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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/30104123/continuous-glucose-monitoring-in-youth-with-cystic-fibrosis-treated-with-lumacaftor-ivacaftor
#1
Angel Li, Tim Vigers, Laura Pyle, Edith Zemanick, Kristen Nadeau, Scott D Sagel, Christine L Chan
BACKGROUND: The effects of lumacaftor-ivacaftor therapy on glycemia have not been thoroughly investigated. Continuous glucose monitoring (CGM) provides detailed information about glycemic patterns and detects glucose abnormalities earlier than traditional screening tools for diabetes. METHODS: CGM measures, HbA1c, and oral glucose tolerance test (OGTT) results were collected and within-subject results compared in F508del homozygous youth with CF before and after initiation of lumacaftor-ivacaftor using the Wilcoxon signed-rank test...
August 10, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30100257/antisense-oligonucleotide-targeting-of-mrnas-encoding-enac-subunits-%C3%AE-%C3%AE-and-%C3%AE-improves-cystic-fibrosis-like-disease-in-mice
#2
Chenguang Zhao, Jeff Crosby, Tinghong Lv, Dong Bai, Brett P Monia, Shuling Guo
BACKGROUND: The epithelial sodium channel ENaC consists of three subunits encoded by Scnn1a, Scnn1b, and Scnn1g and increased sodium absorption through this channel is hypothesized to lead to mucus dehydration and accumulation in cystic fibrosis (CF) patients. METHODS: We identified potent and specific antisense oligonucleotides (ASOs) targeting mRNAs encoding the ENaC subunits and evaluated these ASOs in mouse models of CF-like lung disease. RESULTS: ASOs designed to target mRNAs encoding each ENaC subunit or a control ASO were administered directly into the lungs of mice...
August 10, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30098934/the-open-door-policy-an-important-and-under-recognised-activity-of-the-adult-cf-centre
#3
EDITORIAL
Anna Sze Tai, Sue Morey
No abstract text is available yet for this article.
August 8, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30089531/patient-reported-outcomes-time-for-a-new-approach
#4
EDITORIAL
Janice Abbott
No abstract text is available yet for this article.
August 5, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30060960/respiratory-rate-in-infants-with-cystic-fibrosis-throughout-the-first-year-of-life-and-association-with-lung-clearance-index-measured-shortly-after-birth
#5
Insa Korten, Elisabeth Kieninger, Sophie Yammine, Giulia Cangiano, Sylvia Nyilas, Pinelopi Anagnostopoulou, Florian Singer, Claudia E Kuehni, Nicolas Regamey, Urs Frey, Carmen Casaulta, Ben D Spycher, Philipp Latzin
BACKGROUND: Lung impairment in cystic fibrosis (CF) starts in infancy. However, tools to monitor early lung disease are limited. Respiratory rate (RR) as a key vital sign is easy to assess during sleep and is elevated during acute respiratory disease. Thus, elevated RR could indicate early lung impairment and potentially serve as a diagnostic tool in disease monitoring. METHODS: In a prospective cohort of infants with CF diagnosed by newborn screening and healthy controls RR was measured and respiratory symptoms reported weekly throughout infancy...
July 27, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30033373/the-q359k-t360k-mutation-causes-cystic-fibrosis-in-georgian-jews
#6
M Mei-Zahav, P Stafler, H Senderowitz, L Bentur, G Livnat, M Shteinberg, N Orenstein, L Bazak, D Prais, H Levine, M Gur, N Khazanov, L Simhaev, H Eliyahu, M Cohen, M Wilschanski, H Blau, H Mussaffi
BACKGROUND: The Q359K/T360K mutation, described in Jewish CF patients of Georgian decent, is of questionable clinical significance. METHODS: Clinical records of patients with the Q359K/T360K mutation from three CF centers were studied for phenotypic expression and putative mechanism of dysfunction. Computer models of mutant CFTR were constructed. RESULTS: Nine patients (4 homozygous) of Georgian Jewish origin were included. Age at diagnosis was 9...
July 19, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30030066/r560s-a-class-ii-cftr-mutation-that-is-not-rescued-by-current-modulators
#7
Nikhil T Awatade, Sofia Ramalho, Iris A L Silva, Verónica Felício, Hugo M Botelho, Eyleen de Poel, Annelotte Vonk, Jeffrey M Beekman, Carlos M Farinha, Margarida D Amaral
BACKGROUND: New therapies modulating defective CFTR have started to hit the clinic and others are in trial or under development. The endeavour of drug discovery for CFTR protein rescue is however difficult one since over 2000 mutations have been reported. For most of these, especially the rare ones, the associated defects, the respective functional class and their responsiveness to available modulators are still unknown. Our aim here was to characterize the rare R560S mutation using patient-derived materials (rectal biopsies and intestinal organoids) from one CF individual homozygous for this mutation, in parallel with cellular models expressing R560S-CFTR and to assess the functional and biochemical responses to CFTR modulators...
July 18, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30017327/impact-of-a-program-ensuring-consistent-response-to-acute-drops-in-lung-function-in-children-with-cystic-fibrosis
#8
Michael S Schechter, H Joel Schmidt, Ron Williams, Robert Norton, Deanna Taylor, Andrea Molzhon
BACKGROUND: Variation in CF pulmonary outcomes is multifactorial, but a significant component appears to be dependent upon differences in CF Center care. Previous investigations suggest that high performing CF centers are more consistent and proactive in the treatment of pulmonary exacerbations. We incorporated this approach into a program that could be bundled and shared with other CF Centers. METHODS: The reorganization of CF pulmonary care at the Children's Hospital of Richmond included the development of a pulmonary algorithm to define a standard response to changes in lung function and run charts to track process and outcome measures...
July 13, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/30005828/age-related-heterogeneity-in-dental-caries-and-associated-risk-factors-in-individuals-with-cystic-fibrosis-ages-6-20-years-a-pilot-study
#9
Donald L Chi, Margaret Rosenfeld, Lloyd Mancl, Whasun O Chung, Richard B Presland, Elise Sarvas, Marilynn Rothen, Alaa Alkhateeb, Sharon McNamara, Alan Genatossio, Isabel Virella-Lowell, Carlos Milla, JoAnna Scott
BACKGROUND: The literature conflicts regarding dental caries risk in cystic fibrosis (CF) relative to controls. METHODS: Prospective, observational study of age-related heterogeneity in caries rates and potential risk factors in individuals with CF ages 6-20 at a single clinic in Washington state (N=85). Caries rates for enrolled CF participants and historical controls from NHANES were compared using cubic spline regression models. Generalized linear regression models identified correlates of age and caries in CF...
July 10, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29983355/italian-and-north-american-dietary-intake-after-ivacaftor-treatment-for-cystic-fibrosis-gating-mutations
#10
Nina N Sainath, Joan Schall, Chiara Bertolaso, Carolyn McAnlis, Virginia A Stallings
BACKGROUND: In patients with cystic fibrosis (CF), ivacaftor treatment results in significant weight gain and the impact on diet has not been explored. METHODS: A study in 22 subjects (6.1-61.6 years) compared diet, energy balance, weight gain, and body composition, before and after three months of treatment in Italians and North Americans with CFTR gating mutations. RESULTS: With no differences between groups in energy or macronutrient intake at baseline, fat intake increased in all subjects, and both fat and energy intake increased in Italians...
July 5, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29960875/a-specialized-method-of-sputum-collection-and-processing-for-therapeutic-interventions-in-cystic-fibrosis
#11
O J McElvaney, C Gunaratnam, E P Reeves, N G McElvaney
Cystic fibrosis (CF) lung disease is characterized by aggressive neutrophil-dominated inflammation mediated in large part by neutrophil elastase (NE), an omnivorous protease released by activated or disintegrating neutrophils and a key therapeutic target. To date, several short-term studies have shown that anti-NE compounds can inhibit NE and have anti-inflammatory effects. However, progression to large-scale or multicenter clinical trials has been hampered by the fact that the current gold standard methodology of evaluating airway NE inhibition, bronchoalveolar lavage (BAL), is invasive, difficult to standardize across sites and excludes those with severe lung disease...
June 27, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29941319/electrochemical-measurement-of-membrane-cholesterol-correlates-with-cftr-function-and-is-hdac6-dependent
#12
Binyu Lu, Li Li, Molly Schneider, Craig A Hodges, Calvin U Cotton, James D Burgess, Thomas J Kelley
BACKGROUND: Previous studies have demonstrated that CF epithelial cells exhibit increased cholesterol content at the plasma membrane compared to wild type controls as measured by electrochemical methods. Microtubule dysregulation that impacts intracellular transport has also been identified in CF cells and is reversible with histone deacetylase 6 (HDAC6) inhibition, a regulator of tubulin acetylation. The hypothesis of this study is that increased membrane cholesterol content in CF cells is dependent on HDAC6 regulation...
June 22, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29941318/rna-sequencing-data-from-neutrophils-of-patients-with-cystic-fibrosis-reveals-potential-for-developing-biomarkers-for-pulmonary-exacerbations
#13
Kaiyu Jiang, Kerry E Poppenberg, Laiping Wong, Yanmin Chen, Drucy Borowitz, Danielle Goetz, Daniel Sheehan, Carla Frederick, Vincent M Tutino, Hui Meng, James N Jarvis
BACKGROUND: There is no effective way to predict cystic fibrosis (CF) pulmonary exacerbations (CFPE) before they become symptomatic or to assess satisfactory treatment responses. METHODS: RNA sequencing of peripheral blood neutrophils from CF patients before and after therapy for CFPE was used to create transcriptome profiles. Transcripts with an average transcripts per million (TPM) level > 1.0 and a false discovery rate (FDR) < 0.05 were used in a cosine K-nearest neighbor (KNN) model...
June 22, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29937318/agtr2-absence-or-antagonism-prevents-cystic-fibrosis-pulmonary-manifestations
#14
Rebecca J Darrah, Frank J Jacono, Neha Joshi, Anna L Mitchell, Abdus Sattar, Cara K Campanaro, Paul Litman, Jennifer Frey, David E Nethery, Eric S Barbato, Craig A Hodges, Harriet Corvol, Garry R Cutting, Michael R Knowles, Lisa J Strug, Mitchell L Drumm
BACKGROUND: Pulmonary disease remains the primary cause of morbidity and mortality for individuals with cystic fibrosis (CF). Variants at a locus on the X-chromosome containing the type 2 angiotensin II receptor gene (AGTR2) were identified by a large GWAS as significantly associating with lung function in CF patients. We hypothesized that manipulating the angiotensin-signaling pathway may yield clinical benefit in CF. METHODS: Genetic subset analysis was conducted on a local CF cohort to extend the GWAS findings...
June 21, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29937317/resistin-is-elevated-in-cystic-fibrosis-sputum-and-correlates-negatively-with-lung-function
#15
Osric A Forrest, Daniel M Chopyk, Yael Gernez, Milton R Brown, Carol K Conrad, Richard B Moss, Vin Tangpricha, Limin Peng, Rabindra Tirouvanziam
BACKGROUND: Resistin is an immunometabolic mediator that is elevated in several inflammatory disorders. A ligand for Toll-like receptor 4, resistin modulates the recruitment and activation of myeloid cells, notably neutrophils. Neutrophils are major drivers of cystic fibrosis (CF) lung disease, in part due to the release of human neutrophil elastase- and myeloperoxidase-rich primary granules, leading to tissue damage. Here we assessed the relationship of resistin to CF lung disease. METHODS: Resistin levels were measured in plasma and sputum from three retrospective CF cohorts spanning a wide range of disease...
June 21, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29936070/rescue-of-cftr-nbd2-mutants-n1303k-and-s1235r-is-influenced-by-the-functioning-of-the-autophagosome
#16
Qiangni Liu, Inna Sabirzhanova, Murali K Yanda, Emily A S Bergbower, Clément Boinot, William B Guggino, Liudmila Cebotaru
The missing phenylalanine at position 508, located in nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane regulator (CFTR), is the most common cystic fibrosis mutation. Severe disease-causing mutations also occur in NBD2. To provide information on potential therapeutic strategies for mutations in NBD2, we used a combination of biochemical, cell biological and electrophysiological approaches and newly created cell lines to study two disease-causing NBD2 mutants, N1303K and S1235R. We observed that neither was sensitive to E64, a cysteine protease inhibitor...
June 20, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29936069/spx-101-is-stable-in-and-retains-function-after-exposure-to-cystic-fibrosis-sputum
#17
Juliana I Sesma, Bryant Wu, Timothy J Stuhlmiller, David W Scott
BACKGROUND: In healthy lungs, epithelial sodium channel (ENaC) is regulated by short, palate, lung, and nasal clone 1 (SPLUNC1). In cystic fibrosis (CF), ENaC is hyperactivated in part due to a loss of SPLUNC1 function. We have developed SPX-101 to replace the lost function of SPLUNC1 in the CF lung. METHODS: Expression of SPLUNC1 was determined in sputum from healthy and CF donors. Stability of SPLUNC1, S18 (the ENaC regulatory domain of SPLUNC1), and SPX-101 was determined in sputum from CF donors and towards neutrophil elastase...
June 20, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29934203/cftr-modulator-theratyping-current-status-gaps-and-future-directions
#18
REVIEW
John Paul Clancy, Calvin U Cotton, Scott H Donaldson, George M Solomon, Donald R VanDevanter, Michael P Boyle, Martina Gentzsch, Jerry A Nick, Beate Illek, John C Wallenburg, Eric J Sorscher, Margarida D Amaral, Jeffrey M Beekman, Anjaparavanda P Naren, Robert J Bridges, Philip J Thomas, Garry Cutting, Steven Rowe, Anthony G Durmowicz, Martin Mense, Kris D Boeck, William Skach, Christopher Penland, Elizabeth Joseloff, Hermann Bihler, John Mahoney, Drucy Borowitz, Katherine L Tuggle
BACKGROUND: New drugs that improve the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein with discreet disease-causing variants have been successfully developed for cystic fibrosis (CF) patients. Preclinical model systems have played a critical role in this process, and have the potential to inform researchers and CF healthcare providers regarding the nature of defects in rare CFTR variants, and to potentially support use of modulator therapies in new populations...
June 19, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29934202/the-choice-of-lung-function-reference-equation-affects-clinical-trial-eligibility-results-from-a-cystic-fibrosis-cohort
#19
LETTER
Inger Hee Mathiesen, Andreas Ronit, Tacjana Pressler
No abstract text is available yet for this article.
June 19, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29921503/characteristics-and-outcomes-of-oral-antibiotic-treated-pulmonary-exacerbations-in-children-with-cystic-fibrosis
#20
Jordana E Hoppe, Brandie D Wagner, Frank J Accurso, Edith T Zemanick, Scott D Sagel
BACKGROUND: Pulmonary exacerbations (PEx) in children with cystic fibrosis (CF) are frequently treated in the outpatient setting with oral antibiotics. However, little is known about the characteristics of PEx managed on an outpatient basis and the effectiveness of oral antibiotic therapy. We sought to prospectively evaluate clinical and laboratory changes associated with oral antibiotic treatment for PEx. METHODS: Children with CF between 8 and 18 years of age prescribed two weeks of oral antibiotics for a PEx were eligible to enroll...
June 16, 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
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