Summary of demographic characteristics
| Dose group* . | VWD type† . | N . | Gender, F/M . | Median age, y (range) . | Median weight, kg (range) . |
|---|---|---|---|---|---|
| 50 IU (PK crossover) | Type 3 | 22‡ | 11/11 | 33 (18-60) | 74.8 (43.8-154.4) |
| Severe type 1 | 3 | 2/1 | 25 (19-47) | 82.1 (70.4-130.4) | |
| 20 IU | Type 3 | 5 | 0/5 | 34 (19-55) | 79 (64-131.4) |
| 7.5 IU | Type 3 | 5 | 2/3 | 26 (21-39) | 86.2 (52-135.4) |
| 2 IU | Type 3 | 3 | 1/2 | 38 (26-44) | 130 (101.5-144.8) |
| Total | 32 | 15/17 | 33 (18-60) | 81.1 (43.8-144.8) | |
| Dose group* . | VWD type† . | N . | Gender, F/M . | Median age, y (range) . | Median weight, kg (range) . |
|---|---|---|---|---|---|
| 50 IU (PK crossover) | Type 3 | 22‡ | 11/11 | 33 (18-60) | 74.8 (43.8-154.4) |
| Severe type 1 | 3 | 2/1 | 25 (19-47) | 82.1 (70.4-130.4) | |
| 20 IU | Type 3 | 5 | 0/5 | 34 (19-55) | 79 (64-131.4) |
| 7.5 IU | Type 3 | 5 | 2/3 | 26 (21-39) | 86.2 (52-135.4) |
| 2 IU | Type 3 | 3 | 1/2 | 38 (26-44) | 130 (101.5-144.8) |
| Total | 32 | 15/17 | 33 (18-60) | 81.1 (43.8-144.8) | |
Subjects in the 50 IU PK crossover group received 50 IU/kg VWF:RCo + 38.5 IU/kg rFVIII and 21-25 IU/kg pdFVIII in random order with an 8-day washout period. Subjects in the 20 IU group received 20 IU/kg VWF:RCo + 15.4 IU/kg rFVIII; those in the 7.5 IU received 7.5 IU/kg VWF:RCo + 5.8 IU/kg FVIII; and those in 2 IU received 2 IU/kg VWF:RCo + 1.5 IU/kg FVIII.
Type 3 VWD: ≤3 IU/dL VWF:Ag; severe type 1 VWD: <10% VWF:RCo; and <20% FVIII.
Six subjects in the PK crossover dose group were also enrolled once in a lower-dose group.