Effects of ivacaftor on severely ill patients with cystic fibrosis carrying a G551D mutation

Journal of Cystic Fibrosis

Volumn 12, Issue 6, 2013, Pages 599-603

  Hebestreit, Helge ^a   Sauer Heilborn, Annette ^b   Fischer, Rainald ^c   Käding, Manfred ^d   Mainz, Jochen G ^e  

^a UNIVERSITY HOSPITAL WÜRZBURG   (Germany)

^b HANNOVER MEDICAL SCHOOL   (Germany)

^c LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^d Poliklinik Chemnitz GmbH   (Germany)

^e JENA UNIVERSITY HOSPITAL   (Germany)

Author keywords

Adults; CFTR; FEV1; Mucus; VX 770

Indexed keywords

IVACAFTOR;

ABDOMINAL PAIN; ADULT; ARTICLE; BILIRUBIN BLOOD LEVEL; CLINICAL ARTICLE; CYSTIC FIBROSIS; DISEASE SEVERITY; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; FEMALE; FOLLOW UP; FORCED EXPIRATORY VOLUME; GERMANY; HEADACHE; HOSPITALIZATION; HUMAN; LUNG FUNCTION; LUNG TRANSPLANTATION; MALE; MUCUS SECRETION; MUTATION; PREGNANCY; RESTLESS LEGS SYNDROME; RETROSPECTIVE STUDY; SIDE EFFECT; TREATMENT DURATION;

ADULTS; CFTR; FEV(1); MUCUS; VX-770;

ADULT; AMINOPHENOLS; ANTI-BACTERIAL AGENTS; BRONCHI; CYSTIC FIBROSIS; FEMALE; FORCED EXPIRATORY VOLUME; HUMANS; MALE; PREGNANCY; PREGNANCY COMPLICATIONS; QUINOLONES; RETROSPECTIVE STUDIES; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84888059709     PISSN: 15691993     EISSN: 18735010     Source Type: Journal    
DOI: 10.1016/j.jcf.2013.05.006     Document Type: Article

References (11)

1. Yu H., Burton B., Huang C.J., Worley J., Cao D., Johnson J.P., et al. Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros 2012, 11:237-245.
2. Ramsey B.W., Davies J., McElvaney N.G., Tullis E., Bell S.C., Dřevínek P., et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011, 365:1663-1672.
3. Accurso F.J., Rowe S.M., Clancy J.P., Boyle M.P., Dunitz J.M., Durie P.R., et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med 2010, 363:1991-2003.
4. Davies J.C., Wainwright C.E., Canny G.J., Chilvers M.A., Howenstine M.S., Munck A., et al. Efficacy and safety of ivacaftor in patients aged 6 to 11years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med Apr 3 2013, [Epub ahead of print].
5. U.S. Food and Drug Administration News Release http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289633.htm.
6. European Medicines Agency EPAR summary for the public: Kalydeco EMA/349471/2012. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/002494/WC500130744.pdf.
7. Sherrill D.L., Lebowitz M.D., Knudson R.J., Burrows B. Continuous longitudinal regression equations for pulmonary function measures. Eur Respir J 1992, 5:452-462.
8. Quanjer P.H., Tammeling G.J., Cotes J.E., Pedersen O.F., Peslin R., Yernault J.-C. Lung volumes and forced ventilatory flows. Eur Respir J 1993, 16:5-40. [Suppl.].
9. Shah P.I., Bush A., Canny G.J., Colin A.A., Fuchs H.J., Geddes D.M., et al. Recombinant human DNase I in cystic fibrosis patients with severe pulmonary disease: a short-term, double-blind study followed by six months open-label treatment. Eur Respir J 1995, 8:954-958.
10. Polenakovik H.M., Sanville B. The use of ivacaftor in an adult with severe lung disease due to cystic fibrosis (δF508/G551D). J Cyst Fibros Jan 9 2013, [pii: S1569-1993(12)00237-8, Epub ahead of print]. 10.1016/j.jcf.2012.12.004.
11. McKone E., Li H., Davies J.C. 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. on behalf of the VX08-770-105 Study Group.