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primary studies published RCT

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

  • Year: 2015
  • Date: 2015
  • Author: Konstan MW
Source

Pediatr Pulmonol. 2015 May;50(5):447-55. Epub 2015 Mar 9.

Study design (if review, criteria of inclusion for studies)

data from Phase 3 studies (STRIVE/ENVISION) - post-hoc analysis

Participants

CF patients (n = 209)

Interventions

Post-ho analysis of patients (n = 209) who received 48 weeks of ivacaftor or placebo. Patients were assigned to tertiles according to FEV(1) response.

Outcome measures

FEV(1), sweat chloride, weight, CFQ-R, and pulmonary exacerbation

Main results

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.

Authors' conclusions

This analysis suggests that the majority of patients with clinical characteristics similar to STRIVE/ENVISION patients have the potential to benefit from ivacaftor therapy.

Related topics

CFTR modulators therapy

Keywords: Aminophenols; CFTR Modulators; Genetic Predisposition to Disease; pharmacological_intervention; VX-770; ivacaftor; G551D-CFTR;