Demographics and characteristics at baseline for patients in the modified full analysis set
| Characteristic . | VWD subtype . | |||
|---|---|---|---|---|
| Type 1 (N = 6) . | Type 2A (N = 5) . | Type 3 (N = 22) . | All types (N = 33) . | |
| Age at screening, y: median (range) | 16 (9-52) | 16 (7-46) | 19 (7-61) | 18 (7-61) |
| Age at screening, n (%) | ||||
| 6 to <12 y | 2 (33) | 2 (40) | 5 (23) | 9 (27) |
| 12 to <17 y | 1 (17) | 1 (20) | 4 (18) | 6 (18) |
| ≥17 y | 3 (50) | 2 (40) | 13 (59) | 18 (55) |
| Weight at screening, kg: median (range) | 64 (26-94) | 62 (21-104) | 61 (22-112) | 61 (21-112) |
| Height at screening, cm: median (range) | 158 (136-175) | 168 (124-181) | 167 (118-182) | 167 (118-182) |
| Sex | ||||
| Males | 2 (33) | 3 (60) | 14 (64) | 19 (58) |
| Females | 4 (67) | 2 (40) | 8 (36) | 14 (42) |
| Race, N (%) | ||||
| Black or African American | 1 (17) | - | - | 1 (3) |
| Caucasian | 5 (83) | 5 (100) | 22 (100) | 32 (97) |
| Blood type, n (%) | ||||
| A | 4 (67) | 4 (80) | 10 (45) | 18 (55) |
| B | - | 1 (20) | 4 (18) | 5 (15) |
| AB | 1 (17) | - | - | 1 (3) |
| O | 1 (17) | - | 8 (36) | 9 (27) |
| Family history of VWD, n (%) | 4 (67) | 5 (100) | 9 (41) | 18 (55) |
| Baseline factor level, IU/dL, mean (SD) | ||||
| VWF ristocetin cofactor assay | 10.7 (8.0) | 8.4 (2.6) | 5.2 (0.7) | 6.7 (4.0) |
| FVIII activity, 1-stage assay | 29.1 (35.2) | 30.5 (5.5) | 4.9 (5.3) | 13.2 (18.9) |
| FVIII activity, chromogenic assay | 25.2 (30.5) | 25.0 (7.6) | 4.4 (3.2) | 11.3 (16.1) |
| Characteristic . | VWD subtype . | |||
|---|---|---|---|---|
| Type 1 (N = 6) . | Type 2A (N = 5) . | Type 3 (N = 22) . | All types (N = 33) . | |
| Age at screening, y: median (range) | 16 (9-52) | 16 (7-46) | 19 (7-61) | 18 (7-61) |
| Age at screening, n (%) | ||||
| 6 to <12 y | 2 (33) | 2 (40) | 5 (23) | 9 (27) |
| 12 to <17 y | 1 (17) | 1 (20) | 4 (18) | 6 (18) |
| ≥17 y | 3 (50) | 2 (40) | 13 (59) | 18 (55) |
| Weight at screening, kg: median (range) | 64 (26-94) | 62 (21-104) | 61 (22-112) | 61 (21-112) |
| Height at screening, cm: median (range) | 158 (136-175) | 168 (124-181) | 167 (118-182) | 167 (118-182) |
| Sex | ||||
| Males | 2 (33) | 3 (60) | 14 (64) | 19 (58) |
| Females | 4 (67) | 2 (40) | 8 (36) | 14 (42) |
| Race, N (%) | ||||
| Black or African American | 1 (17) | - | - | 1 (3) |
| Caucasian | 5 (83) | 5 (100) | 22 (100) | 32 (97) |
| Blood type, n (%) | ||||
| A | 4 (67) | 4 (80) | 10 (45) | 18 (55) |
| B | - | 1 (20) | 4 (18) | 5 (15) |
| AB | 1 (17) | - | - | 1 (3) |
| O | 1 (17) | - | 8 (36) | 9 (27) |
| Family history of VWD, n (%) | 4 (67) | 5 (100) | 9 (41) | 18 (55) |
| Baseline factor level, IU/dL, mean (SD) | ||||
| VWF ristocetin cofactor assay | 10.7 (8.0) | 8.4 (2.6) | 5.2 (0.7) | 6.7 (4.0) |
| FVIII activity, 1-stage assay | 29.1 (35.2) | 30.5 (5.5) | 4.9 (5.3) | 13.2 (18.9) |
| FVIII activity, chromogenic assay | 25.2 (30.5) | 25.0 (7.6) | 4.4 (3.2) | 11.3 (16.1) |