Table 2.
Study participant baseline characteristics
| Variable | Safety population N = 22 | Efficacy population (on-demand) arm 2, N = 21 | Efficacy population (prophylaxis) arm 1, N = 1 | Efficacy population (prophylaxis) arm 3, N = 8 | PK population N = 15 |
| Sex, n (%) | |||||
| Male | 10 (45) | 10 (48) | 0 | 6 (75) | 7 (47) |
| Female | 12 (55) | 11 (52) | 1 (100) | 2 (25) | 8 (53) |
| Ethnic origin, n (%) | |||||
| Caucasian | 22 (100) | 21 (100) | 1 (100) | 8 (100) | 15 (100) |
| Age (years) | |||||
| Mean (SD) | 33.6 (15.2) | 33.0 (15.3) | 46.0 (−) | 43.0 (15.9) | 32.5 (11.3) |
| Median (range) | 30.5 (15–68) | 28.0 (15–68) | 46.0 (46–46) | 42.5 (21–68) | 33.0 (15–50) |
| <18 years, n (%) | 3 (14) | 3 (14) | 0 | 0 | 1 (7) |
| ≥18 years, n (%) | 19 (86) | 18 (86) | 1 (100) | 8 (100) | 14 (93) |
| Weight (kg) | |||||
| Mean (SD) | 72.6 (15.0) | 72.5 (15.4) | 75.6 (−) | 81.3 (14.0)a | 74.9 (14.2) |
| Median (range) | 71.0 (51.5–98.5) | 70.0 (51.5–98.5) | 75.6 (75.6–75.6) | 82.5 (61.5–98.5)a | 73.0 (58.0–99.0) |
| Height (cm) | |||||
| Mean (SD) | 168.7 (7.9) | 168.8 (8.1) | 167.0 (−) | 172.3 (7.0) | 169.4 (8.4) |
| Median (range) | 168.0 (155–182) | 168.0 (155–182) | 167.0 (167–167) | 174.0 (162–180) | 168.0 (155–182) |
| VWD, n (%) | |||||
| Type 1 | 5 (23) | 4 (19) | 1 (100) | 1 (13) | 4 (27) |
| Type 2A | 4 (18) | 4 (19) | 0 | 1 (13) | 3 (20) |
| Type 3 | 13 (59) | 13 (62) | 0 | 6 (75) | 8 (53) |
| Years since first diagnosis | |||||
| Mean (SD) | 22.9 (14.8) | 22.7 (15.1) | 2.7 (−) | 32.4 (19.0) | 21.7 (9.1) |
| Median (range) | 17.5 (3–67) | 17.8 (8–67) | 2.7 (2.7–2.7) | 28.4 (10–67) | 20.1 (3–36) |
| Bleeding events per study participant in the past 12 months | |||||
| Mean (SD) | 70.3 (227.6) | 73.6 (232.7) | 1.0 (−) | 27.4 (33.5) | − |
| Median (range) | 10.5 (1–1075) | 12.0 (1–1075) | 1.0 (1.0–1.0) | 13.5 (1–96.0) | − |
n, number of study participants with characteristic; N, total number of study participants; PK, pharmacokinetic; VWD, von Willebrand disease. Among other bleedings, one study participant experienced ulorrhagia after each meal, that is, 2–3 times per day; such bleedings also occurred during the study, but were not reported as they were considered too minor.
aBody weight measured at month 12 (start of prophylactic treatment for arm 3 study participants).