Willmering, Matthew M Roach, David J Kramer, Elizabeth L Walkup, Laura L Cleveland, Zackary I Woods, Jason C
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
Two functional measurements (multiple breath washout [MBW] and hyperpolarized 129Xe ventilation magnetic resonance imaging [129Xe MRI]) have been shown to be more sensitive to cystic fibrosis (CF) lung obstruction than traditional spirometry. However, functional techniques may be sensitive to different underlying structural abnormalities. The purpo...
McElvaney, Oliver J O'Connor, Eoin McEvoy, Natalie L Fraughan, Daniel D Clarke, Jennifer McElvaney, Oisín F Gunaratnam, Cedric O'Rourke, James Curley, Gerard F McElvaney, Noel G
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Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
The clinical course of severe COVID-19 in cystic fibrosis (CF) is incompletely understood. We describe the use of alpha-1 antitrypsin (AAT) as a salvage therapy in a critically unwell patient with CF (PWCF) who developed COVID-19 while awaiting lung transplantation. IV AAT was administered at 120 mg/kg/week for 4 consecutive weeks. Levels of interl...
Moore, John E Millar, B Cherie McCaughan, John O'Neill, Damian Bell, Jane Crossan, Amanda Rendall, Jacqueline C
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
The COVID19 pandemic has shifted the paradigm of how outpatient clinics are delivered within CF care, resulting in a significant reduction of patient visits to CF centres. One consequence of this has been a reduction in the number of sputa/cough swabs that patients submit for routine analysis. This report examines why it is important to maintain op...
Harris, Caroline A Brodlie, Malcolm O'Brien, Christopher Thomas, Matthew F
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
We report the first case of biodegradable airway stent insertion for a patient with bronchomalacia and cystic fibrosis (CF). This female infant with antenatally diagnosed cystic fibrosis developed respiratory distress by three weeks of age. On lower airway examination she was found to have severe left main stem bronchomalacia causing left upper lob...
Berkers, Gitte van der Meer, Renske van Mourik, Peter Vonk, Annelotte M Kruisselbrink, Evelien Suen, Sylvia Wf Heijerman, Harry Gm Majoor, Christof J Koppelman, Gerard H Roukema, Jolt
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Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
The natural food supplements curcumin and genistein, and the drug ivacaftor were found effective as CFTR potentiators in the organoids of individuals carrying a S1251N gating mutation, possibly in a synergistic fashion. Based on these in vitro findings, we evaluated the clinical efficacy of a treatment with curcumin, genistein and ivacaftor, in dif...
Combret, Yann Medrinal, Clement Bonnevie, Tristan Gravier, Francis-Edouard Le Roux, Pascal Lamia, Bouchra Prieur, Guillaume Reychler, Gregory
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
Accurate testing of muscle function is essential in individuals with cystic fibrosis (CF). A literature search was conducted in MEDLINE, CENTRAL, CINAHL, PEDro, ScienceDirect and Web of Science according to PRISMA and COSMIN guidelines from inception to September 2019 to investigate the clinimetric properties of muscle tests in individuals with CF....
Laudus, Nele Audrézet, Marie-Pierre Girodon, Emmanuelle Morris, Michael A Radojkovic, Dragica Raynal, Caroline Seia, Manuela Štambergová, Alexandra Torkler, Heike Yamamoto, Raina
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Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
The clinical spectrum associated with cystic fibrosis transmembrane conductance regulator (CFTR) variant p.Arg117His is highly variable, ranging from full-blown cystic fibrosis (CF) in a small number of cases to CFTR-related disorders (CFTR-RDs) or no symptoms at all. Therefore, taking into account phenotype variability is essential for interpretat...
Voldby, Christian Green, Kent Kongstad, Thomas Ring, Astrid Madsen Sandvik, Rikke Mulvad Skov, Marianne Buchvald, Frederik Pressler, Tacjana Nielsen, Kim Gjerum
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
Using increase in the lung clearance index (LCI) as a trigger for bronchoalveolar lavage (BAL) and associated antimicrobial treatment might benefit clinical outcomes in children with cystic fibrosis (CF). A 2-year, longitudinal, interventional, randomized, controlled pilot study with quarterly visits in 5-18 years old children with CF. LCI and z-sc...
Munck, Anne Kerem, Eitan Ellemunter, Helmut Campbell, Daniel Wang, Linda T Ahluwalia, Neil Owen, Caroline A Wainwright, Claire
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
Tezacaftor/ivacaftor is a CFTR modulator approved to treat people with cystic fibrosis (pwCF) who are homozygous (F/F) or heterozygous for the F508del-CFTR mutation and a residual function mutation (F/RF). This randomized, double-blind, placebo-controlled Phase 3 study evaluated the efficacy, safety, tolerability, and pharmacokinetics (PK) of tezac...
Shaw, Michelle Khan, Umer Clancy, John P Donaldson, Scott H Sagel, Scott D Rowe, Steven M Ratjen, Felix
Published in
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
The PROSPECT study, a post-approval observational study in the U.S., showed no significant changes in lung function as measured by spirometry with clinical initiation of lumacaftor/ivacaftor. A sub-study within the PROSPECT study assessed the lung clearance index (LCI), as measured by multiple breath washout (MBW), a measure of lung function demons...
