Efficacy response in CF patients treated with ivacaftor: Post‐hoc analysis. Issue 5 (9th March 2015)

• Record Type: Journal Article
• Authors: Konstan, Michael W.; Plant, Barry J.; Elborn, J. Stuart; Rodriguez, Sally; Munck, Anne; Ahrens, Richard; Johnson, Charles
• Is Part Of: Pediatric pulmonology. Volume 50:Issue 5(2015:May)
• Page Start: 447
• Page End: 455
• Publication Date: 2015-03-09
• Subjects: Pediatric respiratory diseases -- Periodicals; Pediatrics -- Periodicals; 618.922
• DOI: 10.1002/ppul.23173
• Languages: English
• ISSNs: 8755-6863

Abstract

<abstract abstract-type="main" xml:lang="en"> <title>Summary</title> <sec id="ppul23173-sec-0001" sec-type="section"> <p>Clinical studies in patients with cystic fibrosis and <italic>G551D‐CFTR</italic> showed that the group treated with ivacaftor had improved clinical outcomes. To better understand the effect of ivacaftor therapy across the distribution of individual FEV<sub>1</sub> responses, data from Phase 3 studies (STRIVE/ENVISION) were re‐examined.</p> <p>In this post‐hoc analysis of patients (n = 209) who received 48 weeks of ivacaftor or placebo, patients were assigned to tertiles according to FEV<sub>1</sub> response. These groups were then used to evaluate response (FEV<sub>1</sub>, sweat chloride, weight, CFQ‐R, and pulmonary exacerbation). The number needed to treat (NNT) was calculated for specific thresholds for each outcome.</p> <p>Across all tertiles, numerical improvements in FEV<sub>1</sub>, sweat chloride, CFQ‐R and the frequency of pulmonary exacerbations were observed in ivacaftor‐treated patients: the treatment difference versus placebo was statistically significant for all outcomes in the upper tertile and for some outcomes in the lower and middle tertiles. The NNT for a ≥ 5% improvement in %predicted FEV<sub>1</sub> was 1.90, for a ≥ 5% body weight increase was 5.74, and to prevent a pulmonary exacerbation was 3.85.</p> <p>This analysis suggests that the majority of patients with clinical characteristics similar to STRIVE/ENVISION patients have the potential to benefit from ivacaftor therapy. <bold>Pediatr Pulmonol. 2015; 50:447–455.</bold> © 2015 Wiley Periodicals, Inc.</p> </sec> </abstract>