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Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society

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https://www.readbyqxmd.com/read/29903467/combination-potentiator-co-potentiator-therapy-for-cf-caused-by-cftr-mutants-including-n1303k-that-are-poorly-responsive-to-single-potentiators
#1
Puay-Wah Phuan, Jung-Ho Son, Joseph-Anthony Tan, Clarabella Li, Ilaria Musante, Lorna Zlock, Dennis W Nielson, Walter E Finkbeiner, Mark J Kurth, Luis J Galietta, Peter M Haggie, Alan S Verkman
BACKGROUND: Current modulator therapies for some cystic fibrosis-causing CFTR mutants, including N1303K, have limited efficacy. We provide evidence here to support combination potentiator (co-potentiator) therapy for mutant CFTRs that are poorly responsive to single potentiators. METHODS: Functional synergy screens done on N1303K and W1282X CFTR, in which small molecules were tested with VX-770, identified arylsulfonamide-pyrrolopyridine, phenoxy-benzimidazole and flavone co-potentiators...
June 11, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29887518/physiological-and-pharmacological-characterization-of-the-n1303k-mutant-cftr
#2
Samantha DeStefano, Maarten Gees, Tzyh-Chang Hwang
BACKGROUND: N1303K, one of the common, severe disease-causing mutations in the CFTR gene, causes both defective biogenesis and gating abnormalities of the CFTR protein. The goals of the present study are to quantitatively assess the gating defects associated with the N1303K mutation and its pharmacological response to CFTR modulators including potentiators VX-770 and GLPG1837 and correctors VX-809, and VX-661. METHODS: Gating behavior and pharmacological responses to CFTR potentiators were assessed using patch-clamp technique in the excised, inside-out mode...
June 7, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29885744/continuous-glucose-monitoring-guided-insulin-therapy-is-associated-with-improved-clinical-outcomes-in-cystic-fibrosis-related-diabetes
#3
Freddy Frost, Paula Dyce, Dilip Nazareth, Victoria Malone, Martin J Walshaw
INTRODUCTION: Continuous glucose monitoring (CGM) allows assessment of day to day glycaemic excursions and detects early glucose handling abnormalities that may not be apparent on oral glucose tolerance testing (OGTT). However, there is little published evidence as to whether these early dysglycaemic changes are amenable to treatment. We present outcomes following CGM guided insulin initiation at our centre. METHODS: Adults without a prior diagnosis of cystic fibrosis related diabetes (CFRD) whom underwent >72 h CGM at our adult CF centre were included in the study...
June 6, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29884450/interleukin-1-is-associated-with-inflammation-and-structural-lung-disease-in-young-children-with-cystic-fibrosis
#4
Samuel T Montgomery, A Susanne Dittrich, Luke W Garratt, Lidija Turkovic, Dario L Frey, Stephen M Stick, Marcus A Mall, Anthony Kicic
BACKGROUND: Little is known about the role of interleukin (IL)-1 in the pathogenesis of cystic fibrosis (CF) lung disease. This study investigated the relationship between IL-1 signalling, neutrophilic inflammation and structural lung changes in children with CF. METHODS: Bronchoalveolar lavage fluid (BALf) from 102 children with CF were used to determine IL-1α, IL-1β, IL-8 levels and neutrophil elastase (NE) activity, which were then correlated to structural lung changes observed on chest computed tomography (CT) scans...
June 5, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29866531/measured-fetal-and-neonatal-exposure-to-lumacaftor-and-ivacaftor-during-pregnancy-and-while-breastfeeding
#5
Aaron Trimble, Cameron McKinzie, Mary Terrell, Elizabeth Stringer, Charles R Esther
With the growing class of CFTR modulator therapy available to more patients and with increasing pregnancies in individuals with CF, there is a growing need to understand the effects of these agents during pregnancy. There are few reports of their continued use in the literature, although it is likely that this is not an uncommon occurrence. We report the uncomplicated and successful pregnancy of a woman treated with lumacaftor/ivacaftor, as well as the clinical course of the infant during the first 9 months of life...
June 1, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29866530/cystic-fibrosis-beyond-the-airways-report-on-the-meeting-of-the-basic-science-working-group-in-loutraki-greece
#6
REVIEW
Margarida D Amaral, Sylvia F Boj, James Shaw, Jens Leipziger, Jeffrey M Beekman
The European Cystic Fibrosis Society (ECFS) Basic Science Working Group (BSWG) organized a session on the topic "Cystic Fibrosis: Beyond the Airways", within the 15th ECFS Basic Science Conference which gathered around 200 researchers working in the basic science of CF. The session was organized and chaired by Margarida Amaral (BioISI, University of Lisboa, Portugal) and Jeffrey Beekman (University Medical Centre Utrecht, Netherlands) as Chair and Vice-Chair of the BSWG and its purpose was to bring attention of participants of the ECFS Basic Science Conference to "more forgotten" organs in CF disease...
June 1, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29859835/back-to-the-source-modern-insights-into-pulmonary-exacerbations-and-lung-function-decline-from-cf-registry-data
#7
EDITORIAL
Jonathan H Rayment, Bradley S Quon
No abstract text is available yet for this article.
May 30, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29859834/no-easy-road-to-better-cystic-fibrosis-care-in-eastern-europe
#8
EDITORIAL
Kris De Boeck, Isabelle Fajac
No abstract text is available yet for this article.
May 30, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29807875/primary-sclerosing-cholangitis-is-associated-with-abnormalities-in-cftr
#9
Steven Werlin, Virginie Scotet, Kevin Uguen, Marie-Pierre Audrezet, Michael Cohen, Yasmin Yaakov, Rifaat Safadi, Yaron Ilan, Fred Konikoff, Eitan Galun, Meir Mizrahi, Mordechai Slae, Shirley Sayag, Malena Cohen-Cymberknoh, Michael Wilschanski, Claude Ferec
BACKGROUND: The etiology of primary sclerosing cholangitis (PSC) is unknown. PSC and Cystic Fibrosis related liver disease have common features: chronic inflammation, biliary damage and similar cholangiographic findings. It is unknown whether or not PSC is related to cystic fibrosis transmembrane conductance regulator (CFTR) dysfunction. We hypothesize that a sub-group of PSC patients may be a "single-organ" presentation of CF. METHODS: Patients with PSC underwent nasal potential difference (NPD) measurement, sweat chloride measurement and complete CFTR sequencing by new generation sequencing...
May 25, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29805051/emergence-of-livestock-associated-mrsa-isolated-from-cystic-fibrosis-patients-result-of-a-belgian-national-survey
#10
M Dodémont, M A Argudín, J Willekens, E Vanderhelst, D Pierard, V Y Miendje Deyi, L Hanssens, H Franckx, P Schelstraete, I Leroux-Roels, C Nonhoff, A Deplano, C Knoop, A Malfroot, O Denis
BACKGROUND: This study aims to determine the prevalence and characteristics of Staphylococcus aureus in Belgian cystic fibrosis (CF) patients. METHODS: Non-duplicate respiratory samples from 510 CF-patients (2012-2013) were examined. One isolate per patient was analysed unless different phenotypes were recovered. Isolates were investigated for mecA/mecC, toxins presence, spa-typing, MLST and SCCmec-typing. Potential livestock-associated (LA) isolates were examined for their immune-evasion-cluster (IEC) genes...
May 24, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29805050/multicentre-standardisation-of-chest-mri-as-radiation-free-outcome-measure-of-lung-disease-in-young-children-with-cystic-fibrosis
#11
Mark O Wielpütz, Oyunbileg von Stackelberg, Mirjam Stahl, Bertram J Jobst, Monika Eichinger, Michael U Puderbach, Lutz Nährlich, Sandra Barth, Christian Schneider, Matthias V Kopp, Isabell Ricklefs, Michael Buchholz, Burkhard Tümmler, Christian Dopfer, Jens Vogel-Claussen, Hans-Ulrich Kauczor, Marcus A Mall
BACKGROUND: A recent single-centre study demonstrated that MRI is sensitive to detect early abnormalities in the lung and response to therapy in infants and preschool children with cystic fibrosis (CF) supporting MRI as an outcome measure of early CF lung disease. However, the feasibility of multicentre standardisation remains unknown. OBJECTIVE: To determine the feasibility of multicentre standardisation of chest MRI in infants and preschool children with CF. METHODS: A standardised chest 1...
May 24, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29748153/a-prospective-analysis-of-unplanned-patient-initiated-contacts-in-an-adult-cystic-fibrosis-centre
#12
Espérie Burnet, Dominique Hubert, Juliette Champreux, Isabelle Honoré, Reem Kanaan, Rosewilta Panzo, Pierre-Régis Burgel
BACKGROUND: Timely response should be provided when patients contact the cystic fibrosis (CF) centre in between scheduled visits. Little data exist on unplanned patient-initiated contacts in CF adults. METHODS: A two-stage prospective study was undertaken from 1 January to 31 December 2015 at Cochin Hospital, Paris (France). The first stage included all adults (≥18 years) who initiated unplanned contacts to the CF centre over four months. Four physicians and three nurses systematically recorded unplanned patient-initiated contacts...
May 7, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29735383/apolipoprotein-e-polymorphism-determines-vitamin-k-supplementation-effectiveness-in-cystic-fibrosis-patients
#13
LETTER
Patrycja Krzyżanowska-Jankowska, Paulina Sobkowiak, Olaf Thalmann, Sławomira Drzymała-Czyż, Aleksandra Glapa, Marcin Hołysz, Dariusz Walkowiak, Nataliya Rohovyk, Lyudmyla Bober, Andrzej Pogorzelski, Jarosław Walkowiak
No abstract text is available yet for this article.
May 4, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29724576/dietary-intake-of-energy-dense-nutrient-poor-and-nutrient-dense-food-sources-in-children-with-cystic-fibrosis
#14
Rosie Sutherland, Tamarah Katz, Victoria Liu, Justine Quintano, Rebecca Brunner, Chai Wei Tong, Clare E Collins, Chee Y Ooi
BACKGROUND: Prescription of a high-energy, high-fat diet is a mainstay of nutrition management in cystic fibrosis (CF). However, families may be relying on energy-dense, nutrient-poor (EDNP) foods rather than nutrient-dense (ND) foods to meet dietary targets. We aimed to evaluate the relative contribution of EDNP and ND foods to the usual diets of children with CF and identify sociodemographic factors associated with higher EDNP intakes. METHODS: This is a cross-sectional comparison of children with CF aged 2-18 years and age- and gender-matched controls...
April 30, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29685811/effectiveness-of-ivacaftor-in-cystic-fibrosis-patients-with-non-g551d-gating-mutations
#15
Jennifer Guimbellot, George M Solomon, Arthur Baines, Sonya L Heltshe, Jill VanDalfsen, Elizabeth Joseloff, Scott D Sagel, Steven M Rowe
BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator ivacaftor is approved for patients with CF with gating and residual function CFTR mutations. We report the results of an observational study investigating its effects in CF patients with non-G551D gating mutations. METHODS: Patients with non-G551D gating mutations were recruited to an open-label study evaluating ivacaftor. Primary outcomes included: lung function, sweat chloride, weight gain, and quality of life scores...
April 21, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29685813/eradication-of-early-p-aeruginosa-infection-in-children-7-years-of-age-with-cystic-fibrosis-the-early-study
#16
Felix Ratjen, Alexander Moeller, Martha L McKinney, Irina Asherova, Nipa Alon, Robert Maykut, Gerhild Angyalosi
OBJECTIVE: Antibiotic eradication treatment is the standard-of-care for cystic fibrosis (CF) patients with early Pseudomonas aeruginosa (Pa)-infection; however, evidence from placebo-controlled trials is limited. METHODS: This double-blind, placebo-controlled trial randomised CF patients <7 years (N = 51) with early Pa-infection to tobramycin inhalation solution (TOBI 300 mg) or placebo (twice daily) for 28 days with an optional cross-over on Day 35...
April 20, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29685812/the-cf-canada-sick-kids-program-in-individual-cf-therapy-a-resource-for-the-advancement-of-personalized-medicine-in-cf
#17
Paul D W Eckford, Jacqueline McCormack, Lise Munsie, Gengming He, Sanja Stanojevic, Sergio L Pereira, Karen Ho, Julie Avolio, Claire Bartlett, Jin Ye Yang, Amy P Wong, Leigh Wellhauser, Ling Jun Huan, Jia Xin Jiang, Hong Ouyang, Kai Du, Michelle Klingel, Lianna Kyriakopoulou, Tanja Gonska, Theo J Moraes, Lisa J Strug, Janet Rossant, Felix Ratjen, Christine E Bear
BACKGROUND: Therapies targeting certain CFTR mutants have been approved, yet variations in clinical response highlight the need for in-vitro and genetic tools that predict patient-specific clinical outcomes. Toward this goal, the CF Canada-Sick Kids Program in Individual CF Therapy (CFIT) is generating a "first of its kind", comprehensive resource containing patient-specific cell cultures and data from 100 CF individuals that will enable modeling of therapeutic responses. METHODS: The CFIT program is generating: 1) nasal cells from drug naïve patients suitable for culture and the study of drug responses in vitro, 2) matched gene expression data obtained by sequencing the RNA from the primary nasal tissue, 3) whole genome sequencing of blood derived DNA from each of the 100 participants, 4) induced pluripotent stem cells (iPSCs) generated from each participant's blood sample, 5) CRISPR-edited isogenic control iPSC lines and 6) prospective clinical data from patients treated with CF modulators...
April 20, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29685810/pulmonary-exacerbations-and-acute-declines-in-lung-function-in-patients-with-cystic-fibrosis
#18
Jeffrey S Wagener, Michael J Williams, Stefanie J Millar, Wayne J Morgan, David J Pasta, Michael W Konstan
BACKGROUND: Patients with cystic fibrosis (CF) who experience acute declines in percent predicted FEV1 (ppFEV1 decreased ≥10% relative to baseline) are often not treated with antibiotics for pulmonary exacerbations (PEx), whereas other patients are treated even when they have not experienced a decline in lung function. METHODS: We analyzed 2 patient cohorts using 3 years of Epidemiologic Study of CF data. Cohort 1 (12,837 patients) experienced a ≥10% acute decline in ppFEV1 (n = 22,898) and Cohort 2 (10,416 patients) had a clinician-diagnosed PEx (n = 20,731)...
April 20, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29681443/insights-into-the-cystic-fibrosis-care-in-eastern-europe-results-of-survey
#19
Katarzyna Walicka-Serzysko, Monika Peckova, Jacquelien J Noordhoek, Dorota Sands, Pavel Drevinek
BACKGROUND: The European cystic fibrosis (CF) Society Standards of Care were set to facilitate the delivery of high-quality care throughout Europe. However, their implementation may be difficult for less economically advantaged countries. This survey was performed to explore the gap in the knowledge of the level of CF care in Eastern Europe. METHODS: Questionnaires were sent online to one CF professional and one CF patient representative for every Eastern European country...
April 19, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29680365/prevalence-of-hypoglycemia-during-oral-glucose-tolerance-testing-in-adults-with-cystic-fibrosis-and-risk-of-developing-cystic-fibrosis-related-diabetes
#20
Lisa A Mannik, Kristy A Chang, Pascalyn Q K Annoh, Jenna Sykes, Julie Gilmour, Ronalee Robert, Anne L Stephenson
BACKGROUND: Hypoglycemia in cystic fibrosis (CF) patients during the oral glucose tolerance test (OGTT) has been reported; however, these patients have not been well-characterized. Few studies have examined whether hypoglycemia during the OGTT increases the risk of developing CF-related diabetes (CFRD). Objectives of this study were to describe the characteristics of CF patients with hypoglycemia during the OGTT and to determine the incidence and time to development of CFRD in those with hypoglycemia...
April 18, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
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