Study design (if review, criteria of inclusion for studies)
Data summarized from a placebo-controlled trial
Participants
161 cystic fibrosis patients>/=12years old with the G551D-CFTR mutation
Interventions
Ivacaftor vs placebo.
Outcome measures
Pulmonary exacerbations (PEx). Short-term recovery was measured 2 to 8weeks after treatment, and long-term recovery was determined at the end-of-study, both compared with baseline measured just prior to the PEx.
Main results
Fewer patients receiving ivacaftor experienced a PEx than patients receiving placebo (33.7% vs. 56.4%; P=0.004) and had a lower adjusted incidence rate of PEx (0.589 vs. 1.382; P<0.001). The proportion of PEx followed by full short-term recovery of percent predicted forced expiratory volume in 1s was similar (ivacaftor vs. placebo, 57.1% vs. 53.7), as was the proportion of patients having long-term recovery (46.4% vs. 47.7%).
Authors' conclusions
Ivacaftor treatment reduces the frequency of PEx but does not improve on the rate of complete lung function recovery after PEx when compared with placebo.