Table 1.
Baseline characteristics of patients in COMPACT long-term, open-label study (HRQoL population)
| C1-INH(SC) 40 IU/kg N = 63 |
C1-INH(SC) 60 IU/kg N = 63 |
All C1-INH(SC) N = 126 |
|
|---|---|---|---|
| Age, years, mean (SD)a | 40.8 (14.96) | 40.3 (16.26) | 40.5 (15.56) |
| Range | 8–67 | 10–72 | 8–72 |
| Female, n (%) | 40 (63.5) | 36 (57.1) | 76 (60.3) |
| Mean body weight, kg (SD) | 86.1 (23.27) | 84.26 (24.24) | 85.2 (23.68) |
| Mean BMI, kg/m2 (SD) | 29.6 (6.92) | 28.8 (7.56) | 29.2 (7.23) |
| Race, n (%) | |||
| White | 60 (95.2) | 61 (96.8) | 121 (96.0) |
| Black or African American | 1 (1.6) | 1 (1.6) | 2 (1.6) |
| Asian | 0 | 1 (1.6) | 1 (0.8) |
| Other | 2 (3.2) | 0 | 2 (1.6) |
| HAE history, n (%) | |||
| C1-INH(SC)-HAE type I | 55 (87.3) | 58 (92.1) | 113 (89.7) |
| C1-INH(SC)-HAE type II | 8 (12.7) | 5 (7.9) | 13 (10.3) |
| Mean (SD) number HAE attacks in 3 months before screeningb | 12.8 (8.42) | 12.7 (10.23) | 12.8 (9.33) |
| Prior use of prophylaxis (preceding 3 months), n (%) | 39 (61.9) | 40 (63.5) | 79 (62.7) |
| Prior prophylaxis medication | |||
| C1-INH(SC) (COMPACT study) | 32 (50.8) | 32 (50.8) | 64 (50.8) |
| C1-INH(IV) | 6 (9.5) | 5 (7.9) | 11 (8.7) |
| Oral prophylaxis (danazol) | 1 (1.6) | 3 (4.8) | 4 (3.2) |
BMI body mass index, HAE hereditary angioedema, SD standard deviation, C1-INH(SC) subcutaneous C1-inhibitor, HRQoL health-related quality of life, C1-INH(IV) intravenous C1-inhibitor
aTen patients were < 18 years old (range: 8–16 years, with three patients < 12 years old) and 10 patients were ≥ 65 years old (range: 65–72 years)
bFor C1-INH(SC)-naïve subjects and C1-INH(SC)-interrupted subjects, “screening” was the first visit of the open-label study; for C1-INH(SC)-continuation subjects, the screening visit was prior to entry into the double-blind COMPACT study