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

• DOI: 10.1016/j.lanepe.2023.100690
• Publication Date: 2023-07-28T17:30:15Z
• Authors: Sivagurunathan Su...; Stefanie Dillenho...; 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; Sabine Zirlik; Sabina Schmitt; Florian Stehling; Sivagurunathan Su...; 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 KinderHolge...; 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
• Keywords: Ivacaftor; Vital capacity; Patient registry; Combination therapy

Treatment with elexacaftor/tezacaftor/ivacaftor (ETI) improves multiple clinical outcomes in people cystic fibrosis (pwCF) 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 a large group pwCF.This observational cohort used data from German CF Registry for pwCF who received therapy were followed up period 12 months.The included 2645 67 centres Germany (mean age 28.0 ± 11.5 years). Over first year after was initiated, percent predicted forced expiratory volume 1 s (ppFEV1) increased by 11.3% (95% confidence interval [CI] 10.8-11.8, p < 0.0001), body mass index (BMI) z-score 0.3 CI 0.3-0.4, 0.0001) individuals aged to <18 years BMI adults 1.4 kg/m2 1.3-1.4, exacerbations decreased 75.9% (p mean concentration 50.9 mmol/L -52.6, -49.3, 0.0001). Improvements ppFEV1 over greater had not previously transmembrane conductance regulator (CFTR) modulator (12.6% [95% 11.9-13.4] vs. 9.7% 9.0-10.5] those prior CFTR treatment.These are consistent findings randomised trials, support use as highly effective treatment option have allele.None.