Efficacy response in CF patients treated with ivacaftor: post-hoc analysis

Pediatric Pulmonology
Michael W KonstanCharles Johnson

Abstract

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 characteristic...Continue Reading

References

Jul 3, 2003·The Journal of Pediatrics·Michael W KonstanUNKNOWN Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis
Jul 24, 2007·The Journal of Pediatrics·Michael W KonstanUNKNOWN Scientific Advisory Group and the Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis
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May 14, 2010·American Journal of Respiratory and Critical Care Medicine·Don B SandersChristopher H Goss
Nov 4, 2011·The New England Journal of Medicine·Bonnie W RamseyUNKNOWN VX08-770-102 Study Group
Feb 2, 2012·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Haihui YuFredrick Van Goor
May 9, 2012·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Michael W KonstanWayne J Morgan
Apr 18, 2013·American Journal of Respiratory and Critical Care Medicine·Jane C DaviesUNKNOWN VX08-770-103 (ENVISION) Study Group
May 1, 2013·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Verena I SeligerPeter Mueller
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Jun 14, 2014·American Journal of Respiratory and Critical Care Medicine·Steven M RoweUNKNOWN GOAL Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network

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Citations

Jan 9, 2016·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Tomáš MlčochMilan Macek
Jun 21, 2015·Drugs·Peter J Barry, Andrew M Jones
May 14, 2016·Pediatric Pulmonology·Adrienne P Savant, Susanna A McColley
Oct 4, 2016·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Jacqueline L AndersonAudrey C Tierney
Oct 11, 2016·Expert Review of Respiratory Medicine·Valerie WatersFelix Ratjen
Jan 8, 2019·The Cochrane Database of Systematic Reviews·Mica SkiltonKevin W Southern
Jun 14, 2020·Journal of the Academy of Nutrition and Dietetics·Julianna BaileyJessica A Alvarez
Jun 23, 2020·Journal of the Academy of Nutrition and Dietetics·Catherine M McDonaldMary Rozga

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