Cystic fibrosis: triple drug regimens that target defective ion channel improve lung function, studies show

BMJ ◽  
2018 ◽  
pp. k4377 ◽  
Author(s):  
Susan Mayor
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Ion Channel ◽  
Improve Lung Function ◽  
Drug Regimens

scholarly journals Inhaled beta-agonists improve lung function but not maximal exercise capacity in cystic fibrosis

2005 ◽  
Vol 4 (2) ◽  
pp. 101-105 ◽  
Author(s):  
Jonathan D. Dodd ◽  
Sinead C. Barry ◽  
Leslie E. Daly ◽  
Charles G. Gallagher
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Exercise Capacity ◽  
Maximal Exercise ◽  
Improve Lung Function ◽  
Beta Agonists ◽  
Maximal Exercise Capacity

Long-term azithromycin may improve lung function in children with cystic fibrosis

The Lancet ◽  
1998 ◽  
Vol 351 (9100) ◽  
pp. 420 ◽  
Author(s):  
Adam Jaffé ◽  
Jackie Francis ◽  
Mark Rosenthal ◽  
Andrew Bush
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Improve Lung Function

scholarly journals FESS Does Not Improve Lung Function in Cystic Fibrosis

2011 ◽  
Vol 145 (2_suppl) ◽  
pp. P122-P122
Author(s):  
Javad A. Sajan ◽  
Robert Kempainen ◽  
Frank L. Rimell
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Improve Lung Function

scholarly journals Effect of Ivacaftor on Objective and Subjective Measures of Cough in Patients with Cystic Fibrosis

2016 ◽  
Vol 10 (1) ◽  
pp. 105-108 ◽  
Author(s):  
Shoaib Faruqi ◽  
Dejene Shiferaw ◽  
Alyn H. Morice
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Drug Efficacy ◽  
Improve Lung Function ◽  
Intestinal Function ◽  
End Points ◽  
Time Frames ◽  
Novel Drug ◽  
Objective Improvement

Background and Objectives: Cough is a major symptom in cystic fibrosis. Ivacaftor is a novel drug which targets the G551D mutation and has been demonstrated to improve lung function and weight in the long term. It also improves symptoms of extra-oesophageal reflux. We wanted to evaluate the effect of ivacaftor on cough in cystic fibrosis. Methods: In two patients with cystic fibrosis the Hull Airway Reflux Questionnaire (HARQ) was completed and objective cough counts were measured prior to and within 4 weeks after initiation of treatment with ivacaftor. Spirometry was also undertaken and weight checked at these time frames. Results: In the first patient the HARQ score decreased from 29 to 11 and objective cough counts from 29 to 9 cough events per hour. Similarly in the second patient the HARQ score decreased from 13 to 9 and objective cough count from 76 to 5 cough events per hour. There was no significant change in spirometric parameters or weight. Conclusion: We have observed early subjective and objective improvement in cough measures on treatment with ivacaftor. We suggest that this improvement could be attributed to improvement of gastro-intestinal function and that cough metrics could be used as early and accurate end points of drug efficacy.

scholarly journals Lumacaftor (VX-809) restores the ability of CF macrophages to phagocytose and kill Pseudomonas aeruginosa

2018 ◽  
Vol 314 (3) ◽  
pp. L432-L438 ◽  
Author(s):  
Roxanna Barnaby ◽  
Katja Koeppen ◽  
Amanda Nymon ◽  
Thomas H. Hampton ◽  
Brent Berwin ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Pseudomonas Aeruginosa ◽  
Lung Function ◽  
Genetic Disease ◽  
Premature Death ◽  
Lung Infection ◽  
Airway Epithelial Cells ◽  
Improve Lung Function ◽  
Airway Epithelial ◽  
Progressive Loss

Cystic fibrosis (CF), the most common lethal genetic disease in Caucasians, is characterized by chronic bacterial lung infection and excessive inflammation, which lead to progressive loss of lung function and premature death. Although ivacaftor (VX-770) alone and ivacaftor in combination with lumacaftor (VX-809) improve lung function in CF patients with the Gly551Asp and del508Phe mutations, respectively, the effects of these drugs on the function of human CF macrophages are unknown. Thus studies were conducted to examine the effects of lumacaftor alone and lumacaftor in combination with ivacaftor (i.e., ORKAMBI) on the ability of human CF ( del508Phe/ del508Phe) monocyte-derived macrophages (MDMs) to phagocytose and kill Pseudomonas aeruginosa. Lumacaftor alone restored the ability of CF MDMs to phagocytose and kill P. aeruginosa to levels observed in MDMs obtained from non-CF (WT-CFTR) donors. This effect contrasts with the partial (~15%) correction of del508Phe Cl− secretion of airway epithelial cells by lumacaftor. Ivacaftor reduced the ability of lumacaftor to stimulate phagocytosis and killing of P. aeruginosa. Lumacaftor had no effect on P. aeruginosa-stimulated cytokine secretion by CF MDMs. Ivacaftor (5 µM) alone and ivacaftor in combination with lumacaftor reduced secretion of several proinflammatory cytokines. The clinical efficacy of ORKAMBI may be related in part to the ability of lumacaftor to stimulate phagocytosis and killing of P. aeruginosa by macrophages.

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