40 IU/kg (N = 45) | 60 IU/kg (N = 45) | |||
---|---|---|---|---|
C1-INH (SC) | Placebo | C1-INH (SC) | Placebo | |
Primary efficacy endpoint | ||||
Number of time-normalized HAE attacks/montha—mean (95% CI) | 1.19 (0.54, 1.85) | 3.61 (2.96, 4.26) | 0.52 (0.00, 1.04) | 4.03 (3.51, 4.55) |
Secondary efficacy endpoint | ||||
Uses of rescue medication/montha—mean (95% CI) | 1.13 (− 1.44, 3.69) | 5.55 (3.10, 8.00) | 0.32 (− 0.33, 0.97) | 3.89 (3.23, 4.55) |
Pre-specified exploratory endpoints | ||||
Time-normalized sum of symptom severity scores | ||||
Mean (SD) | 0.10 (0.19) | 0.44 (0.40) | 0.07 (0.15) | 0.45 (0.33) |
Median (range) | 0.02 (0, 0.9) | 0.36 (0, 2.0) | 0.01 (0, 0.9) | 0.38 (0, 1.5) |
N | 43 | 44 | 43 | 42 |
Time-normalized sum of symptom severity scores combined with rescue medication use | ||||
Mean (SD) | 0.17 (0.35) | 0.73 (0.69) | 0.09 (0.18) | 0.73 (0.51) |
Median (range) | 0.04 (0, 1.8) | 0.59 (0, 3.5) | 0.02 (0, 0.9) | 0.65 (0.1, 2.4) |
N | 43 | 44 | 43 | 42 |
Duration of HAE attacks per patient, days | ||||
Mean (SD) | 1.80 (1.08) | 2.08 (1.21) | 1.58 (0.98) | 1.64 (0.66) |
Median (range) | 1.57 (1.0, 5.8) | 1.71 (1.0, 7.0) | 1.0 (1.0, 5.5) | 1.45 (1.0, 4.0) |
Post hoc analysis of treated versus untreated HAE attacks | ||||
Number (%) of patients with HAE attacks | 26 (57.8) | 40 (88.9) | 25 (55.6) | 42 (93.3) |
Number (%) of patients without HAE attacks | 17 (37.8) | 4 (8.9) | 18 (40.0) | 0 |
Number (%) of patients with missing data about occurrence of HAE attacks | 2 (4.4) | 1 (2.2) | 2 (4.4) | 3 (6.7) |
Number of all HAE attacks | 145 | 503 | 71 | 472 |
Number (%) of HAE attacks treated with rescue medication | 99 (68.3) | 421 (83.7) | 35 (49.3) | 358 (75.8) |
With 1 rescue medication | 92 (92.9) | 375 (89.1) | 35 (100) | 318 (88.8) |
C1-INH (IV) Berinert® | 83 (90.2) | 338 (90.1) | 28 (80.0) | 271 (85.2) |
C1-INH (not Berinert IV) | 0 | 1 (0.3) | 0 | 5 (1.6) |
Icatibant (Firazyr®) | 8 (8.7) | 58 (15.5) | 7 (20.0) | 63 (19.8) |
Other | 1 (1.1) | 11 (2.9) | 0 | 1 (0.3) |
With 2 rescue medications | 7 (7.1) | 33 (7.8) | 0 | 27 (7.5) |
C1-INH (IV) Berinert | 7 (100) | 14 (42.4) | 0 | 6 (22.2) |
C1-INH (not Berinert IV) | 0 | 0 | 0 | 0 |
Icatibant (Firazyr) | 0 | 10 (30.3) | 0 | 17 (63.0) |
Other | 0 | 1 (3.0) | 0 | 0 |
With ≥ 3 rescue medications | 0 | 13 (3.1) | 0 | 16 (3.6) |
C1-INH (IV) Berinert | 0 | 5 (38.5) | 0 | 2 (15.4) |
C1-INH (not Berinert IV) | 0 | 0 | 0 | 1 (7.7) |
Icatibant (Firazyr) | 0 | 4 (30.8) | 0 | 7 (53.8) |
Other | 0 | 0 | 0 | 0 |