in FVC% relative decline was found in patients receiving another treatment (− 13.25 ± 34 before vs − 16.61 ± 36.2%/year during treatment; p = 0.343). Using linear mixed effect model, the slope in FVC was − 0.97%/month (95% CI: − 1.42; − 0.52) before treatment and − 0.50%/month (95% CI: − 0.88; 0.13) on nintedanib, with a difference between groups of + 0.47%/month (95% CI: 0.16; 0.78), p = 0.004. The decline in the upper lung volumes measured by CT was − 233 mL/year ± 387 mL/year before nintedanib and − 149 mL/year ± 173 mL/year on nintedanib (p = 0.327). Nintedanib tolerability was unremarkable.ConclusionIn patients with PPFE, nintedanib treatment might be associated with slower decline in lung function, paving the way for prospective, controlled studies.
Highlights
Pleuroparenchymal fibroelastosis (PPFE) is a rare clinicopathological entity initially described in 1992 by Amitani et al in 13 patients presenting with a condition called idiopathic pulmonary upper lobe fibrosis [1]
Baseline characteristics In total, 21 patients were diagnosed with PPFE in multidisciplinary discussion during the study period, including nine who had received nintedanib for 3 months or more
Patients were distributed into the nintedanib group (n = 9), the nonnintedanib treatment group (n = 6; pirfenidone alone [n = 2], prednisone associated or not with mycophenolate mofetil [n = 4]), and the surveillance/no treatment group (n = 6, who were managed with surveillance [n = 5], or received nintedanib for less than 3 months [n = 1])
Summary
Pleuroparenchymal fibroelastosis (PPFE) is a rare clinicopathological entity initially described in 1992 by Amitani et al in 13 patients presenting with a condition called idiopathic pulmonary upper lobe fibrosis [1]. Experience with antifibrotic drugs, which slow disease progression in patients with fibrotic interstitial lung disease (ILD) including idiopathic pulmonary fibrosis (IPF) [8], systemic sclerosis associated ILD [9], ILD with a progressive fibrosing phenotype [10], and unclassifiable idiopathic progressive ILD [11], is scarce in PPFE. Pirfenidone has been used in one patient with idiopathic PPFE with associated basal usual interstitial pneumonia (UIP), who showed a stabilized lung function, yet, the patient died 6 months later from respiratory failure [12]. Volumetric imaging was used as adjunct to pulmonary function to assess lung volume changes at the lobar level during disease course. Pleuroparenchymal fibroelastosis (PPFE) has a variable disease course with dismal prognosis in the majority of patients with no validated drug therapy. Lung volumes were measured with lobar segmentation using chest CT
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