Multicenter Study of Hyperpolarized Xenon Magnetic Resonance Imaging in Children with Cystic Fibrosis Following Initiation of Cystic Fibrosis Transmembrane Regulator Modulator Therapy (HyPOINT)
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Le résumé fourni par la source
Abstract Rationale Elexacaftor/tezacaftor/ivacaftor (ETI) has significantly improved lung function in people with cystic fibrosis (CF), prompting the need for outcome measures that can detect mild disease. In this new era of CFTR (CF transmembrane regulator) modulator therapy, more sensitive endpoints are required to evaluate the progression of early lung disease and to determine the efficacy of new CF therapies. Before the availability of highly effective therapies, xenon-129 magnetic resonance imaging (MRI) was shown to be more sensitive to regional ventilation changes compared with spirometry. Objectives To evaluate the longitudinal changes in pulmonary function and Xe MRI outcomes after treatment with ETI in children and young people with CF. Methods Lung function was assessed longitudinally at baseline and 1, 6, and 12 months after ETI treatment initiation in children and young people with CF between the ages of 6 and 18 years at four study sites. Ventilation defect percentage (VDP), reader defect percentage (RDP), lung clearance index (LCI) and forced expiratory volume in 1 second (FEV1) were reported. Results A total of 28 participants were enrolled; 25 completed at least baseline and one-month measurements. All four measures (RDP, VDP, LCI, and FEV1) improved at one month after ETI initiation, with mean (standard deviation) absolute changes of −1.2 (1.7) in LCI, 6.9 (12.3) in FEV1 % predicted, −4.3 (4.8) in VDP, and −7.8 (9.6) in RDP. Xe MRI outcomes (RDP and VDP) showed the largest relative treatment effects, with mean relative improvements of 43% and 72%, respectively. One-third of participants (8 of 25) had improvements in VDP and RDP but did not show improvements in FEV1. Conclusions Xe MRI captures sustained ventilation improvements after ETI initiation. Xe MRI metrics may provide a suitable endpoint for future interventional trials, particularly for people with CF with mild lung disease.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Multicenter Study of Hyperpolarized Xenon Magnetic Resonance Imaging in Children with Cystic Fibrosis Following Initiation of Cystic Fibrosis Transmembrane Regulator Modulator Therapy (HyPOINT)
- Date Crossref
- 01/12/2025
- Éditeur
- Oxford University Press (OUP)
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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University of Toronto The Hospital for Sick Children pays non établi dans la noticeUniversité ou école supérieure
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Hospital for Sick Children Research Insititute pays non établi dans la noticeÉtablissement de santé
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Dalhousie University Department of Community Health and Epidemiology pays non établi dans la noticeUniversité ou école supérieure
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Cincinnati Children's Hospital Medical Center Department of Pediatrics pays non établi dans la noticeÉtablissement de santé
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Pulmonary Associates pays non établi dans la noticeÉtablissement de santé
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University of Virginia Medical Center Department of Pediatrics pays non établi dans la noticeÉtablissement de santé
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University of Virginia pays non établi dans la noticeUniversité ou école supérieure
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University of Iowa Department of Radiology pays non établi dans la noticeUniversité ou école supérieure
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University of Wisconsin–Madison pays non établi dans la noticeUniversité ou école supérieure
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Center for Pulmonary Imaging Research pays non établi dans la noticeInstitution
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Cincinnati Children\'s Hospital Medical Center pays non établi dans la noticeÉtablissement de santé
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Department of Medical Imaging pays non établi dans la noticeInstitution
The Hospital for Sick Children — University of Toronto, Research Insititute — Hospital for Sick Children et Department of Community Health and Epidemiology — Dalhousie University, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.