Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study

The Lancet Respiratory Medicine

Volumn 5, Issue 2, 2017, Pages 107-118

  Konstan, Michael W ^a   McKone, Edward F ^b   Moss, Richard B ^c   Marigowda, Gautham ^d   Tian, Simon ^d   Waltz, David ^d   Huang, Xiaohong ^d   Lubarsky, Barry ^d   Rubin, Jaime ^d   Millar, Stefanie J ^e   Pasta, David J ^e   Mayer Hamblett, Nicole ^f,g   Goss, Christopher H ^g   Morgan, Wayne ^h   Sawicki, Gregory S ⁱ  

^a CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b ST VINCENT S UNIVERSITY HOSPITAL   (Ireland)

^c STANFORD UNIVERSITY   (United States)

^d VERTEX PHARMACEUTICALS   (United States)

^e ICON CLINICAL RESEARCH   (United States)

^f CYSTIC FIBROSIS FOUNDATION   (United States)

^g UNIVERSITY OF WASHINGTON   (United States)

^h UNIVERSITY OF ARIZONA   (United States)

ⁱ BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

IVACAFTOR PLUS LUMACAFTOR; PLACEBO; 1,3 BENZODIOXOLE DERIVATIVE; AMINOPHENOL DERIVATIVE; AMINOPYRIDINE DERIVATIVE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR DELTA F508; LUMACAFTOR, IVACAFTOR DRUG COMBINATION; QUINOLONE DERIVATIVE;

ABNORMAL LABORATORY RESULT; AGE DISTRIBUTION; ARTICLE; BLOOD PRESSURE; CHILD; COHORT ANALYSIS; COMBINATION CHEMOTHERAPY; COMORBIDITY; COUGHING; CYSTIC FIBROSIS; DISEASE EXACERBATION; DISEASE REGISTRY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; FORCED EXPIRATORY VOLUME; GENE DELETION; GENE MUTATION; HEMOPTYSIS; HOMOZYGOSITY; HUMAN; LONG TERM CARE; LUNG INFECTION; MEDICAL HISTORY; MULTICENTER STUDY (TOPIC); PATIENT COMPLIANCE; PHASE 3 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL (TOPIC); SCHOOL CHILD; SIDE EFFECT; SPUTUM ANALYSIS; TREATMENT DURATION; ADOLESCENT; CLINICAL TRIAL; COMPLICATION; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG ADMINISTRATION; DRUG COMBINATION; DRUG EFFECTS; FEMALE; GENETICS; HOMOZYGOTE; LUNG; MALE; MULTICENTER STUDY; MUTATION; PATHOPHYSIOLOGY; PHASE 3 CLINICAL TRIAL; RANDOMIZED CONTROLLED TRIAL; TIME; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; AMINOPHENOLS; AMINOPYRIDINES; BENZODIOXOLES; CHILD; COUGH; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; DRUG ADMINISTRATION SCHEDULE; DRUG COMBINATIONS; FEMALE; FORCED EXPIRATORY VOLUME; HOMOZYGOTE; HUMANS; LUNG; MALE; MUTATION; QUINOLONES; TIME; TREATMENT OUTCOME; YOUNG ADULT;

EID: 85008221944     PISSN: 22132600     EISSN: 22132619     Source Type: Journal    
DOI: 10.1016/S2213-2600(16)30427-1     Document Type: Article

References (30)

1. 1 Elborn, JS, Cystic fibrosis. Lancet 388 (2016), 2519–2531.
2. 2 Konstan, MW, Butler, SM, Wohl, ME, et al. Growth and nutritional indexes in early life predict pulmonary function in cystic fibrosis. J Pediatr 142 (2003), 624–630.
3. 3 Konstan, MW, Morgan, WJ, Butler, SM, et al. Risk factors for rate of decline in forced expiratory volume in one second in children and adolescents with cystic fibrosis. J Pediatr 151 (2007), 134–139.
4. 4 Corey, M, Edwards, L, Levison, H, Knowles, M, Longitudinal analysis of pulmonary function decline in patients with cystic fibrosis. J Pediatr 131 (1997), 809–814.
5. 5 Schluchter, MD, Konstan, MW, Davis, PB, Jointly modelling the relationship between survival and pulmonary function in cystic fibrosis patients. Stat Med 21 (2002), 1271–1287.
6. 6 Davis, PB, Byard, PJ, Konstan, MW, Identifying treatments that halt progression of pulmonary disease in cystic fibrosis. Pediatr Res 41 (1997), 161–165.
7. 7 de Boer, K, Vandemheen, KL, Tullis, E, et al. Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis. Thorax 66 (2011), 680–685.
8. 1 in adults with cystic fibrosis. J Cyst Fibros 11 (2012), 405–411.
9. 9 Waters, V, Stanojevic, S, Atenafu, EG, et al. Effect of pulmonary exacerbations on long-term lung function decline in cystic fibrosis. Eur Respir J 40 (2012), 61–66.
10. 10 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 106 (2009), 18825–18830.
11. 11 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 187 (2013), 1219–1225.
12. 12 McKone, EF, Borowitz, D, Drevinek, P, et al. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med 2 (2014), 902–910.
13. 13 Ramsey, BW, Davies, J, McElvaney, NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 365 (2011), 1663–1672.
14. 14 Sawicki, GS, McKone, EF, Pasta, DJ, et al. Sustained benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. Am J Respir Crit Care Med 192 (2015), 836–842.
15. 15 Flume, PA, Liou, TG, Borowitz, DS, et al. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. Chest 142 (2012), 718–724.
16. 16 Van Goor, F, Hadida, S, Grootenhuis, PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 108 (2011), 18843–18848.
17. 17 Clancy, JP, Rowe, SM, Accurso, FJ, et al. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax 67 (2012), 12–18.
18. 18 Wainwright, CE, Elborn, JS, Ramsey, BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 373 (2015), 220–231.
19. 19 Hankinson, JL, Odencrantz, JR, Fedan, KB, Spirometric reference values from a sample of the general U.S. population. Am J Respir Crit Care Med 159 (1999), 179–187.
20. 20 Wang, X, Dockery, DW, Wypij, D, Fay, ME, Ferris, BG Jr, Pulmonary function between 6 and 18 years of age. Pediatr Pulmonol 15 (1993), 75–88.
21. 21 Quanjer, PH, Stanojevic, S, Cole, TJ, et al. Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equations. Eur Respir J 40 (2012), 1324–1343.
22. 22 Rubin, DB, Thomas, N, Matching using estimated propensity scores: relating theory to practice. Biometrics 52 (1996), 249–264.
23. 23 Schaedel, C, de Monestrol, I, Hjelte, L, et al. Predictors of deterioration of lung function in cystic fibrosis. Pediatr Pulmonol 33 (2002), 483–491.
24. 24 Cystic Fibrosis Foundation Patient Registry 2014 Annual Data Report, 2015, CF Foundation, Bethesda, MD.
25. 1 decline in cystic fibrosis. Pediatr Pulmonol 46 (2011), 545–553.
26. 1 in cystic fibrosis. Pediatr Pulmonol 49 (2014), 529–536.
27. 27 Konstan, MW, Byard, PJ, Hoppel, CL, Davis, PB, Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med 332 (1995), 848–854.
28. 28 Stephenson, AL, Tom, M, Berthiaume, Y, et al. A contemporary survival analysis of individuals with cystic fibrosis: a cohort study. Eur Respir J 45 (2015), 670–679.
29. 29 Goss, CH, MacNeill, SJ, Quinton, HB, et al. Children and young adults with CF in the USA have better lung function compared with the UK. Thorax 70 (2015), 229–236.
30. 30 Martin, B, Schechter, MS, Jaffe, A, Cooper, P, Bell, SC, Ranganathan, S, Comparison of the US and Australian cystic fibrosis registries: the impact of newborn screening. Pediatrics 129 (2012), e348–e355.