Felix Ratjen, Todd Durham, Tomas Navratil, Amy Schaberg, Frank J

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Presentation transcript:

Long term effects of denufosol tetrasodium in patients with cystic fibrosis Felix Ratjen, Todd Durham, Tomas Navratil, Amy Schaberg, Frank J. Accurso, Claire Wainwright, Matthew Barnes, Richard B. Moss Journal of Cystic Fibrosis Volume 11, Issue 6, Pages 539-549 (December 2012) DOI: 10.1016/j.jcf.2012.05.003 Copyright © 2012 European Cystic Fibrosis Society. Terms and Conditions

Fig. 1 Patient disposition. ITT = intent-to-treat; TID = three times daily. Journal of Cystic Fibrosis 2012 11, 539-549DOI: (10.1016/j.jcf.2012.05.003) Copyright © 2012 European Cystic Fibrosis Society. Terms and Conditions

Fig. 2 Change from baseline in lung function by visit during the 48-week placebo-controlled period. Results are displayed for subjects with data at each time point. Values displayed are the adjusted means (and their standard errors) from an ANCOVA model with effects for pooled site and baseline value. CFB = change from baseline. (A) FEV1 (L); (B) FEV1 percent of predicted (%); (C) FEF25%–75% (L/s). Journal of Cystic Fibrosis 2012 11, 539-549DOI: (10.1016/j.jcf.2012.05.003) Copyright © 2012 European Cystic Fibrosis Society. Terms and Conditions

Fig. 3 Forest plot of the treatment effect (and 95% CI) for the change from baseline FEV1 at Week 48 endpoint for prespecified subgroups. Values displayed are the treatment effects (differences in adjusted mean change from baseline) and their 95% confidence intervals, estimated from an ANCOVA model with effects for pooled site and baseline value. ITT = intent-to-treat. Journal of Cystic Fibrosis 2012 11, 539-549DOI: (10.1016/j.jcf.2012.05.003) Copyright © 2012 European Cystic Fibrosis Society. Terms and Conditions

Fig. 4 Kaplan–Meier estimate of the time to first pulmonary exacerbation for three definitions of exacerbation. (A) Treatment with IV antibiotics for at least one respiratory sign or symptom. (B) Presence of at least 4 out of 12 signs and symptoms. (C) Investigator-defined event. Hazard ratios, their 95% confidence intervals, and P-values were obtained from a proportional hazards model with effects for treatment, pooled study site, and baseline FEV1. Journal of Cystic Fibrosis 2012 11, 539-549DOI: (10.1016/j.jcf.2012.05.003) Copyright © 2012 European Cystic Fibrosis Society. Terms and Conditions