Carlo Vancheri1, Alfredo Sebastiani2, Sara Tomassetti3, Alberto Pesci4, Paola Rogliani5, Laura Tavanti6, Fabrizio Luppi7, Sergio Harari8, Paola Rottoli9, Alessandra Ghirardini10, Klaus-Uwe Kirchgaessler11, Carlo Albera12. 1. University of Catania, Catania, Italy. Electronic address: vancheri@unict.it. 2. San Camillo-Forlanini Hospital, Rome, Italy. 3. Department of Diseases of the Thorax, Ospedale GB Morgagni, Forlì, Italy. 4. University of Milano-Bicocca, Monza, Italy. 5. Respiratory Unit, University of Rome, Tor Vergata, Rome, Italy. 6. University Hospital of Pisa, Pisa, Italy. 7. University Hospital Policlinico di Modena, Modena, Italy. 8. Ospedale San Giuseppe, MultiMedica IRCCS, Milan, Italy. 9. Respiratory Diseases Unit, AOUS-University of Siena, Siena, Italy. 10. Roche SpA, Monza, Italy. 11. F. Hoffmann-La Roche Ltd., Basel, Switzerland. 12. University of Turin, Turin, Italy.
Abstract
RATIONALE: Real-world data on pirfenidone treatment of patients with idiopathic pulmonary fibrosis (IPF) are limited. This study assessed the effectiveness of pirfenidone in a large real-life Italian IPF cohort. METHODS: IRENE was an observational, retrospective study of patients with IPF treated with pirfenidone in routine clinical practice (18 centres). At Month 6, a mandatory re-evaluation of forced vital capacity (FVC) decline (absolute change < 10%) was required to continue pirfenidone. The primary effectiveness outcomes were absolute change from baseline in FVC and the percentage of patients with ≥ 10% absolute decline in % predicted FVC at Month 12. Safety was described by adverse event (AE) occurrence. Prespecified subgroups included sex, age, presence/absence of emphysema, usual interstitial pneumonia (UIP) pattern on high-resolution computed tomography, and baseline lung function. RESULTS: The study included 379 patients (mean age, 67.6 years; 78.1% male). Mean change from baseline in FVC and the percentage of patients with ≥ 10% absolute decline in % predicted FVC at Month 12 were -81.8 mL (SD, 419.6 mL; P = 0.002) and 16.0% (95% CI, 12.2-20.9%), respectively. Disease progression was similar across prespecified subgroups, including patients with definite vs possible UIP. Overall, 211 AEs occurred in 149 patients (39.3%), with serious AEs in 31 patients (8.2%) and 9 discontinuations due to AEs. Skin and gastrointestinal AEs were most frequent. Fifteen patients (4.0%) died. CONCLUSIONS: The decline in FVC and the safety profile observed in this real-world IPF cohort were consistent with the findings of the Phase III pirfenidone trials.
RATIONALE: Real-world data on pirfenidone treatment of patients with idiopathic pulmonary fibrosis (IPF) are limited. This study assessed the effectiveness of pirfenidone in a large real-life Italian IPF cohort. METHODS: IRENE was an observational, retrospective study of patients with IPF treated with pirfenidone in routine clinical practice (18 centres). At Month 6, a mandatory re-evaluation of forced vital capacity (FVC) decline (absolute change < 10%) was required to continue pirfenidone. The primary effectiveness outcomes were absolute change from baseline in FVC and the percentage of patients with ≥ 10% absolute decline in % predicted FVC at Month 12. Safety was described by adverse event (AE) occurrence. Prespecified subgroups included sex, age, presence/absence of emphysema, usual interstitial pneumonia (UIP) pattern on high-resolution computed tomography, and baseline lung function. RESULTS: The study included 379 patients (mean age, 67.6 years; 78.1% male). Mean change from baseline in FVC and the percentage of patients with ≥ 10% absolute decline in % predicted FVC at Month 12 were -81.8 mL (SD, 419.6 mL; P = 0.002) and 16.0% (95% CI, 12.2-20.9%), respectively. Disease progression was similar across prespecified subgroups, including patients with definite vs possible UIP. Overall, 211 AEs occurred in 149 patients (39.3%), with serious AEs in 31 patients (8.2%) and 9 discontinuations due to AEs. Skin and gastrointestinal AEs were most frequent. Fifteen patients (4.0%) died. CONCLUSIONS: The decline in FVC and the safety profile observed in this real-world IPF cohort were consistent with the findings of the Phase III pirfenidone trials.
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