CORK Study in Cystic Fibrosis: Sustained Improvements in Ultra-Low-Dose Chest CT Scores After CFTR Modulation With Ivacaftor

Chest

Volumn 153, Issue 2, 2018, Pages 395-403

  Ronan, Nicola J ^a   Einarsson, Gisli G ^b   Twomey, Maria ^a   Mooney, Denver ^b   Mullane, David ^a   NiChroinin, Muireann ^a   O'Callaghan, Grace ^a   Shanahan, Fergus ^c   Murphy, Desmond M ^a   O'Connor, Owen J ^a   Shortt, Cathy A ^a   Tunney, Michael M ^b   Eustace, Joseph A ^a   Maher, Michael M ^a   Elborn, J Stuart ^b,d   Plant, Barry J ^a  

^a University College Cork and INFANT Centre   (Ireland)

^b QUEEN'S UNIVERSITY BELFAST   (United Kingdom)

^c Department of Medicine ^*  (United Kingdom)

^d ROYAL BROMPTON HOSPITAL   (United Kingdom)

Author keywords

cystic fibrosis; G551D; ivacaftor; low dose chest CT imaging

Indexed keywords

ANTIBIOTIC AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; INTERLEUKIN 1BETA; INTERLEUKIN 6; INTERLEUKIN 8; IVACAFTOR; AMINOPHENOL DERIVATIVE; CHLORIDE CHANNEL STIMULATING AGENT; QUINOLONE DERIVATIVE;

ADOLESCENT; ADULT; ARTICLE; BHALLA SCORE; BLOOD SAMPLING; BODY MASS; CHILD; CLINICAL ARTICLE; CLINICAL ASSESSMENT; CLINICAL FEATURE; COHORT ANALYSIS; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; CYSTIC FIBROSIS; DISEASE EXACERBATION; DOSIMETRY; FEMALE; FORCED EXPIRATORY VOLUME; G551D GENE; GENE; GENE MUTATION; HUMAN; MALE; MICROBIOME; PLEURA THICKENING; POPULATION ABUNDANCE; PRIORITY JOURNAL; PROSPECTIVE STUDY; PROTEIN MODIFICATION; PSEUDOMONAS; RADIOLOGY; SCORING SYSTEM; SHUTTLE WALK TEST; SPUTUM CULTURE; SWEAT TEST; TREATMENT DURATION; TREATMENT RESPONSE; YOUNG ADULT; DIAGNOSTIC IMAGING; GENETICS; MICROBIOLOGY; PATHOPHYSIOLOGY; PHYSIOLOGY; PROCEDURES; SALIVA; THORAX RADIOGRAPHY; X-RAY COMPUTED TOMOGRAPHY;

ADOLESCENT; ADULT; AMINOPHENOLS; CHLORIDE CHANNEL AGONISTS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; FEMALE; FORCED EXPIRATORY VOLUME; HUMANS; MALE; PROSPECTIVE STUDIES; QUINOLONES; RADIOGRAPHY, THORACIC; SALIVA; TOMOGRAPHY, X-RAY COMPUTED; YOUNG ADULT;

EID: 85041896078     PISSN: 00123692     EISSN: 19313543     Source Type: Journal    
DOI: 10.1016/j.chest.2017.10.005     Document Type: Article

References (41)

1. Ramsey, B.W., Davies, J., McElvaney, N.G., et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 365:18 (2011), 1663–1672.
2. Davies, J.C., Wainwright, C.E., Canny, G.J., 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 187:11 (2013), 1219–1225.
3. De Boeck, K., Munck, A., Walker, S., et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros 13:6 (2014), 674–680.
4. Bobadilla, J.L., Macek, M. Jr., Fine, J.P., Farrell, P.M., Cystic fibrosis: a worldwide analysis of CFTR mutations—correlation with incidence data and application to screening. Hum Mutat 19:6 (2002), 575–606.
5. De Boeck, K., Zolin, A., Cuppens, H., Olesen, H., Viviani, L., The relative frequency of CFTR mutation classes in European patients with cystic fibrosis. J Cyst Fibros 13:4 (2014), 403–409.
6. Sheikh, S.I., Long, F.R., McCoy, K.S., Johnson, T., Ryan-Wenger, N.A., Hayes, D. Jr., Computed tomography correlates with improvement with ivacaftor in cystic fibrosis patients with G551D mutation. J Cyst Fibros 14:1 (2015), 84–89.
7. Rowe, S.M., Heltshe, S.L., Gonska, T., et al. Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator potentiator ivacaftor in G551D-mediated cystic fibrosis. Am J Respir Crit Care Med 190:2 (2014), 175–184.
8. Miller, M.R., Hankinson, J., Brusasco, V., et al. Standardisation of spirometry. Eur Respir J 26:2 (2005), 319–338.
9. Quittner, A.L., Buu, A., Messer, M.A., Modi, A.C., Watrous, M., Development and validation of The Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis. Chest 128:4 (2005), 2347–2354.
10. O'Connor, O.J., Vandeleur, M., McGarrigle, A.M., et al. Development of low-dose protocols for thin-section CT assessment of cystic fibrosis in pediatric patients 1. Radiology 257:3 (2010), 820–829.
11. Bhalla, M., Turcios, N., Aponte, V., et al. Cystic fibrosis: scoring system with thin-section CT. Radiology 179:3 (1991), 783–788.
12. Breen, E.C., Reynolds, S.M., Cox, C., et al. Multisite comparison of high-sensitivity multiplex cytokine assays. Clin Vaccine Immunol 18:8 (2011), 1229–1242.
13. Chowdhury, F., Williams, A., Johnson, P., Validation and comparison of two multiplex technologies, Luminex® and Mesoscale Discovery, for human cytokine profiling. J Immunol Methods 340:1 (2009), 55–64.
14. Lundberg, D.S., Yourstone, S., Mieczkowski, P., Jones, C.D., Dangl, J.L., Practical innovations for high-throughput amplicon sequencing. Nat Methods 10:10 (2013), 999–1002.
15. Yu, H., Burton, B., Huang, C.-J., et al. Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros 11:3 (2012), 237–245.
16. Van Goor, F., Hadida, S., Grootenhuis, P.D., et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci 106:44 (2009), 18825–188230.
17. European Medicines Agency. Committee for Medicinal Products for Human Use. Guideline on the clinical development of medicinal products for the treatment of cystic fibrosis. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500017055.pdf. Accessed January 2, 2017.
18. Murphy, K.P., O'Connell, O.J., O'Connor, O.J., Plant, B.J., Maher, M.M., Cumulative radiation exposure to abdominal organs in patients with cystic fibrosis should not be forgotten. Am J Respir Crit Care Med 190:8 (2014), 961–962.
19. O'Connell, O.J., McWilliams, S., McGarrigle, A., et al. Radiologic imaging in cystic fibrosis: cumulative effective dose and changing trends over 2 decades. Chest 141:6 (2012), 1575–1583.
20. Barry, P.J., Jones, A.M., Webb, A.K., Horsley, A.R., Sweat chloride is not a useful marker of clinical response to Ivacaftor. Thorax 69:6 (2014), 586–587.
21. Cox, M.J., Allgaier, M., Taylor, B., et al. Airway microbiota and pathogen abundance in age-stratified cystic fibrosis patients. PLoS One, 5(6), 2010, e11044.
22. Paganin, P., Fiscarelli, E.V., Tuccio, V., et al. Changes in cystic fibrosis airway microbial community associated with a severe decline in lung function. PLoS One, 10(4), 2015, e0124348.
23. Zhao, J., Schloss, P.D., Kalikin, L.M., et al. Decade-long bacterial community dynamics in cystic fibrosis airways. Proc Natl Acad Sci U S A 109:15 (2012), 5809–5814.
24. Shah, V.S., Ernst, S., Tang, X.X., et al. Relationships among CFTR expression, HCO3- secretion, and host defense may inform gene- and cell-based cystic fibrosis therapies. Proc Natl Acad Sci U S A 113:19 (2016), 5382–5387.
25. Seliger, V., Accurso, F., Konstan, M., Dong, Q., Lubarsky, B., Mueller, P., Effect of ivacaftor on circulating inflammatory indices in CF patients with the G551D-CFTR mutation. Pediatr Pulmonol 48:s36 (2013), 298–299.
26. Cantin, A., Cystic fibrosis lung inflammation: early, sustained, and severe. Am J Respir Crit Care Med 151:4 (1995), 939–941.
27. Khan, T.Z., Wagener, J.S., Bost, T., Martinez, J., Accurso, F.J., Riches, D., Early pulmonary inflammation in infants with cystic fibrosis. Am J Respir Crit Care Med, 151(4), 2011.
28. Regelmann, W.E., Siefferman, C.M., Herron, J.M., Elliott, G.R., Clawson, C.C., Gray, B.H., Sputum peroxidase activity correlates with the severity of lung disease in cystic fibrosis. Pediatr Pulmonol 19:1 (1995), 1–9.
29. Birrer, P., McElvaney, N., Rüdeberg, A., et al. Protease-antiprotease imbalance in the lungs of children with cystic fibrosis. Am J Respir Crit Care Med 150:1 (1994), 207–213.
30. Balough, K., McCubbin, M., Weinberger, M., Smits, W., Ahrens, R., Fick, R., The relationship between infection and inflammation in the early stages of lung disease from cystic fibrosis. Pediatr Pulmonol 20:2 (1995), 63–70.
31. Konstan, M.W., Hilliard, K.A., Norvell, T.M., Berger, M., Bronchoalveolar lavage findings in cystic fibrosis patients with stable, clinically mild lung disease suggest ongoing infection and inflammation. Am J Respir Crit Care Med 150:2 (1994), 448–454.
32. Muhlebach, M.S., Noah, T.L., Endotoxin activity and inflammatory markers in the airways of young patients with cystic fibrosis. Am J Respir Crit Care Med 165:7 (2002), 911–915.
33. Dakin, C.J., Numa, A.H., Wang, H., Morton, J.R., Vertzyas, C.C., Henry, R.L., Inflammation, infection, and pulmonary function in infants and young children with cystic fibrosis. Am J Respir Crit Care Med 165:7 (2002), 904–910.
34. Tirelli, A.S., Colombo, C., Torresani, E., et al. Effects of treatment in the levels of circulating cytokines and growth factors in cystic fibrosis and dialyzed patients by multi-analytical determination with a biochip array platform. Cytokine 62:3 (2013), 413–420.
35. Dean, T.P., Dai, Y., Shute, J.K., Church, M.K., Warner, J.O., Interleukin-8 concentrations are elevated in bronchoalveolar lavage, sputum, and sera of children with cystic fibrosis. Pediatr Res 34:2 (1993), 159–161.
36. Nixon, L.S., Yung, B., Bell, S.C., Stuart Elborn, J., Shale, D.J., Circulating immunoreactive interleukin-6 in cystic fibrosis. Am J Respir Crit Care Med 157:6 (1998), 1764–1769.
37. Norman, D., Elborn, J., Cordon, S., et al. Plasma tumour necrosis factor alpha in cystic fibrosis. Thorax 46:2 (1991), 91–95.
38. Schmitt-Grohé S., Naujoks, C., Bargon, J., et al. Interleukin-8 in whole blood and clinical status in cystic fibrosis. Cytokine 29:1 (2005), 18–23.
39. Corvol, H., Fitting, C., Chadelat, K., et al. Distinct cytokine production by lung and blood neutrophils from children with cystic fibrosis. Am J Physiol Lung Cell Mol Physiol 284:6 (2003), L997–L1003.
40. Pohl, K., Hayes, E., Keenan, J., et al. A neutrophil intrinsic impairment affecting Rab27a and degranulation in cystic fibrosis is corrected by CFTR potentiator therapy. Blood 124:7 (2014), 999–1009.
41. Bratcher, P.E., Rowe, S.M., Reeves, G., et al. Alterations in blood leukocytes of G551D-bearing cystic fibrosis patients undergoing treatment with ivacaftor. J Cyst Fibros 15:1 (2016), 67–73.