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DOI: 10.1055/a-2267-2074
Clinical course of mild-to-moderate idiopathic pulmonary fibrosis during therapy with pirfenidone: Results of the non-interventional study AERplus
Klinischer Verlauf einer leichten bis mittelschweren idiopathischen Lungenfibrose unter Therapie mit Pirfenidon – Ergebnisse der nicht-interventionellen Studie AERplusSupported by: Roche Pharma AGClinical Trial: Registration number (trial ID): NCT02622477, Trial registry: ClinicalTrials.gov (http://www.clinicaltrials.gov/), Type of Study: Prospective, multi-center, non-interventional study
Abstract
Introduction Pirfenidone was the first anti-fibrotic drug approved in Europe in 2011 for the treatment of mild-to-moderate idiopathic pulmonary fibrosis.
Objectives To investigate the clinical course of mild-to-moderate idiopathic pulmonary fibrosis in pirfenidone-treated patients in a real-world setting.
Methods The non-interventional study was conducted at 18 sites in Germany from 6/2014–12/2016. Adult patients with mild-to-moderate idiopathic pulmonary fibrosis were treated with pirfenidone (escalated from 3×1 to 3×3 capsules of 267 mg/day within 3 weeks) for 12 months. The observation period comprised 4 follow-up visits at months 3, 6, 9 and 12. Disease progression was defined as decrease of ≥10% in vital capacity or ≥15% in diffusing capacity of the lung for carbon monoxide (DLCO) and/or ≥50m in 6-minute walking distance vs. baseline, or “lack of response/progression“ as reason for therapy discontinuation.
Results A total of 51 patients (80.4% male, mean age 70.6 years) were included in the full analysis set. Disease progression at any visit was reported for 23 (67.6%) of 34 patients with available data. Over the course of the study, lung function parameters, physical resilience, impact of cough severity on quality of life, and the mean Gender, Age and Physiology Index (stage II) remained stable. In total, 29 patients (56.9%) experienced at least one adverse drug reaction (11 patients discontinued due to adverse drug reactions); serious adverse reactions were reported in 12 patients (23.5%).
Conclusions The results of this study are in line with the established benefit-risk profile of pirfenidone. Therefore, pirfenidone can be considered a valuable treatment option to slow disease progression in mild-to-moderate idiopathic pulmonary fibrosis. NCT02622477
Zusammenfassung
Einleitung Pirfenidon war das erste Antifibrotikum, das 2011 in Europa zur Behandlung leichter bis mittelschwerer idiopathischer Lungenfibrose zugelassen wurde.
Ziel Untersuchung des klinischen Verlaufs einer leichten bis mittelschweren idiopathischen Lungenfibrose bei Patient*innen, die unter Real-World-Bedingungen mit Pirfenidon behandelten wurden.
Methoden Die nicht-interventionelle Studie wurde im Zeitraum 6/2014–12/2016 an 18 Standorten in Deutschland durchgeführt. Erwachsene Patient*innen mit leichter bis mittelschwerer idiopathischer Lungenfibrose wurden über 12 Monate mit Pirfenidon (eskaliert von 3×1 auf 3×3 Kapseln à 267 mg/Tag innerhalb von 3 Wochen) behandelt. Der Beobachtungszeitraum umfasste 4 Nachuntersuchungen in den Monaten 3, 6, 9 und 12. Krankheitsprogression wurde definiert als Abnahme der Vitalkapazität um ≥ 10% oder der Diffusionskapazität der Lunge für Kohlenmonoxid (DLCO) um ≥ 15% und/oder um ≥ 50 m der 6-Minuten-Gehstrecke im Vergleich zum Ausgangswert oder „mangelndes Ansprechen/Progression“ als Grund für den Therapieabbruch.
Ergebnisse 51 Patient*innen (80,4% männlich, Durchschnittsalter 70,6 Jahre) wurden in das Full-Analysis-Set einbezogen. Für 23 (67,6%) der 34 Patient*innen mit verfügbaren Daten wurde bei Follow-up-Visiten Krankheitsprogression gemeldet. Im Verlauf der Studie blieben die Lungenfunktionsparameter, die körperliche Belastbarkeit, der Einfluss der Hustenstärke auf die Lebensqualität und der mittlere Gender, Age and Physiology Index (Stadium II) stabil. Insgesamt kam es bei 29 Patient*innen (56,9%) zu mindestens einer unerwünschten Arzneimittelwirkung (11 Patient*innen brachen die Behandlung aufgrund unerwünschter Arzneimittelwirkungen ab); schwerwiegende unerwünschte Arzneimittelwirkungen wurden bei 12 Patient*innen (23,5%) berichtet.
Schlussfolgerung Die Ergebnisse dieser Studie stimmen mit dem etablierten Nutzen-Risiko-Profil von Pirfenidon überein. Daher kann Pirfenidon als nützliche Behandlungsoption zur Verlangsamung des Krankheitsverlaufs bei leichter bis mittelschwerer idiopathischer Lungenfibrose angesehen werden. NCT02622477
Keywords
idiopathic pulmonary fibrosis - pirfenidone - interstitial lung disease - pulmonary function - anti-fibroticSchlüsselwörter
idiopathische Lungenfibrose - Pirfenidon - interstitielle Lungenerkrankung - Lungenfunktion - antifibrotischPublication History
Received: 08 November 2023
Accepted after revision: 11 January 2024
Article published online:
12 April 2024
© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial-License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/).
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