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J Cyst Fibros. 2017 Mar;16(2):267-274. doi: 10.1016/j.jcf.2016.12.004. Epub 2017 Jan 26.

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

Author information

1
Department of Radiology, One Hospital Drive, University of Missouri, Columbia, MO 65212, USA. Electronic address: altest@health.missouri.edu.
2
Vertex Pharmaceuticals Incorporated, 50 Northern Ave Boston, MA 02210, USA. Electronic address: mac_johnson@vrtx.com.
3
Vertex Pharmaceuticals Incorporated, 50 Northern Ave Boston, MA 02210, USA. Electronic address: meredith_fidler@vrtx.com.
4
Vertex Pharmaceuticals Incorporated, 50 Northern Ave Boston, MA 02210, USA. Electronic address: martynbotfield@gmail.com.
5
University of Virginia, 480 Ray C. Hunt Drive, Snyder Building, Office 124, Charlottesville, VA 22903, USA. Electronic address: ntustison@gmail.com.
6
University of Virginia, University Hospital, First Floor 1215 Lee St., Charlottesville, VA, 22908, USA. Electronic address: carlosleivasalinas@gmail.com.
7
University of Virginia, University Hospital, First Floor 1215 Lee St., Charlottesville, VA, 22908, USA. Electronic address: eed6s@virginia.edu.
8
University of Virginia, 100 Hospital Dr #5408, Charlottesville, VA 22903, USA. Electronic address: dkf2x@virginia.edu.
9
University of Virginia, Snyder Translational Research Building - Room 154, 480 Ray C. Hunt Drive, Charlottesville, VA 22903, USA. Electronic address: jpm7r@virginia.edu.

Abstract

BACKGROUND:

This pilot study evaluated the effect of short- and long-term ivacaftor treatment on hyperpolarized 3He-magnetic resonance imaging (MRI)-defined ventilation defects in patients with cystic fibrosis aged ≥12years with a G551D-CFTR mutation.

METHODS:

Part A (single-blind) comprised 4weeks of ivacaftor treatment; Part B (open-label) comprised 48weeks of treatment. The primary outcome was change from baseline in total ventilation defect (TVD; total defect volume:total lung volume ratio).

RESULTS:

Mean change in TVD ranged from -8.2% (p=0.0547) to -12.8% (p=0.0078) in Part A (n=8) and -6.3% (p=0.1953) to -9.0% (p=0.0547) in Part B (n=8) as assessed by human reader and computer algorithm, respectively.

CONCLUSIONS:

TVD responded to ivacaftor therapy. 3He-MRI provides an individual quantification of disease burden that may be able to detect aspects of the disease missed by population-based spirometry metrics. Assessments by human reader and computer algorithm exhibit similar trends, but the latter appears more sensitive. www.clinicaltrials.gov identifier: NCT01161537.

KEYWORDS:

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

PMID:
28132845
DOI:
10.1016/j.jcf.2016.12.004
[Indexed for MEDLINE]
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