Extract
Cystic fibrosis (CF) is an incurable, autosomal recessive multisystem disorder that is associated with multiple complications, in particular progressive lung disease, chronic pulmonary infections, respiratory failure and reduced life expectancy [1, 2]. In this regard, lung transplantation is an established treatment option for end-stage CF lung disease, aiming to improve both quality of life and survival [3].
Abstract
This study observed a pronounced decline of CF-related lung transplants up to −83% in 2021 compared to the mean number in the period between 2012 and 2019, following the broad accessibility of elexacaftor-tezacaftor-ivacaftor in Germany in August 2020 https://bit.ly/3Pl7N9Y
Acknowledgements
The authors thank Axel Jens and the team from Eurotransplant International Foundation, Leiden, the Netherlands for their support and the provision of data. They dedicate this report to our highly respected colleague Burkhard Tümmler, who devoted his impressive scientific career to the fight against CF. The authors received no funding for the present research.
Footnotes
Conflict of interest: F.C. Ringshausen reports grants from the German Center for Lung Research (DZL), the German Center for Infection Research (DZIF), the Innovative Medicines Initiative (IMI; EU/EFPIA), the iABC Consortium (including Alaxia, Basilea, Novartis and Polyphor), Mukoviszidose Institute, Novartis, Insmed Germany, Grifols, Bayer Healthcare and InfectoPharm (paid to his institution), fees for clinical trial participation from Abbvie, AstraZeneca, Boehringer Ingelheim, Celtaxsys, Corbus, Insmed, Novartis, Parion, University of Dundee, Vertex and Zambon (paid to his institution), personal consulting fees from Parion Sciences, Grifols, Zambon, Insmed and Helmholtz Centre for Infection Research, personal honoraria for lectures from !DE Werbeagentur GmbH, Interkongress GmbH, AstraZeneca, Insmed, Grifols and University Hospital Frankfurt, payment for expert testimony from the Social Court Cologne, financial support for attending meetings from the German Kartagener Syndrome, Primary Ciliary Dyskinesia Patient Advisory Group and the German Cystic Fibrosis Patient Advisory Group (Mukoviszidose e.V.), and personal payments for participation on an advisory board from Insmed, Grifols and Shionogi; and is honorary (unpaid) coordinator of the ERN-LUNG Bronchiectasis Core Network, co-chair of the German Bronchiectasis Registry PROGNOSIS, member of the steering committee of the European Bronchiectasis Registry EMBARC, member of the steering committee of the European NTM Registry EMBARC-NTM, co-speaker of the Medical Advisory Board of the German Kartagener Syndrome and Primary Ciliary Dyskinesia Patient Advisory Group, speaker of the Respiratory Infections and TB group of the German Respiratory Society (DGP), speaker of the Cystic Fibrosis group of German Respiratory Society (DGP), principal investigator of the German Center for Lung Research (DZL), member of the Protocol Review Committee of the PCD Clinical Trial Network and member of Physician Association of the German Cystic Fibrosis Patient Advisory Group (Mukoviszidose e.V.). A. Sauer-Heilborn has nothing to declare. T. Büttner reports grants from Mukoviszidose Institute paid to her institution and honorary (unpaid) commitment as member of the protocol review committee of the ECFS-CTN, as well as board membership of the physiotherapy working group within the German Cystic Fibrosis Patient Advisory Group (Mukoviszidose e.V.). A-M. Dittrich reports grants from the German Center for Lung Research (DZL), the German Research Foundation (DFG), the European Cystic Fibrosis Clinical Trial Network (ECFS-CTN), the Christiane-Herzog-Foundation for Cystic Fibrosis, the German Cystic Fibrosis Patient Advisory Group (Mukoviszidose e.V.) and Vertex (paid to her institution), fees for clinical trial participation from Vertex (paid to her institution), personal consulting fees from Vertex, Connect-for-children (c4c) trial network and the Helmholtz Centre for Infection Research, lecture honoraria from Vertex, personal payments for participation on an advisory board from Vertex; and is honorary (unpaid) member of the protocol review committee of the ECFS-CTN, board member of the German Cystic Fibrosis Patient Advisory Group (Mukoviszidose e.V.) and lead respiratory expert of the c4c trial network. N. Schwerk reports personal lecture fees from Abbvie, Allergopharma, InfectoPharm, Novartis and Sanofi, and personal payments for participation on an advisory board from Boehringer Ingelheim and Novartis; and is honorary (unpaid) board member of the Kinderlungenregister, the Deutsche Gesellschaft für Pädiatrische Pneumologie and the Norddeutsche Arbeitsgemeinschaft für pädiatrische Pneumologie und Allergologie. F. Ius reports personal consulting and lecture fees as well as support for attending meetings from Biotest AG. L. Nährlich reports grants or contracts from German Center for Lung Research (DZL), fees from Vertex Pharmaceuticals and Boehringer Ingelheim for clinical trial participating, participation on a trial steering committee for CF STORM; and is medical lead of the German Cystic Fibrosis Registry, pharmacovigilance study manager of the ECFSPR, and receipt of medical writing from Articulate Science. T. Welte reports research grants from the German Center for Lung Research (DZL) and fees for clinical trial participation from Vertex, both paid to his institution. M. Greer reports personal speaker fees from Therakos UK and is honorary (unpaid) chairman of the data monitoring committee of the E-CLAD UK trial for extracorporeal photophoresis in the treatment of chronic allograft dysfunction after lung transplantation (NIHR, UK).
- Received July 11, 2022.
- Accepted December 1, 2022.
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