Preface: Cystic Fibrosis in Children and Adults
This supplement arises out of the 29th one-day meeting on cystic fibrosis (CF) organised by the Section of Paediatrics of the Royal Society of Medicine. This meeting was organised by Dr Iolo Doull and held at the Royal Society of Medicine in London on 17 November 2015. We are grateful to Actavis UK for their support. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 17, 2016 Category: Respiratory Medicine Authors: T.J. David Source Type: research
Preface: Cystic Fibrosis in Children and Adults
(Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 17, 2016 Category: Respiratory Medicine Authors: T J David Source Type: research
Cell Therapy for Bronchopulmonary Dysplasia: Promises and Perils
Despite great achievements in neonatal and perinatal medicine over the past decades, the immature lung remains the most critical organ to care for after premature birth. As a consequence, bronchopulmonary dysplasia (BPD) remains the most common complication of extreme prematurity. BPD impairs normal development and may cause lifelong morbidities. At present, there is no effective treatment for BPD – including preventing premature birth. Recent insights into the biology of stem and progenitor cells have ignited the hope of protecting the immature lung, and even regenerating an already damaged lung by using exogenous stem-...
Source: Paediatric Respiratory Reviews - June 16, 2016 Category: Respiratory Medicine Authors: Marius Alexander M öbius, Bernard Thébaud Tags: Today's Science - Tomorrow's Medicine Source Type: research
Concerns with beta2-agonists in pediatric asthma - a clinical perspective
Beta2-adrenoreceptor agonists ( β2-agonists) are extensively used in the treatment of childhood asthma. However, there have been concerns regarding their adverse effects and safety. In 2005, the FDA commissioned a “Black Box Warning” communicating the potential for an increased risk for serious asthma exacerbations or asthma- related death with the regular use of LABAs. In a meta-analysis of controlled clinical trials, the incidence of severe adverse events appeared to be highest in the 4-11 year age group. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 14, 2016 Category: Respiratory Medicine Authors: Elin T.G. Kersten, Gerard H. Koppelman, Bernard J. Thio Source Type: research
Concerns with beta2-agonists in pediatric asthma - a clinical perspective
Beta2-adrenoreceptor agonists (β2-agonists) are extensively used in the treatment of childhood asthma. However, there have been concerns regarding their adverse effects and safety. In 2005, the FDA commissioned a “Black Box Warning” communicating the potential for an increased risk for serious asthma exacerbations or asthma-related death with the regular use of LABAs. In a meta-analysis of controlled clinical trials, the incidence of severe adverse events appeared to be highest in the 4-11 year age group. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 14, 2016 Category: Respiratory Medicine Authors: Elin T.G. Kersten, Gerard H. Koppelman, Bernard J. Thio Source Type: research
Treatment of pulmonary exacerbations in cystic fibrosis – could do better?
This article describes the nature and significance of pulmonary exacerbations in cystic fibrosis (CF). The effectiveness and safety of current exacerbation treatment are explored. The article concludes with a summary of clinical trials (completed and ongoing) which aim to improve the efficacy and safety of exacerbation treatment. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Alan Smyth Tags: Review Source Type: research
Lifestyle treatments in cystic fibrosis: The NHS should not pay
Lifestyle treatments can be defined as those which may have in impact on quality of life but do not affect health outcomes. Particular treatment options may be preferred by patients because they are for example, easier to use, take up less time or taste better. The impact on adherence needs to be considered. Treatment options that promote greater adherence to therapy are likely to be more efficacious and so are not, by definition, lifestyle treatments. The NHS is facing unprecedented financial pressure and resources are limited. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Jeremy Hull Tags: Review Source Type: research
Treatment of pulmonary exacerbations in cystic fibrosis –could do better?
This article describes the nature and significance of pulmonary exacerbations in cystic fibrosis (CF). The effectiveness and safety of current exacerbation treatment are explored. The article concludes with a summary of clinical trials (completed and ongoing) which aim to improve the efficacy and safety of exacerbation treatment. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Alan Smyth Source Type: research
Electronic patient records, past, present and future
The health informatics revolution was spear-headed in the 1980s by pioneers in primary care who worked in an opportune environment and were able to successfully implement electronic patient records (EPR) as far back as the 1990s. Although the ambitious and costly National Programme for IT failed to deliver an integrated EPR, the project achieved the creation of the Spine, the N3 Network, choose and book, picture archiving, communication systems and standards which have allowed integration. Real change is taking place within the NHS with the launch of exciting new projects focusing on true integration and secure data flows ...
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Daniel Peckham Source Type: research
Lifestyle treatments in cystic fibrosis: The NHS should pay
With the NHS under increasing financial pressure and healthcare costs soaring year on year, it is perhaps not surprising that assessment agencies focus on cost-effectiveness analysis when assessing new therapies. Such an approach does not however, always take sufficient account of treatment burden, lifestyle and patient choice and therefore new equally effective but perhaps “easier to take” formulations and faster delivery systems for current therapies do not always take precedence in current treatment guidelines. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Robert Ian Ketchell Source Type: research
Lifestyle treatments in CF –the NHS should not pay
Lifestyle treatments can be defined as those which may have in impact on quality of life but do not affect health outcomes. Particular treatment options may be preferred by patients because they are for example, easier to use, take up less time or taste better. The impact on adherence needs to be considered. Treatment options that promote greater adherence to therapy are likely to be more efficacious and so are not, by definition, lifestyle treatments. The NHS is facing unprecedented financial pressure and resources are limited. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Jeremy Hull Source Type: research
Cystic Fibrosis Papers of the Year 2015
Studies published in the last year have expanded our knowledge of potential disease modifying agents in the treatment of class II, III and IV CFTR mutations, and included the first report of an efficacious gene therapy for CF. There is also an important message on increasing use of conventional chronic therapies even in milder disease, and the pernicious effect of chronic infection on pulmonary function. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Iolo Doull Source Type: research
Phenotypic variability of R117H-CFTR expression within monozygotic twins
Whilst cystic fibrosis is a monogenic condition, variation in phenotype exists for the same CFTR genotype, which is influenced by multiple genetic and non-genetic (environmental) factors. The R117H-CFTR mutation has variability directly relating to in cis poly-thymidine alleles, producing a differing spectrum of disease. This paper provides evidence of extreme phenotype variability –including fertility status - in the context of male monogenetic twins, discussing mechanisms and highlighting the diagnostic and treatment challenges. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Michael D. Waller, Nicholas J. Simmonds Source Type: research
Is deafness mutation screening required in cystic fibrosis patients?
Aminoglycosides are widely used in cystic fibrosis management. The m.1555A > G mutation predisposes to aminoglycoside ototoxicity. It may cause later onset hearing loss in the absence of aminoglycosides use and gradual hearing loss may be an inevitable consequence of the mutation. Given that aminoglycoside therapy forms the backbone of IV protocols in CF, this article recommends screening for this mutation to allow informed decision-making prior to aminoglycoside administration, to avoid preventable deafness. (Source: Paediatric Respiratory Reviews)
Source: Paediatric Respiratory Reviews - June 13, 2016 Category: Respiratory Medicine Authors: Rania Abusamra, Donna McShane Source Type: research
