Efficacy of Wilate prophylaxis: TABR and SABR in all patients and TABR in patient groups by age, VWD type and sex in the modified full analysis set
| Patients . | On-demand (WIL-29) . | Prophylaxis (WIL-31) . | Change (%)∗ . |
|---|---|---|---|
| TABR | |||
| All (n = 33) | |||
| Mean (SD) | 33.4 (23.6) | 5.2 (7.7) | −84.4 |
| Median (range) | 24.5 (11.0-114.5) | 1.9 (0.0-35.8) | −92.2 |
| Age group | |||
| 6 to <12 y (n = 9) | |||
| Mean (SD) | 32.5 (21.4) | 3.7 (4.8) | −88.6 |
| Median (range) | 24.0 (11.0-75.8) | 1.0 (0.0-13.9) | −95.8 |
| 12 to <17 y (n = 6) | |||
| Mean (SD) | 28.9 (21.0) | 4.3 (4.6) | −85.1 |
| Median (range) | 22.5 (12.8-70.1) | 3.4 (0.0-12.8) | −84.9 |
| ≥17 y (n = 18) | |||
| Mean (SD) | 35.3 (26.3) | 6.3 (9.6) | −82.2 |
| Median (range) | 26.6 (14.8-114.5) | 1.5 (0.0-35.8) | −94.4 |
| VWD type | |||
| Severe type 1 (n = 6) | |||
| Mean (SD) | 28.3 (17.8) | 9.1 (14.2) | −67.8 |
| Median (range) | 20.3 (14.8-60.6) | 2.5 (0.0-35.8) | −87.7 |
| Type 2A (n = 5) | |||
| Mean (SD) | 23.3 (4.6) | 0.8 (1.8) | −96.6 |
| Median (range) | 24.0 (16.4-29.1) | 0.0 (0.0-4.0) | −100 |
| Type 3 (n = 22) | |||
| Mean (SD) | 37.1 (26.9) | 5.2 (5.8) | −86.0 |
| Median (range) | 28.3 (11.0-114.5) | 2.5 (0.0-21.1) | −91.2 |
| Sex | |||
| Male (n = 19) | |||
| Mean (SD) | 33.8 (21.2) | 6.0 (9.0) | −82.2 |
| Median (range) | 28.7 (11.0-92.8) | 3.0 (0.0-35.8) | −89.5 |
| Female (n = 14) | |||
| Mean (SD) | 32.8 (27.3) | 4.2 (5.8) | −87.2 |
| Median (range) | 23.9 (14.8-114.5) | 1.0 (0.0-14.9) | −95.8 |
| Treated TABR | |||
| All (n = 33) | |||
| Mean (SD) | 26.1 (22.4) | 3.7 (6.7) | −85.8 |
| Median (range) | 22.4 (3.7-114.5) | 1.0 (0.0-35.8) | −95.5 |
| SABR | |||
| All (n = 33) | |||
| Mean (SD) | 24.4 (20.1) | 3.2 (5.9) | −86.9 |
| Median (range) | 18.7 (4.9-92.8) | 1.0 (0.0-24.6) | −94.7 |
| Treated SABR | |||
| All (n = 33) | |||
| Mean (SD) | 19.0 (16.3) | 2.3 (5.0) | −87.9 |
| Median (range) | 16.4 (1.9-77.0) | 0.0 (0.0-24.6) | −100.0 |
| Patients . | On-demand (WIL-29) . | Prophylaxis (WIL-31) . | Change (%)∗ . |
|---|---|---|---|
| TABR | |||
| All (n = 33) | |||
| Mean (SD) | 33.4 (23.6) | 5.2 (7.7) | −84.4 |
| Median (range) | 24.5 (11.0-114.5) | 1.9 (0.0-35.8) | −92.2 |
| Age group | |||
| 6 to <12 y (n = 9) | |||
| Mean (SD) | 32.5 (21.4) | 3.7 (4.8) | −88.6 |
| Median (range) | 24.0 (11.0-75.8) | 1.0 (0.0-13.9) | −95.8 |
| 12 to <17 y (n = 6) | |||
| Mean (SD) | 28.9 (21.0) | 4.3 (4.6) | −85.1 |
| Median (range) | 22.5 (12.8-70.1) | 3.4 (0.0-12.8) | −84.9 |
| ≥17 y (n = 18) | |||
| Mean (SD) | 35.3 (26.3) | 6.3 (9.6) | −82.2 |
| Median (range) | 26.6 (14.8-114.5) | 1.5 (0.0-35.8) | −94.4 |
| VWD type | |||
| Severe type 1 (n = 6) | |||
| Mean (SD) | 28.3 (17.8) | 9.1 (14.2) | −67.8 |
| Median (range) | 20.3 (14.8-60.6) | 2.5 (0.0-35.8) | −87.7 |
| Type 2A (n = 5) | |||
| Mean (SD) | 23.3 (4.6) | 0.8 (1.8) | −96.6 |
| Median (range) | 24.0 (16.4-29.1) | 0.0 (0.0-4.0) | −100 |
| Type 3 (n = 22) | |||
| Mean (SD) | 37.1 (26.9) | 5.2 (5.8) | −86.0 |
| Median (range) | 28.3 (11.0-114.5) | 2.5 (0.0-21.1) | −91.2 |
| Sex | |||
| Male (n = 19) | |||
| Mean (SD) | 33.8 (21.2) | 6.0 (9.0) | −82.2 |
| Median (range) | 28.7 (11.0-92.8) | 3.0 (0.0-35.8) | −89.5 |
| Female (n = 14) | |||
| Mean (SD) | 32.8 (27.3) | 4.2 (5.8) | −87.2 |
| Median (range) | 23.9 (14.8-114.5) | 1.0 (0.0-14.9) | −95.8 |
| Treated TABR | |||
| All (n = 33) | |||
| Mean (SD) | 26.1 (22.4) | 3.7 (6.7) | −85.8 |
| Median (range) | 22.4 (3.7-114.5) | 1.0 (0.0-35.8) | −95.5 |
| SABR | |||
| All (n = 33) | |||
| Mean (SD) | 24.4 (20.1) | 3.2 (5.9) | −86.9 |
| Median (range) | 18.7 (4.9-92.8) | 1.0 (0.0-24.6) | −94.7 |
| Treated SABR | |||
| All (n = 33) | |||
| Mean (SD) | 19.0 (16.3) | 2.3 (5.0) | −87.9 |
| Median (range) | 16.4 (1.9-77.0) | 0.0 (0.0-24.6) | −100.0 |
Percent reduction from on-demand treatment to prophylaxis.