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. 2025 Mar 3;11(2):00701-2024.
doi: 10.1183/23120541.00701-2024. eCollection 2025 Mar.

Efficacy and safety of CFTR modulators in patients with interstitial lung disease caused by ABCA3 transporter deficiency

Mathilde Le Brun  1   2 , Nadia Nathan  3   4   5 , Camille Louvrier  3   4   5   6 , Marie Legendre  5   6 , Severine Feuillet  7 , Justine Frija-Masson  8 , Mwetty Onanga  1 , Lise Morer  9   10 , Marie-Pierre Debray  10   11 , Isabelle Fajac  10   12 , Pierre-Régis Burgel  10   13 , Sylvain Marchand-Adam  14 , Bruno Crestani  1   2 , Raphaël Borie  1   2
Affiliations

Efficacy and safety of CFTR modulators in patients with interstitial lung disease caused by ABCA3 transporter deficiency

Mathilde Le Brun et al. ERJ Open Res. .

Abstract

CFTR modulators may be valuable therapy for patients with ABCA3 pathogenic variants https://bit.ly/3TMWKK9.

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Conflict of interest statement

Conflict of interest: M. Le Brun declares conflicts of interest with GSK, AstraZeneca, Sanofi, Novartis, Asten Santé, Boehringer Ingelheim and La revue du praticien (payment for articles and presentations, and support for attending meetings). Conflict of interest: N. Nathan received grants or contracts (CORTICONEHI: clinical trial, Million Dollar Bike Ride, Chancellerie des Universités and Respifil) and payment for articles (La lettre du pneumologue). Conflict of interest: C. Louvrier has nothing to disclose. Conflict of interest: M. Legendre has nothing to disclose. Conflict of interest: S. Feuillet has nothing to disclose. Conflict of interest: J. Frija-Masson received grants or contracts (LVL Medical) and declares conflicts of interest with SOS Oxygène (payment for presentations), and Vitalair, SOS Oxygène, LVL Medical and ADEP Assistance (support for attending meetings). Conflict of interest: M. Onanga has nothing to disclose. Conflict of interest: L. Morer has nothing to disclose. Conflict of interest: M-P. Debray declares conflicts of interest with Sanofi and Boehringer Ingelheim (payment for presentations and support for attending meetings). Conflict of interest: I. Fajac received grants or contracts (Abbvie, Bayer, Boehringer Ingelheim, Insmed, GSK, Vertex Pharmaceuticals and Zambon), consulting fees (Abbvie, Boehringer-Ingelheim, Genvade, Kither Biotech and Vertex Pharmaceuticals) and payment for presentations (Vertex Pharmaceuticals), and is President of the European Cystic Fibrosis Society. Conflict of interest: P-R. Burgel received grants or contracts (GSK and Vertex Pharmaceuticals), consulting fees (AstraZeneca, Chiesi, Insmed, GSK, Vertex Pharmaceuticals, Zambon, MSD and Viatris) and support for attending meetings (AstraZeneca and Chiesi). Conflict of interest: S. Marchand-Adam declares an unrestricted research grant to their institution from Boehringer Ingelheim, consulting fees (in 2023) from AstraZeneca and Boehringer Ingelheim, and support for attending meetings (in 2023) from Boehringer Ingelheim. Conflict of interest: B. Crestani declares conflicts of interest with AstraZeneca, BMS, Boehringer Ingelheim, GSK, Novartis, Roche and Sanofi (payment for presentations, monitoring boards and support for attending meetings), and is the President of the board of trustees of the Fondation du Souffle (a French charity). Conflict of interest: R. Borie declares conflicts of interest with Sanofi, Boehringer Ingelheim and Ferrer (payment for presentations and support for attending meetings).

Figures

FIGURE 1
FIGURE 1
Evolution of CT scan and PFT before and after elexacaftor/tezacaftor/ivacaftor (ETI). Case 1, a) CT scan at diagnosis; b) after prednisone; c) after 3 months of ETI; d) evolution of PFT beginning from diagnosis. Case 2, e) CT scan in 2007 at the age of 8; f) before ETI; g) after 3 months of ETI; h) evolution of PFT beginning from 2010 at the age of 11. CT scan showed improvement of condensation (arrow) after ETI. Case 3, I)) CT scan at diagnosis; j) before ETI; k) after 6 months of ETI; l) evolution of PFT beginning from diagnosis. CT: computed tomography; PFT: pulmonary function test; FVC: forced vital capacity, HCQ: hydroxychloroquine; AZI: azithromycin DLCO: diffusing capacity of the lungs for carbon monoxide.
See this image and copyright information in PMC

References

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