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Gene therapy breakthrough for cystic fibrosis

ConclusionThis RCT showed that a new non-viral-based gene therapy for cystic fibrosis was able to produce “modest” benefits in lung function compared to a placebo. The treatments were given once a month for a year. The study had many strengths, including its double-blind randomised design, recruiting adequate numbers to demonstrate real differences between groups, and using pre-specified outcomes and sub-analysis. This means we can be confident in the reliability of the findings presented. Although the findings of this study are encouraging, there are always limitations. These include: This study was relatively small, recruiting just 140 patients. This is normal for a phase II trial, but large clinical trials are needed to fully assess the effects and safety of this treatment in development. Patients recruited in this trial had to be clinically stable to be included. This means they might be at their optimum respiratory health at this stage. Therefore, we don’t know how the treatment would work in clinically unstable or very severe patient groups.It is important to realise that both groups’ lung function got worse over the year, so the treatment as it stands is quite limited. The new gene therapy was able to lessen some of the deterioration, but not in all. Nonetheless, this gives the researchers hope and scope to work out how to improve it. Optimising the dose, working out why it worked in some people and not others, and trialling the therapy in mor...
Source: NHS News Feed - Category: Consumer Health News Tags: Genetics/stem cells Heart/lungs Source Type: news

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CONCLUSIONS: Diagnosis of non-CF bronchiectasis is often delayed because of a failure to recognize the significance of symptoms. Through clinical investigation, including a HRCT scan of the chest, sweat test, studies of immune function, and ciliary function in a child with a prolonged suppurative cough, remains important. In Turkey, the most common causes of non-CF bronchiectasis are PCD and immunodeficiency, related to a high frequency of consanguinity. PMID: 29605210 [PubMed - in process]
Source: Respiratory Care - Category: Respiratory Medicine Authors: Tags: Respir Med Source Type: research
CONCLUSIONS: There is currently no evidence indicating whether orally administered antibiotics are more beneficial compared to inhaled antibiotics. The recent ERS bronchiectasis guidelines provide a practical approach to the use of long-term antibiotics. New research is needed comparing inhaled versus oral antibiotic therapies for bronchiectasis patients with a history of frequent exacerbations, to establish which approach is the most effective in terms of exacerbation prevention, quality of life, treatment burden, and antibiotic resistance. PMID: 29587336 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Abstract BACKGROUND: Cystic fibrosis (CF) airway secretions are abnormal, contributing to decreased clearance and a cycle of infection and inflammation. CF sputum properties may predict disease progression. We hypothesized that sputum viscoelasticity and clearance abnormalities would inversely correlate with pulmonary function during exacerbation and that sputum properties would return to baseline after therapy. METHODS: We collected sputa longitudinally from 13 CF subjects with moderate to severe lung disease during both clinical stability and exacerbation. Dynamic rheology was analyzed using cone and plate ...
Source: Chest - Category: Respiratory Medicine Authors: Tags: Chest Source Type: research
CONCLUSIONS: Long-term macrolide therapy may reduce the frequency of exacerbations and improve quality of life, although supporting evidence is derived mainly from studies of azithromycin, rather than other macrolides, and predominantly among adults rather than children. However, macrolides should be used with caution, as limited data indicate an associated increase in microbial resistance. Macrolides are associated with increased risk of cardiovascular death and other serious adverse events in other populations, and available data cannot exclude a similar risk among patients with bronchiectasis. PMID: 29543980 [PubMe...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
We present two cases where this modified version of MI‐E was used as an adjunct to traditional ACT in adults during an acute exacerbation of CF.
Source: Respirology Case Reports - Category: Respiratory Medicine Authors: Tags: Case Report Source Type: research
AbstractIntroductionExercise capacity is impaired in patients with bronchiectasis. Incremental shuttle walk test (ISWT) stresses cardiorespiratory system physiologically to symptom-limited maximal exercise capacity. The purpose of this study was to investigate the clinical determinants of ISWT in adults with non-cystic fibrosis (CF) bronchiectasis.MethodsForty-one clinically stable bronchiectasis patients aged 18 –72 years (27 females, 14 males) participated in the study. Subjects’ demographics and physical characteristics were recorded. Bronchiectasis Severity Index was used to identify disease severity. ...
Source: Lung - Category: Respiratory Medicine Source Type: research
CONCLUSIONS: There is moderate-quality evidence to show that treatment with mannitol over a six-month period is associated with an improvement in some measures of lung function in people with cystic fibrosis compared to control. There is low to very low-quality evidence suggesting no difference in quality of life for participants taking mannitol compared to control. This review provides very low-quality evidence suggesting no difference in lung function or quality of life comparing mannitol to dornase alfa alone and to mannitol plus dornase alfa.The clinical implications from this review suggest that mannitol could be cons...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
CONCLUSIONS: While the cough panel was not able to make recommendations, they have made consensus based suggestions and provided direction for future studies to fill the gaps in knowledge. PMID: 29355548 [PubMed - as supplied by publisher]
Source: Chest - Category: Respiratory Medicine Authors: Tags: Chest Source Type: research
AbstractBronchiectasis in pediatric age is a heterogeneous disease associated with significant morbidity.The most common medical conditions leading to bronchial damage are previous pneumonia and recurrent lower airway infections followed by underlying diseases such as immune-deficiencies, congenital airway defects, recurrent aspirations and mucociliary clearance disorders.The most frequent symptom is chronic wet cough. The introduction of high-resolution computed tomography (HRCT) has improved the time of diagnosis allowing earlier treatment.However, the term “bronchiectasis” in pediatric age should be used wit...
Source: Italian Journal of Pediatrics - Category: Pediatrics Source Type: research
Introduction: Bronchiectasis (BE) is a chronic disease associated with high morbidity e mortality. Several studies have enlightened pathophysiology, microbiology and epidemiology of the disease, but few is known about its phenotypes. We aimed study clinical BE phenotypes in relation to comorbidities (i.e. COPD and asthma.)Methods: We consecutively enrolled all adult patients with a CT diagnosis of non-cystic fibrosis BE presenting to our Outpatients Clinic in a 6-months period. Patients with traction BE were excluded. All included patients underwent to clinical and laboratory workup, lung function tests and microbiological...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Clinical Problems Asthma Source Type: research
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