Details

Autor(en) / Beteiligte

• Sivagurunathan Sutharsan
• Stefanie Dillenhoefer
• Matthias Welsner
• Florian Stehling
• Folke Brinkmann
• Manuel Burkhart
• Helmut Ellemunter
• Anna-Maria Dittrich
• Christina Smaczny
• Olaf Eickmeier
• Matthias Kappler
• Carsten Schwarz
• 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
• Sabina Schmitt
• Joachim Bargon
• Malte Cremer
• Sebastian Fähndrich
• Andrea Heinzmann
• Lutz Nährlich
• Stefan Kuhnert
• Sebastian Schmidt
• Bettina Wollschläger
• Anna Nolde
• Wolfgang Kamin
• Felix C. Ringshausen
• Sabine Wege
• Olaf Sommerburg
• Sara Lisa Fleser
• Heinrike Wilkens
• Michael Lorenz
• Paul Vöhringer
• Martin Schebek
• Christian Timke
• Ingrid Bobis
• Thomas Nüßlein
• Doris Dieninghoff
• Ernst Rietschel
• Bastian Klinkhammer
• Freerk Prenzel
• Alexandra Wald
• Axel Kempa
• 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
• Margarethe Pohl
• Andreas Artlich
• Alexander Kiefer
• Nikola Gjorgjevski
• Markus A. Rose
• Friederike Ruf
• Rolf Mahlberg
• Wolfgang Thomas
• Ute Graepler
• Sebastian Bode
• hilipp Meyn
• Cordula Koerner
• Klaus-Michael Keller
• Tina Teßmer
• Helge Hebestreit
• Gerhild Lohse

Titel

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 RegistryResearch in context

Ist Teil von

• The Lancet regional health. Europe, 2023-09, Vol.32, p.100690

Ort / Verlag

Elsevier

Erscheinungsjahr

2023

Link zum Volltext

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• Background: 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. Methods: 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. Findings: 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. Interpretation: 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. Funding: None.