Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2024 Apr;78(4):236-243.
doi: 10.1055/a-2267-2074. Epub 2024 Apr 12.

Clinical course of mild-to-moderate idiopathic pulmonary fibrosis during therapy with pirfenidone: Results of the non-interventional study AERplus

Jens Schreiber  1 , Wolfgang Schütte  2 , Wolfgang Koerber  3 , Bernd Seese  4 , Dirk Koschel  5 , Kathrin Neuland  6 , Christian Grohé  7
Affiliations
Clinical Trial

Clinical course of mild-to-moderate idiopathic pulmonary fibrosis during therapy with pirfenidone: Results of the non-interventional study AERplus

Jens Schreiber et al. Pneumologie. 2024 Apr.

Abstract
in English, German

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.

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.

PubMed Disclaimer

Conflict of interest statement

JS: grants, personal fees and research support from Roche, during the conduct of the study; WS: Research grants, investigator fees, honoraria, speakers fees from Lilly, Merck, AstraZeneca, Roche, Boehringer Ingelheim; WK: The author has no conflicts of interest to declare; BS: Speakers fees from Boehringer Ingelheim, Berlin Chemie, Roche, GSK; DK: consultancy or speaker fees from AstraZeneca, Boehringer Ingelheim, Grifols, Novartis, Roche/Intermune, Sanofi-Aventis and Teva; KN: Employee of Roche; CG: Boehringer Ingelheim, Novartis, Merck Sharp & Dome, Takeda, Boehringer Ingelheim, Roche. CG is editor-in-chief for the journal “Pneumologie”.

Figures

Fig. 1
Fig. 1
Disease progression. Disease progression was defined as relative VC decrease of at least 10% compared to baseline, or relative decrease of DL CO of at least 15% compared to baseline, or decrease of the 6-minute walk distance (6-MWD) of at least 50 m compared to baseline, or if the investigator stated „lack of response/progression” as reason for therapy discontinuation. 6-MWD, 6-Minute walking distance; DL CO , diffusing capacity of the lung for carbon monoxide; VC, vital capacity.
Fig. 2
Fig. 2
Pulmonary function. BL, baseline; DL CO , diffusing capacity of the lung for carbon monoxide; FEV 1 , forced expiratory volume in 1 second; FVC, forced vital capacity; SD, standard deviation; VC, vital capacity.
Fig. 3
Fig. 3
6-Minute Walking Distance. SD, standard deviation.
Fig. 4
Fig. 4
Leicester Cough Questionnaire. Values denote total LCQ score ± standard deviation. The total score ranges from 3–21. Higher scores represent higher quality of life. LCQ, Leicester Cough Questionnaire.
See this image and copyright information in PMC

References

    1. Raghu G, Remy-Jardin M, Richeldi L et al.Idiopathic Pulmonary Fibrosis (an Update) and Progressive Pulmonary Fibrosis in Adults: An Official ATS/ERS/JRS/ALAT Clinical Practice Guideline. Am J Respir Crit Care Med. 2022;205:e18–e47. doi: 10.1164/rccm.202202-0399ST. - DOI - PMC - PubMed
    1. Podolanczuk AJ, Thomson CC, Remy-Jardin M et al.Idiopathic pulmonary fibrosis: state of the art for 2023. Eur Respir J. 2023;61 doi: 10.1183/13993003.00957-2022. - DOI - PubMed
    1. American Thoracic Society . Idiopathic pulmonary fibrosis: diagnosis and treatment. International consensus statement. American Thoracic Society (ATS), and the European Respiratory Society (ERS) Am J Respir Crit Care Med. 2000;161:646–664. doi: 10.1164/ajrccm.161.2.ats3-00. - DOI - PubMed
    1. Kaunisto J, Salomaa ER, Hodgson U et al.Idiopathic pulmonary fibrosis – a systematic review on methodology for the collection of epidemiological data. BMC Pulm Med. 2013;13:53. doi: 10.1186/1471-2466-13-53. - DOI - PMC - PubMed
    1. Olson AL, Gifford AH, Inase N et al.The epidemiology of idiopathic pulmonary fibrosis and interstitial lung diseases at risk of a progressive-fibrosing phenotype. Eur Respir Rev. 2018;27:180077. doi: 10.1183/16000617.0077-2018. - DOI - PMC - PubMed

Publication types

Associated data

LinkOut - more resources