Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two F508del alleles
Extract: Clinical trials of the triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) demonstrated unprecedented clinical benefits including improvements in lung function, respiratory symptoms and nutritional outcomes i...
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| Hauptverfasser: | , , , , , , , , , , , , , , , , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
5 September 2024
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| In: |
The European respiratory journal
Year: 2024, Jahrgang: 64, Heft: 3, Pages: 1-13 |
| ISSN: | 1399-3003 |
| DOI: | 10.1183/13993003.00004-2024 |
| Online-Zugang: | Verlag, kostenfrei, Volltext: https://doi.org/10.1183/13993003.00004-2024 Verlag, kostenfrei, Volltext: https://publications.ersnet.org/content/erj/64/3/2400004 
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| Verfasserangaben: | Mirjam Stahl, Martha Dohna, Simon Y. Graeber, Olaf Sommerburg, Diane M. Renz, Sophia T. Pallenberg, Andreas Voskrebenzev, Katharina Schütz, Gesine Hansen, Felix Doellinger, Eva Steinke, Stephanie Thee, Jobst Röhmel, Sandra Barth, Claudia Rückes-Nilges, Julian Berges, Susanne Hämmerling, Mark O. Wielpütz, Lutz Naehrlich, Jens Vogel-Claussen, Burkhard Tümmler, Marcus A. Mall and Anna-Maria Dittrich |
MARC
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| 245 | 1 | 0 | |a Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two F508del alleles |c Mirjam Stahl, Martha Dohna, Simon Y. Graeber, Olaf Sommerburg, Diane M. Renz, Sophia T. Pallenberg, Andreas Voskrebenzev, Katharina Schütz, Gesine Hansen, Felix Doellinger, Eva Steinke, Stephanie Thee, Jobst Röhmel, Sandra Barth, Claudia Rückes-Nilges, Julian Berges, Susanne Hämmerling, Mark O. Wielpütz, Lutz Naehrlich, Jens Vogel-Claussen, Burkhard Tümmler, Marcus A. Mall and Anna-Maria Dittrich |
| 246 | 3 | 0 | |a elexacaftor tezacaftor ivacaftor |
| 264 | 1 | |c 5 September 2024 | |
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| 520 | |a Extract: Clinical trials of the triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) demonstrated unprecedented clinical benefits including improvements in lung function, respiratory symptoms and nutritional outcomes in patients with cystic fibrosis (CF) aged ≥2 years with at least one copy of the F508del allele [1-11]. Despite the emergence of highly effective CFTR modulator therapy, chronic progressive lung disease remains the main cause of morbidity and mortality in patients with CF; however, early intervention with CFTR-directed therapeutics in children with CF now provides an opportunity to delay or even prevent irreversible lung damage. Because of the limited sensitivity of percentage predicted forced expiratory volume in 1 s (FEV<sub>1</sub>) to capture response to therapy in CF children with preserved spirometry [12, 13], more sensitive outcome measures such as multiple-breath washout (MBW) and lung imaging by computed tomography (CT) and magnetic resonance imaging (MRI) have been established [14-25]. A number of studies demonstrated that the LCI derived from MBW is sensitive to detect response to therapeutic interventions including CFTR modulators in children with CF with normal spirometry [9-15, 18]. | ||
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