High treatment burden in adults with cystic fibrosis: Challenges to disease self-management

Sawicki, Gregory S.; Sellers, Deborah E.; Robinson, Walter M.

doi:10.1016/j.jcf.2008.09.007

Cited by 407 publications

(357 citation statements)

References 15 publications

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“…Patients with CF require numerous therapies to manage these symptoms (3), including mucolytic and antibiotic agents and chest physiotherapy to treat the airway disease and digestive enzymes to replace the loss of exocrine pancreatic function. These and other interventions have increased life expectancy dramatically, but improvement is needed to reduce the high treatment burden and increase survival (6,7).…”

mentioning

confidence: 99%

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

Goor

Hadida

Grootenhuis

et al. 2011

Proc. Natl. Acad. Sci. U.S.A.

951

946

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Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator ( CFTR ) gene that impair the function of CFTR, an epithelial chloride channel required for proper function of the lung, pancreas, and other organs. Most patients with CF carry the F508del CFTR mutation, which causes defective CFTR protein folding and processing in the endoplasmic reticulum, resulting in minimal amounts of CFTR at the cell surface. One strategy to treat these patients is to correct the processing of F508del-CFTR with small molecules. Here we describe the in vitro pharmacology of VX-809, a CFTR corrector that was advanced into clinical development for the treatment of CF. In cultured human bronchial epithelial cells isolated from patients with CF homozygous for F508del, VX-809 improved F508del-CFTR processing in the endoplasmic reticulum and enhanced chloride secretion to approximately 14% of non-CF human bronchial epithelial cells (EC 50 , 81 ± 19 nM), a level associated with mild CF in patients with less disruptive CFTR mutations. F508del-CFTR corrected by VX-809 exhibited biochemical and functional characteristics similar to normal CFTR, including biochemical susceptibility to proteolysis, residence time in the plasma membrane, and single-channel open probability. VX-809 was more efficacious and selective for CFTR than previously reported CFTR correctors. VX-809 represents a class of CFTR corrector that specifically addresses the underlying processing defect in F508del-CFTR.

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mentioning

confidence: 99%

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

Goor

Hadida

Grootenhuis

et al. 2011

Proc. Natl. Acad. Sci. U.S.A.

951

946

View full text Add to dashboard Cite

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“…High-frequency chest wall oscillation is used by 37% of people with CF in the USA [17], but only 3% in Canada [18] and 1% in the UK. Exercise is the most common airway clearance technique in New Zealand (46% of adults) [19], compared with 16% in the UK.…”

Section: Variation By Countrymentioning

confidence: 99%

“…This is evidenced by the low percentages of people with CF who reported that they did not use any airway clearance techniques: 10% in this UK study, 4% in the New Zealand data, 8% in the Canadian registry data and no other reports within the other data identified in this paper [16][17][18]20].…”

Section: Variation By Countrymentioning

confidence: 99%

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Airway clearance techniques used by people with cystic fibrosis in the UK

et al. 2015

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Please cite this article as: Hoo ZH, Daniels T, Wildman MJ, Teare MD, Bradley JM, Airway clearance techniques used by people with cystic fibrosis in the UK, Physiotherapy (2015), http://dx.doi.org/10.1016/j.physio. 2015.01.008 This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain. Setting and participants All people with CF in the UK aged ≥11 years (n=6372). ResultsOf the 6372 people on the UK CF registry in 2011, 89% used airway clearance techniques.The most commonly used primary techniques were forced expiratory techniques (28%) and oscillating positive expiratory pressure (PEP) (23%). Postural drainage and high-frequency chest wall oscillation were used by 4% and 1% of people with CF, respectively. The male:female ratio of individuals who used exercise as their primary airway clearance technique was 2:1, compared with 1:1 for other techniques. Individuals with more severe lung disease tended to use devices such as non-invasive ventilation or high-frequency chest wall oscillation.Conclusions Forced expiratory techniques and oscillating PEP are the most common airway clearance techniques used by people with CF in the UK, and postural drainage and high-frequency chest wall oscillation are the least common techniques. This is significant in terms of planning airway clearance technique trials, where postural drainage has been used traditionally as the comparator. The use of airway clearance techniques varies between countries, but the reasons for these differences are unknown.

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“…Factors related to disease severity, such as reduced lung function may restrict employment choices for adults with CF, and the treatment burden further compounds this; adults with CF are generally expected to perform physiotherapy regularly and there are the added demands of taking large numbers of therapies, including frequent visits to hospital (Sawicki et al 2009). …”

Section: Longitudinal Employment Status In People With Cfmentioning

confidence: 99%

A Life Course Perspective on Health Trajectories and Transitions

Burton-Jeangros

Cullati

Sacker

et al. 2015

Life Course Research and Social Policies

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Life course research has been developing quickly these last decades for good reasons. Life course approaches focus on essential questions about individuals' trajectories, longitudinal analyses, cross-fertilization across disciplines like lifespan psychology, developmental social psychology, sociology of the life course, social demography, socio-economics, social history. Life course is also at the crossroads of several fields of specialization like family and social relationships, migration, education, professional training and employment, and health. This Series invites academic scholars to present theoretical, methodological, and empirical advances in the analysis of the life course, and to elaborate on possible implications for society and social policies applications.More information about this series at

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High treatment burden in adults with cystic fibrosis: Challenges to disease self-management

Cited by 407 publications

References 15 publications

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

Airway clearance techniques used by people with cystic fibrosis in the UK

A Life Course Perspective on Health Trajectories and Transitions

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