Table 2. Primary and Secondary Outcomes Among Patients With Hereditary Angioedema Attacks Taking Lanadelumab vs Placeboa.
| Lanadelumab | Placebo (n = 41) | |||
|---|---|---|---|---|
| Every 4 Weeks | 300 mg Every 2 Weeks (n = 27) | |||
| 150 mg (n = 28) | 300 mg (n = 29) | |||
| Primary End Point | ||||
| No. of attacks per mo, d 0-182 | ||||
| Mean (95% CI)b,c | 0.48 (0.31 to 0.73) | 0.53 (0.36 to 0.77) | 0.26 (0.14 to 0.46) | 1.97 (1.64 to 2.36) |
| Difference (95% CI)d | −1.49 (−1.90 to −1.08) | −1.44 (−1.84 to −1.04) | −1.71 (−2.09 to −1.33) | |
| P value | <.001 | <.001 | <.001 | |
| Rate ratio (95% CI)c | 0.24 (0.15 to 0.39) | 0.27 (0.18 to 0.41) | 0.13 (0.07 to 0.24) | |
| P valuee | <.001 | <.001 | <.001 | |
| Secondary End Points | ||||
| No. of attacks requiring acute treatment per mo, d 0-182 | ||||
| Mean (95% CI)b,c | 0.31 (0.18 to 0.53) | 0.42 (0.28 to 0.65) | 0.21 (0.11 to 0.40) | 1.64 (1.34 to 2.00) |
| Difference (95% CI)d | −1.32 (−1.69 to −0.95) | −1.21 (−1.58 to −0.85) | −1.43 (−1.78 to −1.07) | |
| P value | <.001 | <.001 | <.001 | |
| Rate ratio (95% CI)c | 0.19 (0.11 to 0.34) | 0.26 (0.16 to 0.41) | 0.13 (0.07 to 0.25) | |
| P valuee | <.001 | <.001 | <.001 | |
| No. of moderate or severe attacks per mo, d 0-182 | ||||
| Mean (95% CI)b,c | 0.36 (0.22 to 0.58) | 0.32 (0.20 to 0.53) | 0.20 (0.11 to 0.39) | 1.22 (0.97 to 1.52) |
| Difference (95% CI)d | −0.86 (−1.18 to −0.53) | −0.89 (−1.20 to −0.58) | −1.01 (−1.32 to −0.71) | |
| P value | <.001 | <.001 | <.001 | |
| Rate ratio (95% CI)c | 0.30 (0.17 to 0.50) | 0.27 (0.16 to 0.46) | 0.17 (0.08 to 0.33) | |
| P valuee | <.001 | <.001 | <.001 | |
| No. of attacks per mo, d 14-182 | ||||
| Mean (95% CI)b,c | 0.44 (0.28 to 0.70) | 0.49 (0.33 to 0.73) | 0.22 (0.12 to 0.41) | 1.99 (1.65 to 2.39) |
| Difference (95% CI)d | −1.54 (−1.96 to −1.12) | −1.50 (−1.91 to −1.09) | −1.77 (−2.16 to −1.38) | |
| P value | <.001 | <.001 | <.001 | |
| Rate ratio (95% CI)c | 0.22 (0.14 to 0.36) | 0.25 (0.16 to 0.38) | 0.11 (0.06 to 0.21) | |
| P valuee | <.001 | <.001 | <.001 | |
a
All patients received injections every 2 weeks, with those in the every-4-week groups receiving placebo in between active treatments.
b
Attack rates are model-based mean attacks per month, defined as 4 weeks.
c
Results are from a Poisson regression model accounting for overdispersion; treatment group and normalized baseline attack rate were fixed effects. The logarithm of time (days) each patient was observed during the treatment period was an offset variable. All P values (Wald test) reported vs placebo.
d
Estimated from a nonlinear function of the model parameters. All P values (Wald test) reported vs placebo.
e
P value adjusted for multiple testing.