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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Publication date: Available online 30 January 2020Source: Joint Bone SpineAuthor(s): Ana Catarina Duarte, Joanna Porter, Maria José LeandroABSTRACTBronchiectasis is defined as irreversibly damaged and dilated bronchi and is one of the most common pulmonary manifestations in patients with rheumatoid arthritis (RA).The model of RA-associated autoimmunity induced in some individuals by chronic bacterial infection in bronchiectasis is becoming increasingly acceptable, although a genetic predisposition to RA-associated bronchiectasis has also been demonstrated.Bronchiectasis should be suspected in RA patients with chroni...
Source: Joint Bone Spine - Category: Orthopaedics Source Type: research
Pulmonary disease remains the most significant determinant of morbidity and mortality in cystic fibrosis (CF). Patients with CF typically experience progressive decline in lung function punctuated by pulmonary exacerbations [1], acute episodes characterized by worsened cough and sputum production and thickness, as well as decreases in pulmonary function, appetite, and weight. Over 34% of patients with CF are treated with IV antibiotics for an exacerbation each year [2]; median duration of hospitalization for these events is 9.3 days, and the median duration of IV antibiotics is 13 days [2].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Original Article Source Type: research
A 51-year-old Caucasian male with cystic fibrosis (CF) (homozygous ΔF508) and mild obstructive lung disease (baseline forced expiratory volume in 1 second (FEV1) was 2.59L, 70% predicted), presented to our clinic with a history of recurrent fevers, cough with purulent sputum, and dyspnea on exertion. Comorbidities included chronic infection with Pseudomonas aerugi nosa, intermittent colonization with Exophiala dermatitidis, pancreatic insufficiency, adenomatous colon polyps (resected), osteopenia and CF-related diabetes.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research
Background: Chronic respiratory diseases contribute to the development of anxiety and depression. In bronchiectasis (BE), it is unclear whether these comorbidities are related to general or disease-related quality of life (HRQoL) or symptoms.Aims: To evaluate the correlation between anxiety and depression and QoL in patients with BE.Methods: 26 subjects with non-cystic fibrosis BE of both sexes, 56±16 years, classified by the Bronchiectasis Severity Index (BSI), answered to the Mini Mental State Examination (MMSE), Anxiety and Depression Scale (HADS), Medical Outcomes Study 36 – Item Short – Form Health ...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Physiotherapists Source Type: research
Chronic rhinosinusitis (CRS) and nasal polyposis are common comorbidities among Cystic Fibrosis (CF) patients. These conditions are managed with education to routine nasal irrigations, periodical follow-ups that consists of quality of life questionnaires, annual nasal fibroscopy and nasal lavage fluid analysis.Aim: To retrospectively analyze CF patients with symptoms of chronic rhinosinusitis and nasal polyposis.An observational retrospective analysis was performed from 2017 to 2018 among clinically stable adult CF patients. Respiratory function indices, anthropometric data, routine airway clearance techniques and Sino-nas...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Physiotherapists Source Type: research
Aim: To investigate the role of different types of bronchial infection and respiratory symptoms upon lung function decline.Methods: 89 children and adults with CF (mean (SD) age: 11.71 (6) years) performed 1340 serial Multiple Breath Washout (MBW) tests and 980 spirometries over a 5-year period. Respiratory symptoms (cough, sputum, nasal congestion) and Pulmonary Exacerbations (PEx) were recorded. Pseudomonas Aeruginosa, Staphylococcus aureus infection, as well as aspergillus, candida, Achromobacter, Acinetobacter, Serratia and Stenotrophomonas Maltophilia isolated from cough swabs/ sputum cultures were recorded.Results: M...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Cystic fibrosis Source Type: research
Conclusions: this is the largest study in pediatric NCFB. As the management of NCFB affected patients remains problematic, a better definition of clinical, radiological and microbiological features of NCFB is required to improve their management.Reference:1. BMC Pediatrics 2014;14: 299
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Paediatric bronchology Source Type: research
Introduction: Patients with neuromuscular diseases (NMD) frequently have respiratory complications including secretion retention related to weak cough, aspiration syndromes, and chest wall deformity. Nebulised hypertonic saline (HTS) is useful in cystic fibrosis to remove secretions and decrease exacerbations. We know of no data on use in NMD.Hypothesis: Nebulised HTS in NMD decreases respiratory complications and is acceptable to patients.Aims: To assess whether treatment with nebulised HTS in NMD 1) decreases respiratory exacerbations, 2) improves ease of airway clearance, and 3) explore the participants’ perceptio...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Rehabilitation and chronic care Source Type: research
In conclusion, the incidence of M abscessus does not seem to be increasing in non-CF patients in our institution. There may be an epidemiological link with acid suppression and reflux disease, this hypothesis has been previously reported in patients with CF.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Respiratory infections Source Type: research
AbstractPurposeAustralian data regarding the management of patients with bronchiectasis is scarce. We sought to compare the management of adults with bronchiectasis attending tertiary Australian centres with recent national and international guidelines.MethodsThe Australian Bronchiectasis Registry is a centralised database of patients with radiologically confirmed bronchiectasis unrelated to cystic fibrosis recruited from 14 tertiary Australian hospitals. We excluded children (
Source: Lung - Category: Respiratory Medicine Source Type: research
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