Premium
Efficacy response in CF patients treated with ivacaftor: Post‐hoc analysis
Author(s) -
Konstan Michael W.,
Plant Barry J.,
Elborn J. Stuart,
Rodriguez Sally,
Munck Anne,
Ahrens Richard,
Johnson Charles
Publication year - 2015
Publication title -
pediatric pulmonology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.866
H-Index - 106
eISSN - 1099-0496
pISSN - 8755-6863
DOI - 10.1002/ppul.23173
Subject(s) - ivacaftor , medicine , exacerbation , post hoc analysis , placebo , cystic fibrosis , cystic fibrosis transmembrane conductance regulator , pathology , alternative medicine
Summary Clinical studies in patients with cystic fibrosis and G551D‐CFTR 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 1 responses, data from Phase 3 studies (STRIVE/ENVISION) were re‐examined. 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 1 response. These groups were then used to evaluate response (FEV 1 , sweat chloride, weight, CFQ‐R, and pulmonary exacerbation). The number needed to treat (NNT) was calculated for specific thresholds for each outcome. Across all tertiles, numerical improvements in FEV 1 , 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 1 was 1.90, for a ≥ 5% body weight increase was 5.74, and to prevent a pulmonary exacerbation was 3.85. This analysis suggests that the majority of patients with clinical characteristics similar to STRIVE/ENVISION patients have the potential to benefit from ivacaftor therapy. Pediatr Pulmonol. 2015; 50:447–455. © 2015 Wiley Periodicals, Inc.