1. Use of hyperpolarized helium-3 MRI to assess response to ivacaftor treatment in patients with cystic fibrosis 
Talissa A. Altes, Mac Johnson, Meredith Fidler, Martyn Botfield, Nicholas J. Tustison, Carlos Leiva-Salinas, Eduard E. de Lange, Deborah Froh, John P. Mugler 
Journal of Cystic Fibrosis 
Volume 16, Issue 2, Pages (March 2017)
DOI: /j.jcf
Copyright © 2016 The Authors Terms and Conditions

2. Fig. 1 Study design. *Screening visit (on the day before day 1 visit).
q12h, every 12h.
Journal of Cystic Fibrosis  , DOI: ( /j.jcf )
Copyright © 2016 The Authors Terms and Conditions

3. Fig. 2
Part A (n=8) primary outcomes: (A) change in TVDH; (B) change in TVDC.
Secondary ancillary outcomes: (C) absolute change in ppFEV1; (D) absolute change in sweat chloride; and (E) absolute change in CFQ-R respiratory health domain score. Day 15=baseline; day 43=end of ivacaftor treatment; day 57=end of placebo washout. Shaded area represents ivacaftor treatment period; change was only assessed from baseline (day 15) to end of ivacaftor treatment (day 43).
*p<0.05 for change from baseline at the end of ivacaftor treatment.
CFQ-R, Cystic Fibrosis Questionnaire–Revised; ppFEV1, percent predicted forced expiratory volume in 1s; SD, standard deviation; TVD, total ventilation defect.
Journal of Cystic Fibrosis  , DOI: ( /j.jcf )
Copyright © 2016 The Authors Terms and Conditions

4. Fig. 3
Part B (n=9) primary outcomes: (A) change in TVDH; (B) change in TVDC.
Secondary outcomes: (C) absolute change in ppFEV1; (D) absolute change in sweat chloride; and (E) absolute change in CFQ-R respiratory health domain score. Day 1=baseline; week 48=end of ivacaftor treatment; follow-up (week 50)=end of safety follow-up. Shaded area represents ivacaftor treatment period; change was only calculated from baseline (day 1) at the end of ivacaftor treatment (week 48).
*p<0.05 for change from baseline at the end of ivacaftor treatment. CFQ-R, Cystic Fibrosis Questionnaire–Revised; ppFEV1, percent predicted forced expiratory volume in 1s; SD, standard deviation; TVD, total ventilation defect.
Journal of Cystic Fibrosis  , DOI: ( /j.jcf )
Copyright © 2016 The Authors Terms and Conditions

5. Fig. 4
Coronal hyperpolarized 3He-MRI images of patient 002 in Part A. Typical images are shown (expanded image sets of the same case are given in Supplemental Fig. 4). Following inhalation of the hyperpolarized gas, well-ventilated lung regions appear bright white, and poorly ventilated regions (termed “ventilation defects”) appear dark gray/black. A) Baseline (day 15): ppFEV1: 62.2; TVDH: 51.7%; TVDC: 48.4%. B) End of 4weeks of ivacaftor treatment (day 43): ppFEV1: 83.0; TVDH: 27.1%; TVDC: 17.4%. C) End of 2weeks of placebo washout (day 57): ppFEV1: 71.6; TVDH: 57.4%; TVDC: 40.9%.
Journal of Cystic Fibrosis  , DOI: ( /j.jcf )
Copyright © 2016 The Authors Terms and Conditions