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volume 32 pages 100690

Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry

Sivagurunathan Sutharsan
Stefanie Dillenhoefer
Matthias Welsner
Manuel Burkhart
Olaf Eickmeier
Matthias Kappler
Sarah Sieber
Susanne Naehrig
Lutz Naehrlich
Klaus Tenbrock
Claus Pfannenstiel
Dirk Steffen
Jochen Meister
Britta Welzenbach
Anette Scharschinger
Markus Kratz
Maike Pincus
Tobias Tenenbaum
Mirjam Stahl
Kerstin Landwehr
Stefanie Dillenhöfer
Hans Kössel
Petra Kaiser
Manfred Käding
Simone Stolz
Stefan Blaas
Jutta Hammermann
Monika Gappa
Antje Schuster
Dana Spittel
Sabine Zirlik
Sabina Schmitt
Florian Stehling
Sivagurunathan Sutharsan
Joachim Bargon
Malte Cremer
Christina Smaczny
Sebastian Fähndrich
Andrea Heinzmann
Lutz Nährlich
Stefan Kuhnert
Sebastian Schmidt
Bettina Wollschläger
Anna Nolde
Inka Held
Wolfgang Kamin
Felix C. Ringshausen
Anna-Maria Dittrich
Sabine Wege
Olaf Sommerburg
Norbert Geier
Sara Lisa Fleser
Heinrike Wilkens
Helmut Ellemunter
Michael Lorenz
Paul Vöhringer
Martin Schebek
Christian Timke
Ingrid Bobis
Thomas Nüßlein
Doris Dieninghoff
Ernst Rietschel
Bastian Klinkhammer
Freerk Prenzel
Alexandra Wald
Axel Kempa
Folke Brinkmann
Eva Lücke
Ines Adams
Krystyna Poplawska
Simone Lehmkühler
Monika Bauck
Anne Pfülb
Rainald Fischer
Gudrun Schopper
Susanne Nährig
Matthias Griese
Jörg Grosse
Peter Küster
Birte KinderHolger Köster
Susanne Büsing
Margarethe Pohl
Carsten Schwarz
Andreas Artlich
Alexander Kiefer
Manfred Ballmann
Nikola Gjorgjevski
Markus A. Rose
Friederike Ruf
Rolf Mahlberg
Wolfgang Thomas
Ute Graepler
Sebastian Bode
hilipp Meyn
Josef Rosenecker
Cordula Koerner
Klaus-Michael Keller
Tina Teßmer
Helge Hebestreit
Gerhild Lohse
Publication typeJournal Article
Publication date2023-09-01
scimago Q1
wos Q1
SJR4.088
CiteScore22.8
Impact factor13.0
ISSN26667762
Oncology
Health Policy
Internal Medicine
Abstract
Treatment with elexacaftor/tezacaftor/ivacaftor (ETI) improves multiple clinical outcomes in people with cystic fibrosis (pwCF) with at least one F508del allele. This study evaluated the real-world impact of ETI on lung function, nutritional status, pulmonary exacerbation frequency, and sweat chloride concentrations in a large group of pwCF.This observational cohort study used data from the German CF Registry for pwCF who received ETI therapy and were followed up for a period of 12 months.The study included 2645 pwCF from 67 centres in Germany (mean age 28.0 ± 11.5 years). Over the first year after ETI was initiated, percent predicted forced expiratory volume in 1 s (ppFEV1) increased by 11.3% (95% confidence interval [CI] 10.8-11.8, p < 0.0001), body mass index (BMI) z-score increased by 0.3 (95% CI 0.3-0.4, p < 0.0001) in individuals aged 12 to <18 years and BMI in adults increased by 1.4 kg/m2 (95% CI 1.3-1.4, p < 0.0001), pulmonary exacerbations decreased by 75.9% (p < 0.0001) and mean sweat chloride concentration decreased by 50.9 mmol/L (95% CI -52.6, -49.3, p < 0.0001). Improvements in ppFEV1 over the first year of therapy were greater in pwCF who had not previously received cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy (12.6% [95% CI 11.9-13.4] vs. 9.7% [95% CI 9.0-10.5] in those with prior CFTR modulator treatment.These real-world data are consistent with the findings of randomised clinical trials, and support the use of ETI as a highly effective treatment option for pwCF who have at least one F508del allele.None.
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GOST Copy
Sutharsan S. et al. Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry // The Lancet Regional Health - Europe. 2023. Vol. 32. p. 100690.
GOST all authors (up to 50) Copy
Sutharsan S. et al. Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry // The Lancet Regional Health - Europe. 2023. Vol. 32. p. 100690.
RIS |
Cite this
RIS Copy
TY - JOUR
DO - 10.1016/j.lanepe.2023.100690
UR - https://doi.org/10.1016/j.lanepe.2023.100690
TI - Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry
T2 - The Lancet Regional Health - Europe
AU - Sutharsan, Sivagurunathan
AU - Dillenhoefer, Stefanie
AU - Welsner, Matthias
AU - Burkhart, Manuel
AU - Eickmeier, Olaf
AU - Kappler, Matthias
AU - Sieber, Sarah
AU - Naehrig, Susanne
AU - Naehrlich, Lutz
AU - Tenbrock, Klaus
AU - Pfannenstiel, Claus
AU - Steffen, Dirk
AU - Meister, Jochen
AU - Welzenbach, Britta
AU - Scharschinger, Anette
AU - Kratz, Markus
AU - Pincus, Maike
AU - Tenenbaum, Tobias
AU - Stahl, Mirjam
AU - Landwehr, Kerstin
AU - Dillenhöfer, Stefanie
AU - Kössel, Hans
AU - Kaiser, Petra
AU - Käding, Manfred
AU - Stolz, Simone
AU - Blaas, Stefan
AU - Hammermann, Jutta
AU - Gappa, Monika
AU - Schuster, Antje
AU - Spittel, Dana
AU - Zirlik, Sabine
AU - Schmitt, Sabina
AU - Stehling, Florian
AU - Sutharsan, Sivagurunathan
AU - Bargon, Joachim
AU - Cremer, Malte
AU - Smaczny, Christina
AU - Fähndrich, Sebastian
AU - Heinzmann, Andrea
AU - Nährlich, Lutz
AU - Kuhnert, Stefan
AU - Schmidt, Sebastian
AU - Wollschläger, Bettina
AU - Nolde, Anna
AU - Held, Inka
AU - Kamin, Wolfgang
AU - Ringshausen, Felix C.
AU - Dittrich, Anna-Maria
AU - Wege, Sabine
AU - Sommerburg, Olaf
AU - Geier, Norbert
AU - Fleser, Sara Lisa
AU - Wilkens, Heinrike
AU - Ellemunter, Helmut
AU - Lorenz, Michael
AU - Vöhringer, Paul
AU - Schebek, Martin
AU - Timke, Christian
AU - Bobis, Ingrid
AU - Nüßlein, Thomas
AU - Dieninghoff, Doris
AU - Rietschel, Ernst
AU - Klinkhammer, Bastian
AU - Prenzel, Freerk
AU - Wald, Alexandra
AU - Kempa, Axel
AU - Brinkmann, Folke
AU - Lücke, Eva
AU - Adams, Ines
AU - Poplawska, Krystyna
AU - Lehmkühler, Simone
AU - Bauck, Monika
AU - Pfülb, Anne
AU - Fischer, Rainald
AU - Schopper, Gudrun
AU - Nährig, Susanne
AU - Griese, Matthias
AU - Grosse, Jörg
AU - Küster, Peter
AU - KinderHolger Köster, Birte
AU - Büsing, Susanne
AU - Pohl, Margarethe
AU - Schwarz, Carsten
AU - Artlich, Andreas
AU - Kiefer, Alexander
AU - Ballmann, Manfred
AU - Gjorgjevski, Nikola
AU - Rose, Markus A.
AU - Ruf, Friederike
AU - Mahlberg, Rolf
AU - Thomas, Wolfgang
AU - Graepler, Ute
AU - Bode, Sebastian
AU - hilipp Meyn
AU - Rosenecker, Josef
AU - Koerner, Cordula
AU - Keller, Klaus-Michael
AU - Teßmer, Tina
AU - Hebestreit, Helge
AU - Lohse, Gerhild
PY - 2023
DA - 2023/09/01
PB - Elsevier
SP - 100690
VL - 32
PMID - 37554663
SN - 2666-7762
ER -
BibTex
Cite this
BibTex (up to 50 authors) Copy
@article{2023_Sutharsan,
author = {Sivagurunathan Sutharsan and Stefanie Dillenhoefer and Matthias Welsner and Manuel Burkhart and Olaf Eickmeier and Matthias Kappler and Sarah Sieber and Susanne Naehrig and Lutz Naehrlich and Klaus Tenbrock and Claus Pfannenstiel and Dirk Steffen and Jochen Meister and Britta Welzenbach and Anette Scharschinger and Markus Kratz and Maike Pincus and Tobias Tenenbaum and Mirjam Stahl and Kerstin Landwehr and Stefanie Dillenhöfer and Hans Kössel and Petra Kaiser and Manfred Käding and Simone Stolz and Stefan Blaas and Jutta Hammermann and Monika Gappa and Antje Schuster and Dana Spittel and Sabine Zirlik and Sabina Schmitt and Florian Stehling and Sivagurunathan Sutharsan and Joachim Bargon and Malte Cremer and Christina Smaczny and Sebastian Fähndrich and Andrea Heinzmann and Lutz Nährlich and Stefan Kuhnert and Sebastian Schmidt and Bettina Wollschläger and Anna Nolde and Inka Held and Wolfgang Kamin and Felix C. Ringshausen and Anna-Maria Dittrich and Sabine Wege and Olaf Sommerburg and others},
title = {Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry},
journal = {The Lancet Regional Health - Europe},
year = {2023},
volume = {32},
publisher = {Elsevier},
month = {sep},
url = {https://doi.org/10.1016/j.lanepe.2023.100690},
pages = {100690},
doi = {10.1016/j.lanepe.2023.100690}
}