Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR Jennifer L. Taylor-Cousar, Manu Jain, Tara.

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Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR Jennifer L. Taylor-Cousar, Manu Jain, Tara Lynn Barto, Tarik Haddad, Jeffrey Atkinson, Simon Tian, Rui Tang, Gautham Marigowda, David Waltz, Joseph Pilewski Journal of Cystic Fibrosis Volume 17, Issue 2, Pages 228-235 (March 2018) DOI: 10.1016/j.jcf.2017.09.012 Copyright © 2017 The Authors Terms and Conditions

Fig. 1 Study design. IVA, ivacaftor; LUM, lumacaftor; q12h, every 12h. *Dose modification to LUM 200mg q12h/IVA 125mg q12h (half dose) was permitted (at initiation or at any point during the study) at the discretion of the treating physician and after discussion with and approval by the medical monitor. Journal of Cystic Fibrosis 2018 17, 228-235DOI: (10.1016/j.jcf.2017.09.012) Copyright © 2017 The Authors Terms and Conditions

Fig. 2 Absolute change from baseline in (A) ppFEV1, (B) CFQ-R respiratory domain score, (C) sweat chloride, and (D) BMI at each study visit. Panels A–C are based on MMRM analysis, and panel D is based on descriptive statistics. The number of patients is noted below each corresponding study visit. BMI, body mass index; CFQ-R, Cystic Fibrosis Questionnaire–Revised; CI, confidence interval; LS, least squares; MMRM, mixed-effects model for repeated measures; ppFEV1, percent predicted forced expiratory volume in 1s; SE, standard error. Journal of Cystic Fibrosis 2018 17, 228-235DOI: (10.1016/j.jcf.2017.09.012) Copyright © 2017 The Authors Terms and Conditions

Fig. 3 (A) Total duration of IV antibiotics for sinopulmonary signs and symptoms and (B) annualized all-cause hospitalization event rate in the 24weeks prior to the study and through 24weeks on study drug (N=46). *Wilcoxon signed-rank test. †Number of hospitalizations divided by the total number of days during the period was used to calculate event rate; annualized event rate (ie, event rate per year) was derived by multiplying the event rate by 48weeks (7days/week). ‡A Poisson distribution of the number of events on each period was assumed. The log of rate ratio thus approximated the normal distribution. The definition of hospitalization pre-study may not be the same as protocol-defined hospitalization. The p value was for testing the null hypothesis that the log of rate ratio=0. CI, confidence interval; IV, intravenous; SE, standard error. Journal of Cystic Fibrosis 2018 17, 228-235DOI: (10.1016/j.jcf.2017.09.012) Copyright © 2017 The Authors Terms and Conditions