A5102939108- Cystic Fibrosis Research Advances
- Neonatal Respiratory Health Research
- Atomic and Subatomic Physics Research
- Tracheal and airway disorders
- Congenital Diaphragmatic Hernia Studies
- Advanced MRI Techniques and Applications
- Advanced NMR Techniques and Applications
- Respiratory Support and Mechanisms
- Chronic Obstructive Pulmonary Disease (COPD) Research
- Interstitial Lung Diseases and Idiopathic Pulmonary Fibrosis
- Transplantation: Methods and Outcomes
- Respiratory viral infections research
- Hematopoietic Stem Cell Transplantation
- Medical Imaging Techniques and Applications
- Pediatric health and respiratory diseases
- Inhalation and Respiratory Drug Delivery
- Breast Cancer Treatment Studies
- Cell Adhesion Molecules Research
- Coronary Artery Anomalies
- Immunodeficiency and Autoimmune Disorders
- NF-κB Signaling Pathways
- Pneumonia and Respiratory Infections
- Nosocomial Infections in ICU
- Antifungal resistance and susceptibility
- Mobile Health and mHealth Applications
British Columbia Children's Hospital
2018-2025
University of British Columbia
2018-2025
University of Pennsylvania
2023
Université de Montpellier
2023
University of Alabama at Birmingham
2023
Institut de Recherche en Infectiologie de Montpellier
2023
British Columbia Institute of Technology
2022
University of Manitoba
2021
Hospital for Sick Children
2013-2019
University of Toronto
2017-2019
Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients ≥12 years of age with cystic fibrosis at least one F508del-CFTR (cystic transmembrane conductance regulator) allele, but it has not been evaluated children <12 age. Objectives: To assess the safety, pharmacokinetics, efficacy ELX/TEZ/IVA 6 through 11 F508del-minimal function or F508del-F508del genotypes. Methods: In this 24-week open-label phase 3 study, (N = 66) weighing <30 kg...
Rationale: Elexacaftor/tezacaftor/ivacaftor has been shown to be safe and effective in people with cystic fibrosis (CF) aged ≥6 years ≥1 F508del-CFTR allele but not studied younger children. Objectives: To evaluate the safety, pharmacokinetics, pharmacodynamics, efficacy of elexacaftor/tezacaftor/ivacaftor children CF 2 through 5 years. Methods: In this Phase 3, open-label, two-part study (Parts A B), weighing <14 kg (on Day 1) received elexacaftor 80 mg once daily, tezacaftor 40 ivacaftor...
Hyperpolarized (HP) 129 Xe MRI uniquely images pulmonary ventilation, gas exchange, and terminal airway morphology rapidly safely, providing novel information not possible using conventional imaging modalities or function tests. As such, there is mounting interest in expanding the use of biomarkers derived from HP as outcome measures multi-site clinical trials across a range disorders. Until recently, techniques have been developed largely independently at limited number academic centers,...
A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children aged 6-11 years with cystic fibrosis (CF) one or more
Background Elevated markers of systemic and pulmonary inflammation are associated with failure to recover lung function following exacerbations in people cystic fibrosis (pwCF). Our aim was determine whether adjuvant oral prednisone treatment would improve recovery forced expiratory volume 1 s (FEV ) % pred CF not responding antibiotic therapy. Methods This a randomised, double-blind, placebo-controlled trial pwCF treated intravenous antibiotics for exacerbation. At day 7, those who had...
Abstract Myhre syndrome is an increasingly diagnosed ultrarare condition caused by recurrent germline autosomal dominant de novo variants in SMAD4 . Detailed multispecialty evaluations performed at the Massachusetts General Hospital (MGH) Syndrome Clinic (2016–2023) and collaborating specialists have facilitated deep phenotyping, genotyping natural history analysis. Of 47 patients (four previously reported), most (81%) returned to MGH least once. For followed for 5 years, symptom progression...
Pulmonary magnetic resonance imaging using hyperpolarised 129Xe gas (XeMRI) can quantify ventilation inhomogeneity by measuring the percentage of unventilated lung volume (ventilation defect per cent (VDP)). While previous studies have demonstrated its sensitivity for detecting early cystic fibrosis (CF) disease, utility XeMRI to monitor response therapy in CF is unknown. The aim this study was assess ability capture treatment paediatric patients undergoing inpatient antibiotic a pulmonary...
Background Antibiotic treatment for pulmonary symptoms in preschool children with cystic fibrosis (CF) varies among clinicians. The lung clearance index (LCI) is sensitive to early CF disease, but its utility monitor exacerbations young has not been assessed. Objective We aim (1) understand how LCI changes during lower respiratory tract relative a recent clinically stable measurement, (2) determine whether can identify antibiotic response and (3) compare spirometric indices. Methods...
Rationale: Previous phase 3 trials showed that treatment with lumacaftor/ivacaftor was safe and efficacious in people aged ⩾2 years cystic fibrosis (CF) homozygous for the F508del mutation CFTR (CF transmembrane conductance regulator) (F/F genotype). Objectives: To assess safety, pharmacokinetics, pharmacodynamics of children 1 to <2 F/F genotype. Methods: This open-label, study consisted two parts (part A [n = 14] part B 46]) which cohorts were enrolled on basis age (cohort 1, 18 <24 mo;...
The rapid growth in popularity of e-cigarettes over the past decade has prompted concerns about their impact on long-term respiratory health. Small airway injury is suspected to be a direct consequence e-cigarette use and may quantifiable by novel structural functional diagnostic modalities. In multicentre observational longitudinal study, participants will enrolled either an adolescent (ages ≥12 <19 years) or adult arm (≥19 years old) followed 3 across three time points (baseline, 18 months...
Importance A resurgence of respiratory syncytial virus (RSV)–associated acute tract infection (ARI) was observed in 2022 and 2023 after the COVID-19 pandemic. Changes demographic characteristics, disease severity, outcomes patients were observed, which could impact identification risk groups for interventions aimed at reducing severity RSV disease. Objectives To identify factors associated with severe clinical among children hospitalized RSV-associated ARIs 2023. Design, Setting,...
ABSTRACT MRI can provide localized assessment of lung function for monitoring people with disease. Hyperpolarized 129 Xe directly images pulmonary gas distribution but requires specialized hardware. Conventional 1 H acquisitions also functional maps using free‐breathing approaches. The purpose this study is to evaluate regional ventilation derived from 3D ultrashort echo‐time (UTE) Motion‐Compensated Low‐Rank constrained reconstruction (MoCoLoR), by comparing against and testing as...
Background Current diagnostic tests for pulmonary chronic graft versus host disease (p-cGvHD) are either invasive or challenging children to perform. Multiple breath washout (MBW) has been proposed as a feasible and sensitive tool the diagnosis of p-cGvHD. In this study, we aimed determine feasibility sensitivity MBW monitor p-cGvHD in after haematopoietic stem cell transplantation (HSCT). Methods This was prospective, single-center cohort recruiting >3 years age undergoing HSCT between...
Background Common variable immunodeficiency (CVID) is an inborn error of immunity (IEI) characterized by hypogammaglobulinemia and impaired specific antibody responses. This fundamental crippling predisposes affected individuals to respiratory infections, inflammation, malignancy, all which contribute CVID-associated lung disease. Early detection treatment disease in with CVID crucial for reducing both morbidity mortality. However, existing diagnostic techniques carry limitations, ranging...