Use of hyperpolarized helium-3 MRI to assess response to ivacaftor treatment in patients with cystic fibrosis

Journal of Cystic Fibrosis

Volumn 16, Issue 2, 2017, Pages 267-274

  Altes, Talissa A ^a   Johnson, Mac ^b   Fidler, Meredith ^b   Botfield, Martyn ^b   Tustison, Nicholas J ^c   Leiva Salinas, Carlos ^d   de Lange, Eduard E ^d   Froh, Deborah ^c   Mugler, John P ^c  

^a University of Missouri   (United States)

^b VERTEX PHARMACEUTICALS   (United States)

^c UNIVERSITY OF VIRGINIA   (United States)

^d UNIVERSITY OF VIRGINIA HEALTH SYSTEM   (United States)

Author keywords

cystic fibrosis transmembrane conductance regulator modulator; forced expiratory volume; pulmonary

Indexed keywords

CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; HELIUM; HELIUM 3; IVACAFTOR; UNCLASSIFIED DRUG; AMINOPHENOL DERIVATIVE; CHLORIDE CHANNEL STIMULATING AGENT; HELIUM-3; ISOTOPE; QUINOLONE DERIVATIVE;

ADULT; ALGORITHM; ARTICLE; CYSTIC FIBROSIS; FEMALE; GENE MUTATION; HUMAN; HUMAN TISSUE; LUNG FUNCTION; LUNG VENTILATION; MALE; NUCLEAR MAGNETIC RESONANCE IMAGING; OPEN STUDY; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL; PILOT STUDY; READING; SINGLE BLIND PROCEDURE; TOTAL LUNG CAPACITY; CLINICAL TRIAL; DRUG ADMINISTRATION; DRUG EFFECTS; DRUG MONITORING; FORCED EXPIRATORY VOLUME; GENETICS; MIDDLE AGED; MUTATION; PHYSIOLOGY; PROCEDURES;

ADULT; AMINOPHENOLS; CHLORIDE CHANNEL AGONISTS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DRUG ADMINISTRATION SCHEDULE; DRUG MONITORING; FEMALE; FORCED EXPIRATORY VOLUME; HELIUM; HUMANS; ISOTOPES; MAGNETIC RESONANCE IMAGING; MALE; MIDDLE AGED; MUTATION; OUTCOME ASSESSMENT (HEALTH CARE); PILOT PROJECTS; PULMONARY VENTILATION; QUINOLONES; SINGLE-BLIND METHOD;

EID: 85010871752     PISSN: 15691993     EISSN: 18735010     Source Type: Journal    
DOI: 10.1016/j.jcf.2016.12.004     Document Type: Article

References (28)

1. [1] Cosio, M., Ghezzo, H., Hogg, J.C., et al. The relations between structural changes in small airways and pulmonary-function tests. N Engl J Med 298 (1978), 1277–1281.
2. [2] Verbanck, S., Schuermans, D., Meysman, M., Paiva, M., Vincken, W., Noninvasive assessment of airway alterations in smokers: the small airways revisited. Am J Respir Crit Care Med 170 (2004), 414–419.
3. [3] Macklem, P.T., Mead, J., Resistance of central and peripheral airways measured by a retrograde catheter. J Appl Physiol 22 (1967), 395–401.
4. [4] Gaffin, J.M., Shotola, N.L., Martin, T.R., Phipatanakul, W., Clinically useful spirometry in preschool-aged children: evaluation of the 2007 American Thoracic Society Guidelines. J Asthma 47 (2010), 762–767.
5. [5] Mentore, K., Froh, D.K., de Lange, E.E., Brookeman, J.R., Paget-Brown, A.O., Altes, T.A., Hyperpolarized HHe 3 MRI of the lung in cystic fibrosis: assessment at baseline and after bronchodilator and airway clearance treatment. Acad Radiol 12 (2005), 1423–1429.
6. [6] Kirby, M., Svenningsen, S., Ahmed, H., et al. Quantitative evaluation of hyperpolarized helium-3 magnetic resonance imaging of lung function variability in cystic fibrosis. Acad Radiol 18 (2011), 1006–1013.
7. [7] Sun, Y., O'Sullivan, B.P., Roche, J.P., et al. Using hyperpolarized 3He MRI to evaluate treatment efficacy in cystic fibrosis patients. J Magn Reson Imaging 34 (2011), 1206–1211.
8. [8] Bannier, E., Cieslar, K., Mosbah, K., et al. Hyperpolarized 3He MR for sensitive imaging of ventilation function and treatment efficiency in young cystic fibrosis patients with normal lung function. Radiology 255 (2010), 225–232.
9. [9] Woodhouse, N., Wild, J.M., van Beek, E.J., Hoggard, N., Barker, N., Taylor, C.J., Assessment of hyperpolarized 3He lung MRI for regional evaluation of interventional therapy: a pilot study in pediatric cystic fibrosis. J Magn Reson Imaging 30 (2009), 981–988.
10. [10] van Beek, E.J., Hill, C., Woodhouse, N., et al. Assessment of lung disease in children with cystic fibrosis using hyperpolarized 3-Helium MRI: comparison with Shwachman score, Chrispin-Norman score and spirometry. Eur Radiol 17 (2007), 1018–1024.
11. [11] McMahon, C.J., Dodd, J.D., Hill, C., et al. Hyperpolarized 3helium magnetic resonance ventilation imaging of the lung in cystic fibrosis: comparison with high resolution CT and spirometry. Eur Radiol 16 (2006), 2483–2490.
12. [12] Koumellis, P., van Beek, E.J., Woodhouse, N., et al. Quantitative analysis of regional airways obstruction using dynamic hyperpolarized 3He MRI-preliminary results in children with cystic fibrosis. J Magn Reson Imaging 22 (2005), 420–426.
13. [13] Donnelly, L.F., MacFall, J.R., McAdams, H.P., et al. Cystic fibrosis: combined hyperpolarized 3He-enhanced and conventional proton MR imaging in the lung—preliminary observations. Radiology 212 (1999), 885–889.
14. [14] Altes, T.A., Meyer, C.H., Mata, J.H., et al. Hyperpolarized helium-3 MR imaging of a non-sedated infant: a proof-of-concept study [abstract]. Proc Int Soc Magn Med, 20, 2012, 1355.
15. [15] Kalydeco [package insert]. 2015, Vertex Pharmaceuticals Incorporated, Boston, MA.
16. [16] 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 (2013), 1219–1225.
17. [17] 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 (2011), 1663–1672.
18. [18] 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 (2014), 674–680.
19. [19] Moss, R.B., Flume, P.A., Elborn, J.S., et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med 3 (2015), 524–533.
20. [20] Modi, A.C., Quittner, A.L., Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis. J Pediatr Psychol 28 (2003), 535–545.
21. [21] Quittner, A.L., Modi, A.C., Wainwright, C., Otto, K., Kirihara, J., Montgomery, A.B., Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa airway infection. Chest 135 (2009), 1610–1618.
22. [22] Tustison, N.J., Avants, B.B., Flors, L., et al. Ventilation-based segmentation of the lungs using hyperpolarized (3)He MRI. J Magn Reson Imaging 34 (2011), 831–841.
23. [23] Tustison, N.J., Contrella, B., Altes, T.A., Avants, B.B., de Lange, E.E., Mugler, J.P., Longitudinal assessment of treatment effects on pulmonary ventilation using 1H/3He MRI multivariate templates. Proc. SPIE 8672, Medical Imaging 2013: Biomedical Applications in Molecular, Structural, and Functional Imaging, 86720Y, March 29, 2013, 10.1117/12.2006951.
24. [24] Altes, T.A., Powers, P.L., Knight-Scott, J., et al. Hyperpolarized 3He MR lung ventilation imaging in asthmatics: preliminary findings. J Magn Reson Imaging 13 (2001), 378–384.
25. [25] Lee, E.Y., Sun, Y., Zurakowski, D., Hatabu, H., Khatwa, U., Albert, M.S., Hyperpolarized 3He MR imaging of the lung: normal range of ventilation defects and PFT correlation in young adults. J Thorac Imaging 24 (2009), 110–114.
26. [26] European Medicines Agency, Report of the workshop on endpoints for cystic fibrosis clinical trials. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2012/12/WC500136159.pdf, November 29, 2012 [accessed: June 18, 2015].
27. [27] Ramsey, B.W., Outcome measures for development of new therapies in cystic fibrosis: are we making progress and what are the next steps?. Proc Am Thorac Soc 4 (2007), 367–369.
28. [28] Tiddens, H.A., Puderbach, M., Venegas, J.G., et al. Novel outcome measures for clinical trials in cystic fibrosis. Pediatr Pulmonol 50 (2015), 302–315.