Table 1. Baseline demographic and clinical characteristics.
| Parameter | Prophylaxis ( N = 25) | On-demand ( N = 29) | Surgery ( N = 62) | All ( N = 111) |
|---|---|---|---|---|
| Age at study entry [y], median (range) | 14.0 (1.0–83.0) | 20.0 (0–76.0) | 35.5 (2.0–77.5) | 27.0 (0.8–83.2) |
| Age at diagnosis of VWD [y], median (range) | 7.5 (0–60.9) | 15.0 (0–59.0) a | 20.6 (0.4–71.4) b | 13.5 (0–71.4) c |
| Time since diagnosis of VWD [y], median (range) | 5.0 (0.2–40.0) | 2.1 (0.1–60.0) a | 7.0 (0–60.0) b | 5.0 (−0.6–60.0) c |
| Sex, n (%) | ||||
| Male | 7 (28%) | 9 (31%) | 19 (31%) | 35 (32%) |
| Female | 18 (72%) | 20 (69%) | 43 (69%) | 76 (68%) |
| Type of VWD, n (%) | ||||
| 1 | 3 (12%) | 12 (41%) | 35 (56%) | 50 (45%) |
| 2 | 8 (32%) | 12 (41%) | 18 (29%) | 32 (29%) |
| 2, not specified | 1 | 3 | 4 | 5 |
| 2A | 5 | 3 | 6 | 14 |
| 2B | 1 | 3 | 4 | 5 |
| 2M | 0 | 1 | 2 | 3 |
| 2N | 1 | 2 | 2 | 5 |
| 3 | 14 (56%) | 5 (17%) | 8 (13%) | 20 (18%) |
| Not available | 0 | 0 | 1 (2%) | 8 (7%) |
| Not applicable d | 0 | 0 | 0 | 1 (1%) |
| Pre-study exposure to FVIII/VWF products by EDs, n (%) | ||||
| 0 | 3 (12%) | 6 (21%) | 30 (48%) | 37 (33%) |
| < 150 EDs | 14 (56%) | 21 (72%) | 27 (44%) | 57 (51%) |
| ≥150 EDs | 8 (32%) | 2 (7%) | 3 (5%) | 14 (13%) |
| Not available | 0 | 0 | 2 (3%) | 3 (3%) |
Abbreviations: ED, exposure day; FVIII, factor VIII; VWD, von Willebrand disease; VWF, von Willebrand factor.
a
N = 28.
b
N = 58.
c
N = 107.
d
One patient was diagnosed during the study as having hemophilia A rather than VWD.