Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis
Abstract Background Pleuroparenchymal 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 tertia...
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| Format: | Article |
| Language: | English |
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BMC
2021-10-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-021-02043-5 |
| _version_ | 1818719073982218240 |
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| author | Mouhamad Nasser Salim Si-Mohamed Ségolène Turquier Julie Traclet Kaïs Ahmad François Philit Philippe Bonniaud Lara Chalabreysse Françoise Thivolet-Béjui Vincent Cottin |
| author_facet | Mouhamad Nasser Salim Si-Mohamed Ségolène Turquier Julie Traclet Kaïs Ahmad François Philit Philippe Bonniaud Lara Chalabreysse Françoise Thivolet-Béjui Vincent Cottin |
| author_sort | Mouhamad Nasser |
| collection | DOAJ |
| description | Abstract Background Pleuroparenchymal 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. Results Out 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. Conclusion In patients with PPFE, nintedanib treatment might be associated with slower decline in lung function, paving the way for prospective, controlled studies. |
| first_indexed | 2024-12-17T20:01:09Z |
| format | Article |
| id | doaj.art-7af799ca0f644686a2d8802625a763a4 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-12-17T20:01:09Z |
| publishDate | 2021-10-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-7af799ca0f644686a2d8802625a763a42022-12-21T21:34:28ZengBMCOrphanet Journal of Rare Diseases1750-11722021-10-011611910.1186/s13023-021-02043-5Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosisMouhamad Nasser0Salim Si-Mohamed1Ségolène Turquier2Julie Traclet3Kaïs Ahmad4François Philit5Philippe Bonniaud6Lara Chalabreysse7Françoise Thivolet-Béjui8Vincent Cottin9Department of Respiratory Medicine, National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, Hospices Civils de LyonRadiology Department, Hospices Civils de LyonDepartment of Respiratory Physiology, Hospices Civils de Lyon, Louis Pradel HospitalDepartment of Respiratory Medicine, National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, Hospices Civils de LyonDepartment of Respiratory Medicine, National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, Hospices Civils de LyonDepartment of Respiratory Medicine, Croix Rousse Hospital, Hospices Civil de LyonDepartment of Pulmonary Medicine and Intensive Care Unit, Constitutive Reference Center for Rare Pulmonary Diseases, François Mitterrand Teaching Hospital, Inserm U1231, University Bourgogne-Franche ComtéDepartment of Pathology, Louis Pradel Hospital, Hospices Civils de LyonDepartment of Pathology, Louis Pradel Hospital, Hospices Civils de LyonDepartment of Respiratory Medicine, National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, Hospices Civils de LyonAbstract Background Pleuroparenchymal 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. Results Out 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. Conclusion In patients with PPFE, nintedanib treatment might be associated with slower decline in lung function, paving the way for prospective, controlled studies.https://doi.org/10.1186/s13023-021-02043-5Pulmonary fibrosisPleuroparenchymal fibroelastosisPulmonary function testsCT volumetryNintedanibProgression |
| spellingShingle | Mouhamad Nasser Salim Si-Mohamed Ségolène Turquier Julie Traclet Kaïs Ahmad François Philit Philippe Bonniaud Lara Chalabreysse Françoise Thivolet-Béjui Vincent Cottin Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis Orphanet Journal of Rare Diseases Pulmonary fibrosis Pleuroparenchymal fibroelastosis Pulmonary function tests CT volumetry Nintedanib Progression |
| title | Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis |
| title_full | Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis |
| title_fullStr | Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis |
| title_full_unstemmed | Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis |
| title_short | Nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis |
| title_sort | nintedanib in idiopathic and secondary pleuroparenchymal fibroelastosis |
| topic | Pulmonary fibrosis Pleuroparenchymal fibroelastosis Pulmonary function tests CT volumetry Nintedanib Progression |
| url | https://doi.org/10.1186/s13023-021-02043-5 |
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