The effect of ivacaftor in adolescents with cystic fibrosis (G551D mutation): An exercise physiology perspective

Pediatric Physical Therapy

Volumn 26, Issue 4, 2014, Pages 454-461

  Saynor, Zoe Louise ^a,b   Barker, Alan Robert ^a   Oades, Patrick John ^b   Williams, Craig Anthony ^a  

^a UNIVERSITY OF EXETER   (United Kingdom)

^b ROYAL DEVON AND EXETER NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

Adolescent; Cardiopulmonary exercise test; Cystic fibrosis; Drug therapy; Exercise physiology

Indexed keywords

AMINOPHENOL DERIVATIVE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; N-(2,4-DI-TERT-BUTYL-5-HYDROXYPHENYL)-4-OXO-1,4-DIHYDROQUINOLINE-3-CARBOXAMIDE; QUINOLONE DERIVATIVE;

ADOLESCENT; AGONISTS; CASE REPORT; CYSTIC FIBROSIS; EXERCISE TEST; FEMALE; HUMAN; LUNG FUNCTION TEST; MALE; MORPHOMETRICS;

ADOLESCENT; AMINOPHENOLS; BODY WEIGHTS AND MEASURES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EXERCISE TEST; FEMALE; HUMANS; MALE; QUINOLONES; RESPIRATORY FUNCTION TESTS;

EID: 84923260475     PISSN: 08985669     EISSN: 1538005X     Source Type: Journal    
DOI: 10.1097/PEP.0000000000000086     Document Type: Article

References (20)

1. Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci USA. 2009;106(44):18825-18830.
2. Accurso FJ, Rowe SM, Clancy J, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010;363(21):1991-2003.
3. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672.
4. McKone E, Li H, Davies JC; on behalf of the VX08-770-105 Study Group. Long-term safety and efficacy of ivacaftor in subjects with cystic fibrosis who have the G551D-CFTR mutation (abstract). J Cyst Fibros. 2012;11(suppl 1):S13.
5. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013;187(11):1219-1225.
6. Hebestreit H, Sauer-Heilborn A, Fischer R, Käding M, Mainz JG. Effects of ivacaftor on severely ill patients with cystic fibrosis carrying a G551D mutation. J Cystic Fibros. 2013;12(6):599-603.
7. Harrison MJ, Murphy DM, Plant BJ. Ivacaftor in a G551D homozygote with cystic fibrosis. New Engl J Med. 2013;369(13):1280-1282.
8. Welsh MJ. Targeting the basic defect in cystic fibrosis. N Engl J Med. 2010;363(21):2056-2057.
9. Nixon PA, Orenstein DM, Kelsey SF, Doershuk CF. The prognostic value of exercise testing in patients with cystic fibrosis. N Engl J Med. 1992;327(25):1785-1788.
10. Pianosi P, Leblanc J, Almudevar A. Peak oxygen uptake and mortality in children with cystic fibrosis. Thorax. 2005;60(1):50-54.
11. De Jong W, Kaptein AA, Van Der Schans CP, et al. Quality of life in patients with cystic fibrosis. Pediatr Pulmonol. 1997;23(2):95-100.
12. Pérez M, Groeneveld IF, Santana-Sosa E, et al. Aerobic fitness is associated with lower risk of hospitalization in children with cystic fibrosis. Pediatr Pulmonol. 2014;49(7):641-649.
13. Bell SC, Morris NR. Exercise testing in patients with cystic fibrosis: why and which? J Cyst Fibrosis. 2010;9(5):299-301.
14. Whipp BJ, Davis JA, Torres F, Wasserman K. A test to determine parameters of aerobic function during exercise. J Appl Physiol. 1981;50(1):217-221.
15. Saynor ZL, Barker AR, Oades PJ, Williams CA. A protocol to determine valid O2max in young cystic fibrosis patients. J Sci Med Sport. 2013;16(6):539-544.
16. Saynor ZL, Barker AR, Oades PJ, Williams CA. Reproducibility of maximal cardiopulmonary exercise testing for young cystic fibrosis patients. J Cystic Fibros. 2013;12(6):644-650.
17. Stanojevic S, Wade A, Cole TJ, et al. Asthma UK Collaborative Group. Spirometry centile charts for young Caucasian children: the asthma UK collaborative initiative. Am J Respir Crit Care Med. 2009;180(6):547-552.
18. Beaver WL, Wasserman K, Whipp BJ. A new method for detecting anaerobic threshold by gas exchange. J Appl Physiol. 1986;60(6):2020-2027.
19. Pianosi PT. Impedance cardiography accurately measures cardiac output during exercise in children with cystic fibrosis. Chest. 1997;111(2):333-337.
20. Hulzebos HJ, Sneider H, Van Der Net J, Helders PJM, Takken T. High-intensity interval training in an adolescent with cystic fibrosis: a physiological perspective. Physiother Theory Pract. 2011;27(3):231-237.