Stéphane Jouneau1, Anne-Sophie Gamez2, Julie Traclet3, Hilario Nunes4, Sylvain Marchand-Adam5, Romain Kessler6, Dominique Israël-Biet7, Raphael Borie8, Indiana Strombom9, Astrid Scalori10, Bruno Crestani8, Dominique Valeyre4, Vincent Cottin3. 1. Respiratory Diseases Department, Pontchaillou Hospital, IRSET UMR 1085, Rennes 1 University, Rennes, France, stephane.jouneau@chu-rennes.fr. 2. Pulmonology, Montpellier University Hospital, Montpellier, France. 3. Pulmonology Department, National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, University Claude Bernard Lyon 1, Lyon, France. 4. Pulmonology Department, Constitutive Site of the Reference Center for Rare Lung Diseases, Avicenne Hospital, Paris University 13, Bobigny, France. 5. Pulmonology Department, Tours Regional University Hospital, Tours, France. 6. Pulmonology, Strasbourg University Hospital, Strasbourg, France. 7. Pulmonology Department, Georges Pompidou European Hospital, AP-HP, Paris Descartes University, Paris, France. 8. Pulmonology Department, Bichat Hospital, Paris, France. 9. Genentech, San Francisco, California, USA. 10. Roche, Welwyn Garden City, United Kingdom.
Abstract
BACKGROUND: The European observational, prospective PASSPORT study evaluated the long-term safety of pirfenidone under real-world conditions in idiopathic pulmonary fibrosis (IPF), over up to 2 years following its initiation. OBJECTIVES: The FAS (French Ancillary Study) assessed the clinical outcomes of IPF patients participating in PASSPORT (n = 192). METHODS: Efficacy data were collected retrospectively and prospectively. The primary efficacy endpoints were: change in percent predicted forced vital capacity (FVC) and change in the distance travelled during the 6-min walk test (6MWD). RESULTS: The mean baseline FVC was 71.7% of predicted value. The mean absolute change in the percentage of predicted FVC was -2.4% and -3.8% at months 12 and 24. The mean change in 6MWD was 8.6 and 3.1 m at months 12 and 24, with a range of 23.4-51.7 m. Acute IPF exacerbation and pulmonary hypertension occurred in 20.0 and 8.4% of patients, respectively. The most common reasons for prematurely discontinuing PASSPORT were adverse drug reactions (ADRs) related to pirfenidone (31.3%), death (11.5%), and disease progression (10.9%). The median progression-free survival was 18.4 months (95% CI 12.9, not estimable). The median exposure was 16.3 months (0.5-28.5). The most frequently reported ADRs leading to pirfenidone discontinuation were decreased weight (4.2%), rash (4.2%), and photosensitivity reactions (3.1%). CONCLUSIONS: The efficacy data of FAS are consistent with the efficacy results of published phase III clinical trials in IPF. Approximately one third of IPF patients treated with pirfenidone in real-life settings were still under treatment 2 years after initiation. Safety data are consistent with the known safety profile of pirfenidone.
BACKGROUND: The European observational, prospective PASSPORT study evaluated the long-term safety of pirfenidone under real-world conditions in idiopathic pulmonary fibrosis (IPF), over up to 2 years following its initiation. OBJECTIVES: The FAS (French Ancillary Study) assessed the clinical outcomes of IPF patients participating in PASSPORT (n = 192). METHODS: Efficacy data were collected retrospectively and prospectively. The primary efficacy endpoints were: change in percent predicted forced vital capacity (FVC) and change in the distance travelled during the 6-min walk test (6MWD). RESULTS: The mean baseline FVC was 71.7% of predicted value. The mean absolute change in the percentage of predicted FVC was -2.4% and -3.8% at months 12 and 24. The mean change in 6MWD was 8.6 and 3.1 m at months 12 and 24, with a range of 23.4-51.7 m. Acute IPF exacerbation and pulmonary hypertension occurred in 20.0 and 8.4% of patients, respectively. The most common reasons for prematurely discontinuing PASSPORT were adverse drug reactions (ADRs) related to pirfenidone (31.3%), death (11.5%), and disease progression (10.9%). The median progression-free survival was 18.4 months (95% CI 12.9, not estimable). The median exposure was 16.3 months (0.5-28.5). The most frequently reported ADRs leading to pirfenidone discontinuation were decreased weight (4.2%), rash (4.2%), and photosensitivity reactions (3.1%). CONCLUSIONS: The efficacy data of FAS are consistent with the efficacy results of published phase III clinical trials in IPF. Approximately one third of IPF patients treated with pirfenidone in real-life settings were still under treatment 2 years after initiation. Safety data are consistent with the known safety profile of pirfenidone.
Authors: Sebastian Majewski; Adam J Białas; Małgorzata Buchczyk; Paweł Gomółka; Katarzyna Górska; Hanna Jagielska-Len; Agnieszka Jarzemska; Ewa Jassem; Dariusz Jastrzębski; Aleksander Kania; Marek Koprowski; Rafał Krenke; Jan Kuś; Katarzyna Lewandowska; Magdalena M Martusewicz-Boros; Kazimierz Roszkowski-Śliż; Alicja Siemińska; Krzysztof Sładek; Małgorzata Sobiecka; Karolina Szewczyk; Małgorzata Tomczak; Witold Tomkowski; Elżbieta Wiatr; Dariusz Ziora; Beata Żołnowska; Wojciech J Piotrowski Journal: BMC Pulm Med Date: 2020-05-04 Impact factor: 3.317
Authors: Ondřej Májek; Jakub Gregor; Nesrin Mogulkoć; Katarzyna Lewandowska; Martina Šterclová; Veronika Müller; Marta Hájková; Mordechai R Kramer; Jasna Tekavec-Trkanjec; Dragana Jovanović; Michael Studnicka; Natalia Stoeva; Klaus-Uwe Kirchgässler; Simona Littnerová; Ladislav Dušek; Martina Koziar Vašáková Journal: PLoS One Date: 2022-09-01 Impact factor: 3.752