European Respiratory Journal

, volume 64 , issue 3 , pages 2400004

Impact of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Children with Cystic Fibrosis and One or TwoF508delAlleles

Mirjam Stahl ^{1, 2, 3, 4}

Martha Dohna ^{2, 5}

Simon Y. Graeber ^{1, 2, 3, 4}

Olaf Sommerburg ^{2, 6, 7}

Diane Miriam Renz ⁵

Sophia T Pallenberg ^{8, 9}

Andreas Voskrebenzev ⁵

Katharina Schütz ^{8, 9}

Gesine Hansen ^{8, 9, 10}

Felix Doellinger ¹¹

Eva Steinke ^{1, 3, 4}

Stephanie Thee ^{1, 3, 4}

Jobst Röhmel ^{1, 3, 4}

Sandra Barth ^{12, 13}

Claudia Rückes-Nilges ^{12, 13}

Julian Berges ^{6, 7}

Susanne Hämmerling ⁶

Mark O Wielpütz ^{2, 7}

Lutz Naehrlich ^{12, 13, 14}

Jens Vogel-Claussen ^{5, 9, 14}

Burkhard Tümmler ^{8, 9, 14}

Marcus Mall ^{1, 3, 4, 14}

Anna-Maria Dittrich ^{8, 9, 14}

Hide authors affiliations Show authors affiliations: 14 affiliations

Berlin Institute of Health (BIH) at Charité – Universitätsmedizin Berlin, Berlin, Germany

Charité - Universitätsmedizin Berlin

Berlin Institute of Health at Charité - Universitätsmedizin Berlin

Department of Diagnostic and Interventional Radiology, University of Heidelberg, Heidelberg, Germany |

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany

Humboldt University of Berlin

Free University of Berlin

Charité - Universitätsmedizin Berlin

⁴

German Center for Lung Research (DZL), associated partner site, Berlin, Germany |

⁵

Department for Radiology, Hannover Medical School, Hannover, Germany |

⁶

Division of Pediatric Pulmonology & Allergy and Cystic Fibrosis Center, Department of Pediatrics, University of Heidelberg, Heidelberg, Germany |

⁷

Translational Lung Research Center Heidelberg (TLRC), German Center for Lung Research (DZL), University of Heidelberg, Heidelberg, Germany

Heidelberg University

German Center for Lung Research

⁸

Department for Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany |

⁹

German Center for Lung Research, Biomedical Research in Endstage and Obstructive Lung Disease (BREATH), Hannover Medical School, Hannover, Germany

Hannover Medical School

German Center for Lung Research

¹⁰

Cluster of Excellence RESIST (EXC 2155), German Research Foundation (DFG), Hannover Medical School, Hannover, Germany |

¹¹

Department of Radiology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany

Humboldt University of Berlin

Free University of Berlin

Charité - Universitätsmedizin Berlin

¹²

Department of Pediatrics, Justus-Liebig-University Giessen, Giessen, Germany |

¹³

Universities of Giessen and Marburg Lung Center (UGMLC), German Center for Lung Research (DZL), Giessen, Germany

Universities of Giessen and Marburg Lung Center

German Center for Lung Research

¹⁴

M.S., M.D., S.Y.G., O.S. contributed equally as first authors

Publication type: Journal Article

Publication date: 2024-06-20

European Respiratory Society (ERS)

European Respiratory Journal

scimago Q1

wos Q1

SJR: 4.502

CiteScore: 23.0

Impact factor: 21.0

ISSN: 09031936, 13993003

DOI: 10.1183/13993003.00004-2024

Copy DOI

PubMed ID: 38901883

Abstract

Background

We recently demonstrated that elexacaftor/tezacaftor/ivacaftor (ETI) improves the lung clearance index (LCI) and abnormalities in lung morphology detected by magnetic resonance imaging (MRI) in adolescent and adult patients with cystic fibrosis (CF). However, real-world data on the effect of ETI on these sensitive outcomes of lung structure and function in school-age children with CF have not been reported. The aim of this study was therefore to examine the effect of ETI on the LCI and the lung MRI score in children aged 6–11 years with CF and one or twoF508delalleles.

Methods

This prospective, observational, multicentre, post-approval study assessed the longitudinal LCI up to 12 months and the lung MRI score before and 3 months after initiation of ETI.

Results

A total of 107 children with CF including 40 heterozygous forF508deland a minimal function mutation (F/MF) and 67 homozygous forF508del(F/F) were enrolled in this study. Treatment with ETI improved the median (interquartile range (IQR)) LCI in F/MF (−1.0 (−2.0– −0.1); p<0.01) and F/F children (−0.8 (−1.9– −0.2); p<0.001) from 3 months onwards. Further, ETI improved the median (IQR) MRI global score in F/MF (−4.0 (−9.0–0.0); p<0.01) and F/F children (−3.5 (−7.3– −0.8); p<0.001).

Conclusions

ETI improves early abnormalities in lung ventilation and morphology in school-age children with CF and at least oneF508delallele in a real-world setting. Our results support early initiation of ETI to reduce or even prevent lung disease progression in school-age children with CF.

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Stahl M. et al. Impact of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Children with Cystic Fibrosis and One or TwoF508delAlleles // European Respiratory Journal. 2024. Vol. 64. No. 3. p. 2400004.

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RIS |

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RIS Copy

TY - JOUR

DO - 10.1183/13993003.00004-2024

UR - http://erj.ersjournals.com/lookup/doi/10.1183/13993003.00004-2024

TI - Impact of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Children with Cystic Fibrosis and One or TwoF508delAlleles

T2 - European Respiratory Journal

AU - Stahl, Mirjam

AU - Dohna, Martha

AU - Graeber, Simon Y.

AU - Sommerburg, Olaf

AU - Renz, Diane Miriam

AU - Pallenberg, Sophia T

AU - Voskrebenzev, Andreas

AU - Schütz, Katharina

AU - Hansen, Gesine

AU - Doellinger, Felix

AU - Steinke, Eva

AU - Thee, Stephanie

AU - Röhmel, Jobst

AU - Barth, Sandra

AU - Rückes-Nilges, Claudia

AU - Berges, Julian

AU - Hämmerling, Susanne

AU - Wielpütz, Mark O

AU - Naehrlich, Lutz

AU - Vogel-Claussen, Jens

AU - Tümmler, Burkhard

AU - Mall, Marcus

AU - Dittrich, Anna-Maria

PY - 2024

DA - 2024/06/20

PB - European Respiratory Society (ERS)

SP - 2400004

IS - 3

VL - 64

PMID - 38901883

SN - 0903-1936

SN - 1399-3003

ER -

BibTex |

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BibTex (up to 50 authors) Copy

@article{2024_Stahl,

author = {Mirjam Stahl and Martha Dohna and Simon Y. Graeber and Olaf Sommerburg and Diane Miriam Renz and Sophia T Pallenberg and Andreas Voskrebenzev and Katharina Schütz and Gesine Hansen and Felix Doellinger and Eva Steinke and Stephanie Thee and Jobst Röhmel and Sandra Barth and Claudia Rückes-Nilges and Julian Berges and Susanne Hämmerling and Mark O Wielpütz and Lutz Naehrlich and Jens Vogel-Claussen and Burkhard Tümmler and Marcus Mall and Anna-Maria Dittrich and others},

title = {Impact of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Children with Cystic Fibrosis and One or TwoF508delAlleles},

journal = {European Respiratory Journal},

year = {2024},

volume = {64},

publisher = {European Respiratory Society (ERS)},

month = {jun},

url = {http://erj.ersjournals.com/lookup/doi/10.1183/13993003.00004-2024},

number = {3},

pages = {2400004},

doi = {10.1183/13993003.00004-2024}

}

MLA

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MLA Copy

Stahl, Mirjam, et al. “Impact of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Children with Cystic Fibrosis and One or TwoF508delAlleles.” European Respiratory Journal, vol. 64, no. 3, Jun. 2024, p. 2400004. http://erj.ersjournals.com/lookup/doi/10.1183/13993003.00004-2024.

Publisher

European Respiratory Society (ERS)

Journal

European Respiratory Journal

scimago Q1

wos Q1

SJR

4.502

CiteScore

23.0

Impact factor

21.0

ISSN

09031936 (Print)

13993003 (Electronic)