ERS 2016. PA1257. Ivacaftor-as effective in clinical practice?
A. Aziz et al.
Results: 15 patients with CF were started on ivacaftor, mean age of 32 years (range 18-69). The mean predicted FEV1 was 59.6% (range 20-94%) at baseline. There was a significant increase in percentage points in predicted FEV1 (11.3% at 6 months, 7.5% at 1 year, 9.7% at 18 months and 11.8% at 2 years (p<0.0001), in BMI (7.2% at 6 months, 9.4% at 1 year, 8.4% at 18 months and 8.1% at 2 years (p=0.0083) and a decrease in sweat chloride from baseline (-47.2mmol/l at 1 year and -55mmol/l at 2 years (p<0.0001). There was a reduction in pulmonary exacerbations of 40.6% at 1 year and of 39.3% at 2 years. Only 3/15 patients showed a temporary derangement in LFTs. For 3/15 patients bone density measurements were available at baseline and 2 years later which showed an improvement irrespective of vitamin D levels. There was one successful pregnancy whilst on treatment in this cohort.
Conclusions: In practice, ivacaftor therapy has realised clinically relevant improvements in FEV1, BMI, pulmonary exacerbation rate and bone density in CF patients.
CARE Faculty Perspective: In clinical trials, Ivacaftor has demonstrated consistent efficacy and safety data for patients with cystic fibrosis. This study aimed to look at whether the efficacy results differed at all when used in real life clinical practice. Based on a retrospective analysis of 324 patients over the course of 3 years, results suggest ivacaftor does in fact treat CF effectively (demonstrated by improvements in FEV1, BMI, pulmonary exacerbation rate, and bone density).
Ivacaftor has also been investigated in combination with Lumacaftor. The combination (Orkambi®) therapy has recently been approved by FDA and Health Canada for CF patients homozygous for the most common CFTR mutation (F508del/F508del).
Better access to appropriate CF therapy could help to improve the quality and length of their lives. Cystic Fibrosis Canada has made a submission to the Canadian Drug Expert Committee (CDEC) of the Canadian Agency for Drugs and Technologies in Health (CADTH). [i] They have requested that Orkambi be reconsidered at the next CDEC meeting in September 2016. Moreover, they have requested that the CDEC consider the recommendations made by a panel of Cystic Fibrosis specialists across Canada regarding its clinical use.