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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.
Sutharsan, Sivagurunathan; Dillenhoefer, Stefanie; Welsner, Matthias; Stehling, Florian; Brinkmann, Folke; Burkhart, Manuel; Ellemunter, Helmut; Dittrich, Anna-Maria; Smaczny, Christina; Eickmeier, Olaf; Kappler, Matthias; Schwarz, Carsten; Sieber, Sarah; Naehrig, Susanne; Naehrlich, Lutz.
Afiliación
  • Sutharsan S; Department of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, Essen, Germany.
  • Dillenhoefer S; Department of Pediatric Pulmonology, Cystic Fibrosis Center, University Children's Hospital of Ruhr University Bochum at St. Josef-Hospital, Bochum, Germany.
  • Welsner M; Department of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, Essen, Germany.
  • Stehling F; Pediatric Pulmonology and Sleep Medicine, Children's University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
  • Brinkmann F; Department of Pediatric Pneumology & Allergology, The University of Lübeck, University Medical Center Schleswig-Holstein, Campus Centrum Lübeck, Member of Airway Research Center North (ARCN) of the German Center of Lung Research (DZL), Lübeck, Germany.
  • Burkhart M; Mukoviszidose Institut gGmbH (MI), Bonn, Germany.
  • Ellemunter H; Medical University of Innsbruck, Cystic Fibrosis Centre Innsbruck, Innsbruck, Austria.
  • Dittrich AM; Department of Paediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany.
  • Smaczny C; Biomedical Research in Endstage and Obstructive Lung Disease Hannover (BREATH), Member of the German Center for Lung Research (DZL), Germany.
  • Eickmeier O; University Hospital Frankfurt/Main, Goethe University, Pneumology and Allergology, Christiane Herzog CF Center Frankfurt/Main, Frankfurt/Main, Germany.
  • Kappler M; Pediatric Allergology, Pulmonology & Cystic Fibrosis, Christiane Herzog CF Center- Frankfurt a.M., University Hospital Frankfurt a.M., Germany.
  • Schwarz C; Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, LMU Munich, Germany.
  • Sieber S; Division Cystic Fibrosis, HMU-Health and Medical University Potsdam, Clinic Westbrandenburg, Potsdam, Germany.
  • Naehrig S; STAT-UP Statistical Consulting & Data Science GmbH, Munich, Germany.
  • Naehrlich L; Department of Internal Medicine V, Cystic Fibrosis Center for Adults, University Hospital, Ludwig Maximilian University (LMU) Munich, Germany.
Lancet Reg Health Eur ; 32: 100690, 2023 Sep.
Article en En | MEDLINE | ID: mdl-37554663
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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Idioma: En Revista: Lancet Reg Health Eur Año: 2023 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Idioma: En Revista: Lancet Reg Health Eur Año: 2023 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Reino Unido