Table 1.
Subject demographics and baseline characteristics
| Arm 1: individualized prophylaxis (n = 118) | Arm 2: weekly prophylaxis (n = 24) | Arm 3: episodic treatment (n = 23) | Total (n = 165) | |
|---|---|---|---|---|
| Age, y, median (min, max) | 29 (12, 65) | 31.5 (18, 59) | 34 (13, 62) | 30 (12, 65) |
| Weight, kg, median (min, max) | 71.65 (42.0, 127.4) | 75.85 (50.0, 105.0) | 70.00 (48.0, 110.4) | 71.60 (42.0, 127.4) |
| Race, n (%) | ||||
| White | 79 (66.9) | 12 (50.0) | 16 (69.6) | 107 (64.8) |
| Black | 7 (5.9) | 1 (4.2) | 2 (8.7) | 10 (6.1) |
| Asian | 27 (22.9) | 11 (45.8) | 5 (21.7) | 43 (26.1) |
| Other | 5 (4.2) | 0 (0) | 0 (0) | 5 (3.0) |
| Geographic location, n (%) | ||||
| Europe | 34 (28.8) | 3 (12.5) | 4 (17.4) | 41 (24.8) |
| North America | 44 (37.3) | 5 (20.8) | 7 (30.4) | 56 (33.9) |
| Other* | 40 (33.9) | 16 (66.7) | 12 (52.2) | 68 (41.2) |
| Genotype, n (%) | ||||
| Intron 22 inversion | 41 (35.0) | 7 (33.3) | 9 (39.1) | 57 (35.4) |
| Frameshift | 24 (20.5) | 4 (19.0) | 6 (26.1) | 34 (21.1) |
| Missense mutation | 22 (18.8) | 4 (19.0) | 1 (4.3) | 27 (16.8) |
| Nonsense mutation | 19 (16.2) | 6 (28.6) | 1 (4.3) | 26 (16.1) |
| Splice site change | 7 (6.0) | 0 (0) | 4 (17.4) | 11 (6.8) |
| Intron 1 inversion | 3 (2.6) | 0 (0) | 1 (4.3) | 4 (2.5) |
| Duplication | 1 (0.9) | 0 (0) | 0 (0) | 1 (0.6) |
| NA | 0 (0) | 0 (0) | 1 (4.3) | 1 (0.6) |
| VWF antigen, IU/dL, median (IQR) | 118.0 (85, 151) | 129.0 (86, 166) | 131.0 (83, 155) | 118.0 (85, 153) |
| Prestudy FVIII regimen, n (%) | ||||
| Prophylaxis | 87 (73.7) | 0 (0) | 0 (0) | 87 (52.7) |
| Episodic | 31 (26.3) | 24 (100) | 23 (100) | 78 (47.3) |
| Estimated bleeding events in prior 12 mo, median (IQR)† | ||||
| Prior prophylaxis | 6.0 (2, 15) | — | — | 6.0 (2, 15) |
| Prior episodic | 27.0 (17, 41) | 29.5 (19, 44) | 24.0 (15, 36) | 27.0 (18, 40) |
| 1 or more target joint, n (%) | ||||
| Prior prophylaxis | 47 (39.8) | — | — | 47 (28.5) |
| Prior episodic | 26 (22.0) | 22 (91.7) | 18 (78.3) | 66 (40.0) |
| HIV-positive, n (%) | 25 (21.2) | 4 (16.7) | 7 (30.4) | 36 (21.8) |
| HCV-positive, n (%) | 55 (46.6) | 14 (58.3) | 13 (56.5) | 82 (49.7) |
—, none; HCV, hepatitis C virus; NA, not applicable.
*
Other included Australia, New Zealand, Brazil, Hong Kong, India, Japan, Russia, and South Africa.
†
Calculation was based on available data.