Heliyon
Department of Medicine, University of Alabama at Birmingham, AL, United States.
Published: April 2024
Background: Use of elexacaftor/tezacaftor/ivacaftor (ETI) for treatment of cystic fibrosis (CF) has resulted in unprecedented clinical improvements necessitating development of outcome measures for monitoring disease course. Intranasal micro-optical coherence tomography (μOCT) has previously helped detect and characterize mucociliary abnormalities in patients with CF. This study was done to determine if μOCT can define the effects of ETI on nasal mucociliary clearance and monitor changes conferred to understand mechanistic effects of CFTR modulators beyond CFTR activation.
Methods: 26 subjects, with at least 1 F508del mutation were recruited and followed at baseline (visit 1), +1 month (visit 2) and +6 months (visit 4) following initiation of ETI therapy. Clinical outcomes were computed at visits 1, 2 and 4. Intranasal μOCT imaging and functional metrics analysis including mucociliary transport rate (MCT) estimation were done at visits 1 and 2.
Results: Percent predicted forced expiratory volume in 1 s (ppFEV) showed a significant increase of +10.9 % at visit 2, which sustained at visit 4 (+10.6 %). Sweat chloride levels significantly decreased by -36.6 mmol/L and -41.3 mmol/L at visits 2 and 4, respectively. μOCT analysis revealed significant improvement in MCT rate (2.8 ± 1.5, visit 1 vs 4.0 ± 1.5 mm/min, visit 2; P = 0.048).
Conclusions: Treatment with ETI resulted in significant and sustained clinical improvements over 6 months. Functional improvements in MCT rate were evident within a month after initiation of ETI therapy indicating that μOCT imaging is sensitive to the treatment effect of HEMT and suggests improved mucociliary transport as a probable mechanism of action underlying the clinical benefits.
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http://dx.doi.org/10.1016/j.heliyon.2024.e29188 | DOI Listing |
Turk Arch Pediatr
January 2025
Department of Pediatric Pulmonology, Marmara University School of Medicine, İstanbul, Türkiye.
Objective: Nebulizer contamination has potential harmful effects on the respiratory system. The aim was to investigate the contamination profile of the nebulizers in cystic fibrosis patients and evaluate the relationship between hygiene practices and microbial contamination. Materials and Methods: Microbiological swab samples were taken from 3 different locations of the nebulizers of 102 patients.
View Article and Find Full Text PDFJ Cyst Fibros
January 2025
Department NEUROFARBA, University of Florence; Paediatric and Liver Unit, Meyer Children's Hospital IRCCS, Florence, Italy.
Background: Elexacaftor-tezacaftor-ivacaftor (ETI) has significantly improved the clinical course of people with cystic fibrosis (pwCF) and eligible CFTR variants. In this study, we prospectively evaluated liver elastography, liver fibrosis indices and liver tests in children with CF aged 6-12 years started on ETI therapy.
Methods: Body mass index, sweat test, percent predicted forced expiratory volume in one second, serum markers of liver injury or portal hypertension, liver fibrosis indices, controlled attenuation parameter and liver stiffness were assessed before starting ETI and three and twelve months post-ETI, according to new international guidelines.
J Cyst Fibros
January 2025
The Lundquist Institute, Harbor-UCLA Medical Center, Torrance 90502 CA, USA. Electronic address:
Background: Cystic Fibrosis-related Bone Disease is an emerging challenge faced by 50 % of adult people with cystic fibrosis (CF). The multifactorial causes of this comorbidity remain elusive. However, congenital bone defects have been observed in animal models with CFTR mutations, suggesting its importance.
View Article and Find Full Text PDFIntroduction: The Lung Clearance Index (LCI) is an established research test, but its role in clinical decision-making is not well defined. This study estimated the proportion of treatment decisions that are changed or supported by the added information provided by LCI.
Methods: A mixed methods prospective observational study was conducted in North America.
Dig Liver Dis
January 2025
Unit of Clinical and Molecular Epidemiology, IRCCS San Raffaele Pisana, 00166 Rome, Italy; Department of Human Sciences and Quality of Life Promotion, San Raffaele University, 00166 Rome, Italy.
Background: In pediatric patients, celiac disease (CD) may influence the health-related quality of life (HRQoL).
Aims: The study aimed to assess HRQoL and further characterise the clinical factors associated with reduced HRQoL, in a large multicenter pediatric cohort with CD.
Methods: The disease-specific questionnaire CD Dutch Questionnaire (CDDUX) and the generic questionnaire Paediatric Quality of Life Inventory (PedsQL) were used to assess the HRQoL.
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