Table 2.
Summary of key safety outcomes across US and UK annual analyses of ivacaftor versus comparator safety cohorts
| Relative risk (95% CI)/Fisher's exact P valuea | 2012 | 2013 | 2014 | 2015 | 2016 |
|---|---|---|---|---|---|
| US CFFPR | |||||
| Death | 0.43 (0.17, 1.07) | 0.37 (0.15, 0.93) | 0.41 (0.20, 0.84) | 0.18 (0.07, 0.44) | 0.41 (0.20, 0.86) |
| Organ transplants | 0.22 (0.05, 0.89) | 0.19 (0.05, 0.76) | 0.15 (0.04, 0.59) | 0.55 (0.27, 1.09) | 0.29 (0.13, 0.67) |
| Pulmonary exacerbation | 0.70 (0.62, 0.78) | 0.62 (0.56, 0.69) | 0.64 (0.58, 0.70) | 0.69 (0.63, 0.76) | 0.72 (0.66, 0.79) |
| Hospitalizationsb | 0.67 (0.60, 0.76) | 0.59 (0.53, 0.66) | 0.64 (0.58, 0.70) | 0.66 (0.60, 0.72) | 0.67 (0.62, 0.73) |
| UK CFR | |||||
| Death | NA | 0.45, P = 0.41 | 0.52 (0.16, 1.70) | 0.75 (0.27, 2.15) | 0.47, P = 0.41 |
| Organ transplants | NA | 0.76, P = 1.00 | 0.56, P = 0.56 | NA, P = 0.06 | 0.24, P = 0.20 |
| Pulmonary exacerbation | NA | 0.89 (0.79, 1.00) | 0.61 (0.53, 0.70) | 0.61 (0.53, 0.70) | 0.58 (0.50, 0.67) |
| Hospitalization due to pulmonary exacerbation | NA | 0.87 (0.75, 1.01) | 0.57 (0.48, 0.68) | 0.62 (0.53, 0.73) | 0.57 (0.48, 0.67) |
NA not applicable
aFisher's exact P values are shown when the expected value is < 5 in ≥ 1 cell of the contingency table
bReasons for hospitalization included pulmonary exacerbation, pulmonary complication, gastrointestinal complication, transplant related, sinus infection, nontransplant surgery, and other