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1,320 results for “Cystic fibrosis”
CARE-CF-1 Clinical trial data: An exploratory, randomized, double-blind, placebo-controlled 6-arm clinical trial examining cysteamine as an adjunct therapy for the treatment of pulmonary exacerbations of cystic fibrosis
<p><em>Background:</em> Emerging data suggests a possible role for cysteamine as an adjunct treatment for pulmonary exacerbations of cystic fibrosis (CF) that continue to be a major clinical challenge. There are no studies investigating the use of cysteamine in pulmonary exacerbations of CF. This exploratory randomized clinical trial was conducted to answer the question: In future pivotal trials of cysteamine as an adjunct treatment in pulmonary exacerbations of CF, which candidate cysteamine dosing regimens should be tested and which are the most appropriate, clinically meaningful outcome measures to employ as endpoints?</p> <p><em>Methods and findings: </em>Multicentre double-blind randomized clinical trial. Adults experiencing a pulmonary exacerbation of CF being treated with standard care that included aminoglycoside therapy were randomized equally to a concomitant 14-day course of placebo, or one of 5 dosing regimens of cysteamine. Outcomes were recorded on days 0, 7, 14 and 21 and included sputum bacterial load and the patient reported outcome measures (PROMs): Chronic Respiratory Infection Symptom Score (CRISS), the Cystic Fibrosis Questionnaire–Revised (CFQ-R); FEV1, blood leukocyte count, and inflammatory markers. Eighty nine participants in fifteen US and EU centres were randomized, 78 completed the 14-day treatment period. Cysteamine had no significant effect on sputum bacterial load, however technical difficulties limited interpretation. The most consistent findings were for cysteamine 450mg twice daily that had effects additional to that observed with placebo, with improved symptoms, CRISS additional 9.85 points (95% CI 0.02, 19.7) p=0.05, reduced blood leukocyte count by 2.46x109 /l (95% CI 0.11, 4.80), p=0.041 and reduced CRP by geometric mean 2.57 nmol/l (95% CI 0.15, 0.99), p=0.049.</p> <p><em>Conclusion:</em> In this exploratory study cysteamine appeared to be safe and well-tolerated. Future pivotal trials investigating the utility of cysteamine in pulmonary exacerbations of CF need to include the cysteamine 450mg doses and CRISS and blood leukocyte count as outcome measures.</p>
Data from: High virulence sub-populations in Pseudomonas aeruginosa long-term cystic fibrosis airway infections
Background: Pseudomonas aeruginosa typically displays loss of virulence-associated secretions over the course of chronic cystic fibrosis infections. This has led to the suggestion that virulence is a costly attribute in chronic infections. However, previous reports suggest that overproducing (OP) virulent pathotypes can coexist with non-producing mutants in the CF lung for many years. The consequences of such within-patient phenotypic diversity for the success of this pathogen are not fully understood. Here, we provide in-depth quantification of within-host variation in the production of three virulence associated secretions in the Liverpool cystic fibrosis epidemic strain of P. aeruginosa, and investgate the effect of this phenotypic variation on virulence in acute infections of an insect host model. Results: Within-patient variation was present for all three secretions (pyoverdine, pyocyanin and LasA protease). In two out of three patients sampled, OP isolates coexisted with under-producing mutants. In the third patient, all 39 isolates were under-producers of all three secretions relative to the transmissible ancestor LESB58. Finally, this phenotypic variation translated into variation in virulence in an insect host model. Conclusions: Within population variation in the production of P. aeruginosa virulence-associated secretions can lead to high virulence sub-populations persisting in patients with chronic CF infections.
Data from: Rapid diversification of Pseudomonas aeruginosa in cystic fibrosis lung-like conditions
Chronic infection of the cystic fibrosis (CF) airway by the opportunistic pathogen Pseudomonas aeruginosa is the leading cause of morbidity and mortality for adult CF patients. Prolonged infections are accompanied by adaptation of P. aeruginosa to the unique conditions of the CF lung environment, as well as marked diversification of the pathogen into phenotypically and genetically distinct strains that can coexist for years within a patient. Little is known, however, about the causes of this diversification and its impact on patient health. Here, we show experimentally that, consistent with ecological theory of diversification, the nutritional conditions of the CF airway can cause rapid and extensive diversification of P. aeruginosa. Mucin, the substance responsible for the increased viscosity associated with the thick mucus layer in the CF airway, had little impact on within-population diversification but did promote divergence among populations. Furthermore, in vitro evolution recapitulated traits thought to be hallmarks of chronic infection, including reduced motility and increased biofilm formation, and the range of phenotypes observed in a collection of clinical isolates. Our results suggest that nutritional complexity and reduced dispersal can drive evolutionary diversification of P. aeruginosa independent of other features of the CF lung such as an active immune system or the presence of competing microbial species. We suggest that diversification, by generating extensive phenotypic and genetic variation on which selection can act, may be a key first step in the development of chronic infections.
Data from: Stratified assessment of the role of inhaled hypertonic saline in reducing cystic fibrosis pulmonary exacerbations: a retrospective analysis
Objective: Limited data exist concerning the role of inhaled hypertonic saline (HS) in decreasing pulmonary exacerbations in cystic fibrosis (CF), especially as more advanced stages of CF lung disease were excluded in prior studies. Herein we retrospectively determined the efficacy of inhaled HS in reducing CF pulmonary exacerbations when stratified according to the severity of CF lung disease. Stratification was based on the framework of the Pulmonary Therapeutics Committee's published gradation of obstructive lung physiology in CF, i.e., mild (FEV1 > 70%), moderate (FEV1 40-70%) and severe (FEV1 < 40%) lung disease, respectively. Design: A retrospective review of the Port CF® database over a 3-year period performed at an academic CF care center. Results: 340 pulmonary exacerbations were identified; inhaled HS was being used in 99 of these cases. Univariate analysis demonstrated a significant reduction in pulmonary exacerbations only in mild obstruction (OR=0.09, CI 0.01-0.81, p=0.012); however, multivariate logistic regression that adjusted for confounding variables showed a reduction in pulmonary exacerbations across the entire spectrum of obstructive lung disease when using inhaled HS i.e., mild obstructive CF lung disease (OR=0.17, CI 0.05-0.58, p=0.004), moderate obstructive CF lung disease (OR=0.39, CI 0.16-0.93, p=0.034), as well as severe obstructive CF lung disease (OR=0.02, CI 0.001-0.45, p=0.015). Moreover, inhaled HS appeared reasonably well tolerated across all stages of lung disease severity, and was discontinued in only 7% of cases (n=4) with severe lung disease. Conclusion: In this study, inhaled HS appeared to reduce pulmonary exacerbations in CF lung disease at all stages of obstruction. This underscores the importance of therapeutic inhaled HS in CF lung disease, regardless of severity of lung obstruction.
Cystic Fibrosis Telemedicine in the era of COVID-19 patient and staff surveys
<p>The coronavirus disease 2019 (COVID-19) pandemic has resulted in large scale changes to incorporate telemedicine for delivery of care. People with cystic fibrosis (CF) have care considerations that pose challenges to telemedicine; they include frequent visits for pulmonary disease progression, medication management and evaluation by a multidisciplinary team of providers. We share our center's experience with video visits replacing in-person clinic evaluation, using quality improvement strategies to create a replicable workflow. Key considerations include incorporation of the multidisciplinary team into the visit, limitations of remote delivery of care, as well as patient and staff perceptions of this care model. Results revealed that video visits were convenient, efficacious, and comparable to in-person visits with interest for its continued incorporation into the traditional CF care model.</p>
Carriers of a single cystic fibrosis transmembrane conductance regulator pathogenic variant and COVID-19 in pregnancy: A retrospective longitudinal cohort study
Open the record for dataset details and reuse information.
Dataset of laboratory research of paper "Mutation Analysis and Characteristics of the 5T Allele in the Cystic Fibrosis Trans Membrane Gene"
<p>Dataset contains</p> <p>1. Blood Lymphocite</p> <p>2. Utensils and raw Materials</p>
Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
ClinicalTrials.gov study NCT00743483. IPD Sharing: Not stated. Countries: 2. Publications: 0.
Airway Clearance for Pediatric and Adult Cystic Fibrosis Patients Using a Portable Intra-Pulmonary Percussion Device
ClinicalTrials.gov study NCT04743206. IPD Sharing: NO. Countries: 1. Publications: 0.
Pilot and Feasibility Study for the Treatment of Pre-diabetes in Patients With Cystic Fibrosis
ClinicalTrials.gov study NCT00763412. IPD Sharing: UNDECIDED. Countries: 1. Publications: 0.
Effect of Andecaliximab on FEV1 in Adults With Cystic Fibrosis
ClinicalTrials.gov study NCT02759562. IPD Sharing: Not stated. Countries: 5. Publications: 0.
Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
ClinicalTrials.gov study NCT03451045. IPD Sharing: UNDECIDED. Countries: 21. Publications: 0.
A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
ClinicalTrials.gov study NCT02730208. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Registry Study on Cystic Fibrosis in Chinese Children
ClinicalTrials.gov study NCT02753374. IPD Sharing: Not stated. Countries: 0. Publications: 6.
Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis
ClinicalTrials.gov study NCT02566044. IPD Sharing: Not stated. Countries: 2. Publications: 0.
Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
ClinicalTrials.gov study NCT05274269. IPD Sharing: NO. Countries: 15. Publications: 0.
Pilot Trial of Phototherapy for Acute Depression in Hospitalized Cystic Fibrosis Patients
ClinicalTrials.gov study NCT01822197. IPD Sharing: NO. Countries: 1. Publications: 0.
Prevention of Cystic Fibrosis Diabetes
ClinicalTrials.gov study NCT00967798. IPD Sharing: UNDECIDED. Countries: 1. Publications: 0.
Rifaximin for Treatment of Bloating in Children and Adults With Cystic Fibrosis
ClinicalTrials.gov study NCT05408910. IPD Sharing: Not stated. Countries: 0. Publications: 1.
Oral Health Status of Cystic Fibrosis Patients. An Online Survey in Collaboration With the Vaincre la Mucoviscidose Patient Association.
ClinicalTrials.gov study NCT06356246. IPD Sharing: YES. Countries: 1. Publications: 0.
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