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Cystic Fibrosis

Gavin Barlow, Alyn Morice
No abstract text is available yet for this article.
April 17, 2018: Journal of Antimicrobial Chemotherapy
Maggie A Middleton, Mehdi Layeghifard, Michelle Klingel, Sanja Stanojevic, Yvonne C W Yau, James E A Zlosnik, Adele Coriati, Felix A Ratjen, Elizabeth D Tullis, Anne Stephenson, Pearce Wilcox, Andreas Freitag, Mark Chilvers, Martha McKinney, Annick Lavoie, Pauline W Wang, David S Guttman, Valerie J Waters
RATIONALE: The extent of the genetic relatedness among Pseudomonas aeruginosa isolates and its impact on clinical outcomes in the cystic fibrosis (CF) population is poorly understood. OBJECTIVES: The objectives of this study were to determine the prevalence of clonal P. aeruginosa infection in Canada and to associate P. aeruginosa genotypes with clinical outcomes. METHODS: This was an observational study of adult and pediatric CF patients across Canada...
June 18, 2018: Annals of the American Thoracic Society
Ema Robinson, Kelvin D MacDonald, Kai Slaughter, Madison McKinney, Siddharth Patel, Conroy Sun, Gaurav Sahay
The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically realized despite years of effort toward correcting the underlying genetic defect in the cystic fibrosis transmembrane conductance regulator (CFTR). mRNA therapy via nanoparticle delivery represents a powerful technology for the transfer of genetic material to cells with large, widespread populations, such as airway epithelia. We deployed a clinically relevant lipid-based nanoparticle (LNP) for packaging and delivery of large chemically modified CFTR mRNA (cmCFTR) to patient-derived bronchial epithelial cells, resulting in an increase in membrane-localized CFTR and rescue of its primary function as a chloride channel...
June 14, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
Kevin P Lonabaugh, Katherine S O'Neal, Heather McIntosh, Michelle Condren
OBJECTIVE: The purpose of this study was to improve patient education in clinic by evaluating patient perceptions of education provided, as well as patient self-confidence related to topics within cystic fibrosis (CF). The study assessed whether self-confidence matched knowledge and defined patient-specific goals for education timing and information sources. METHODS: Age specific questionnaires were distributed over four months to patients 11 years of age and older and caregivers of patients of all ages at a single CF Foundation accredited care center...
June 9, 2018: Patient Education and Counseling
Sanni Kumar, Ena Gupta, Sanket Kaushik, Anupam Jyoti
Neutrophils are the forerunner in innate immunity by defending the host organisms against infectious pathogens. During such process, neutrophils reach the site of inflammation/infection and eliminate the pathogens by phagocytosis as well as by forming the neutrophil extracellular traps (NETs). NETs trap and eradicate a number of microbes including bacteria, fungi, protozoa, viruses. NETs consist of DNA which is decorated with histones and granular proteins such as neutrophil elastase (NE), gelatinase, myeloperoxidase...
June 2018: Iranian Journal of Allergy, Asthma, and Immunology
Lisa T Hong, Theodore G Liou, Rishi Deka, Jordan B King, Vanessa Stevens, David C Young
BACKGROUND: Several clinical trials have demonstrated the efficacy of continuous infusion (CI) beta-lactam (BL) antibiotics in cystic fibrosis (CF) patients; however, little is known regarding pharmacokinetic changes during treatment of an acute pulmonary exacerbation (APE). Identifying and understanding these changes may assist in optimizing antibiotic dosing during APE treatment. METHODS: This study is a retrospective cohort study of 162 adult CF patients admitted to the University of Utah Hospital between January 1, 2008 and May 15, 2014 for treatment of an APE with both a CI BL and intravenous tobramycin...
June 13, 2018: Chest
Zihang Lu, Rachel E Foong, Krzysztof Kowalik, Theo J Moraes, Ayanna Boyce, Aimee Dubeau, Susan Balkovec, Per Magnus Gustafsson, Allan B Becker, Piush J Mandhane, Stuart E Turvey, Wendy Lou, Felix Ratjen, Malcolm Sears, Padmaja Subbarao
BACKGROUND: The care of infants with recurrent wheezing relies largely on clinical assessment. The lung clearance index (LCI), a measure of ventilation inhomogeneity, is a sensitive marker of early airway disease in children with cystic fibrosis, but its utility has not been explored in infants with recurrent wheezing. OBJECTIVE: To assess ventilation inhomogeneity using LCI among infants with a history of recurrent wheezing compared with healthy controls. METHODS: This is a case-control study, including 37 infants with recurrent wheezing recruited from outpatient clinics, and 113 healthy infants from a longitudinal birth cohort, the Canadian Healthy Infant Longitudinal Development study...
June 15, 2018: Thorax
Heather Kornmehl, Farzam Gorouhi, Thomas Konia, Maxwell A Fung, Danielle M Tartar
Fixed drug eruption (FDE) is an adverse drug reaction characterized by the development of well-circumscribed, round, dusky erythematous macules and plaques on cutaneous or mucosal surfaces. The reaction occurs on the same mucosal or cutaneous site with subsequent exposures to the offending drug. Although FDE usually manifests as a single lesion, in rare instances, more than one lesion may arise and this is referred to as a generalized eruption. Herein, we present a 31year-old man with history of cystic fibrosis who developed a generalized fixed drug eruption to piperacillin/tazobactam (Zosyn, Pfizer)...
April 15, 2018: Dermatology Online Journal
R Bryan Sutton
No abstract text is available yet for this article.
2018: The Southwest respiratory and critical care chronicles
Chi Wang, Andrei A Aleksandrov, Zhengrong Yang, Farhad Forouhar, Elizabeth A Proctor, Pradeep Kota, Jianli An, Anna Kaplan, Netaly Khazanov, Grégory Boël, Brent R Stockwell, Hanoch Senderowitz, Nikolay V Dokholyan, John R Riordan, Christie G Brouillette, John F Hunt
Many disease-causing mutations impair protein stability. Here, we explore a thermodynamic strategy to correct the disease-causing F508del mutation in the human cystic fibrosis transmembrane conductance regulator (hCFTR). F508del destabilizes nucleotide-binding domain 1 (hNBD1) in hCFTR relative to an aggregation-prone intermediate. We developed a fluorescence self-quenching assay for compounds that prevent aggregation of hNBD1 by stabilizing its native conformation. Unexpectedly, we found that dTTP and nucleotide analogs with exocyclic methyl groups bind to hNBD1 more strongly than ATP and preserve electrophysiological function of full-length F508del-hCFTR channels at temperatures up to 37 °C...
June 14, 2018: Journal of Biological Chemistry
Apparao B Kummarapurugu, Daniel K Afosah, Nehru Viji Sankaranarayanan, Rahaman Navaz Gangji, Shuo Zheng, Thomas P Kennedy, Bruce K Rubin, Judith A Voynow, Umesh R Desai
Cystic fibrosis (CF)1 is a multi-factorial disease in which dysfunction of protease-antiprotease balance plays a key role. The current CF therapy relies on dornase alfa, low dose macrolide antibiotics, hypertonic saline and antibiotics, and does not address the high neutrophil elastase (NE) activity observed in the lung and sputum of CF patients. Our hypothesis is that variants of heparin, which potently inhibit NE but are not anticoagulant, would help restore the protease-antiprotease balance in CF. To realize this concept, we studied molecular principles governing the effectiveness of different heparins, especially 2-O,3-O-desulfated heparin (ODSH), in the presence of sputum components and therapeutic agents...
June 14, 2018: Journal of Biological Chemistry
Andrew Thomas, Yashaswini Ramananda, Kyushik Mun, Anjaparavanda P Naren, Kavisha Arora
The World Health Organization (WHO) has reported a world-wide surge in cases of cholera caused by the intestinal pathogen Vibrio cholerae, and, combined, such surges have claimed several million lives, mostly in early childhood. Elevated cAMP production in intestinal epithelial cells challenged with cholera toxin (CTX) results in diarrhea due to chloride transport by a cAMP-activated channel, the cystic fibrosis transmembrane conductance regulator (CFTR). However, the identity of the main cAMP-producing proteins that regulate CFTR in the intestine and may be relevant for secretory diarrhea is unclear...
June 14, 2018: Journal of Biological Chemistry
Stephanie Chin, Maurita Hung, Amy Won, Yu-Sheng Wu, Saumel Ahmadi, Donghe Yang, Salma Elmallah, Krimo Toutah, C Michael Hamilton, Robert N Young, Russell D Viirre, Christopher M Yip, Christine E Bear
The major Cystic Fibrosis (CF) causing mutation, the deletion of phenylalanine at position 508 (F508del) at the cystic fibrosis transmembrane conductance regulator (CFTR), occurs in approximately 90% of the CF population. Recently, a combination therapy, comprising a corrector (VX-809) that rescues the processing defects of F508del-CFTR and a potentiator (VX-770) that rescues mutant channel activity, was approved for CF patients homozygous for this mutation. However, clinical studies revealed that the efficacy of this drug on lung function was modest and variable amongst patients...
June 14, 2018: Molecular Pharmacology
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
Terence R Flotte
No abstract text is available yet for this article.
June 2018: Human Gene Therapy
Sarah Tavernier, Andrea Sass, Michiel De Bruyne, Femke Baeke, Riet De Rycke, Aurélie Crabbé, Ilse Vandecandelaere, Filip Van Nieuwerburgh, Tom Coenye
Background: Streptococcus anginosus, Pseudomonas aeruginosa and Staphylococcus aureus are often co-isolated from the sputum of cystic fibrosis patients. It was recently shown that S. anginosus is protected from the activity of vancomycin when it grows in a multispecies biofilm with P. aeruginosa and S. aureus. Objectives: Elucidating the underlying cause of the reduced susceptibility of S. anginosus to vancomycin when growing in a multispecies biofilm with P. aeruginosa and S...
June 12, 2018: Journal of Antimicrobial Chemotherapy
Zhe Hui Hoo, Rachael Curley, Michael J Campbell, Stephen J Walters, Martin J Wildman
RATIONALE, AIMS AND OBJECTIVE: Cross-country comparisons of cystic fibrosis (CF) outcomes can potentially identify variation in care but are dependent on data quality. An important assumption is that the UK annual review FEV1 is only collected during periods of clinical stability. If this assumption does not hold, results of FEV1 comparisons may be biased in favour of registries with encounter-based FEV1 . We aimed to test the assumption that CF annual reviews in the UK are only performed during periods of clinical stability...
June 14, 2018: Journal of Evaluation in Clinical Practice
Emma Tippett, Samantha Ellis, John Wilson, Tom Kotsimbos, Denis Spelman
Background: Mycobacterium abscessus complex (MAbsC) is a significant management dilemma when taking care of patients with cystic fibrosis (CF). Methods: We undertook a retrospective cohort analysis of all CF patients in whom MAbsC was isolated from 2005 to 2014. The natural history of MAbsC was determined and clinical factors examined in an attempt to predict transient compared to persistent colonization. Results: No correlation was found between recurrent MAbsC isolation and clinical factors such as body mass index, respiratory function, or age...
April 2018: International Journal of Mycobacteriology
Thomas Radtke, Lukas Böni, Peter Bohnacker, Marion Maggi-Beba, Peter Fischer, Susi Kriemler, Christian Benden, Holger Dressel
BACKGROUND: Regular airway clearance by chest physiotherapy and/or exercise is critical to lung health in cystic fibrosis (CF). Combination of cycling exercise and chest physiotherapy using the Flutter® device on sputum properties has not yet been investigated. METHODS: This prospective, randomized crossover study compared a single bout of continuous cycling exercise at moderate intensity (experiment A, control condition) vs a combination of interval cycling exercise plus Flutter® (experiment B)...
June 14, 2018: BMC Pulmonary Medicine
Susannah Ahern, Geoff Sims, Arul Earnest, Scott C Bell
The Australian Cystic Fibrosis Data Registry is positioning itself as an exemplar of a rare disease registry for the future. While it continues to inform cystic fibrosis (CF) clinicians of patterns of CF disease and quality of care, its capability is increasing as a resource for further research into CF subpopulations, as a platform for clinical trials, and as an interface for patient experiences.
June 2018: Internal Medicine Journal
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