Patient characteristics | All Kovaltry (n = 29) | Prophylactic Kovaltry (n = 22) | Prophylactic EHL (n = 60) |
---|---|---|---|
Age group n (%) | |||
0–5 years | 5 (17.2) | 3 (13.6) | 3 (5.0) |
6–11 years | 15 (51.7) | 11 (50.0) | 15 (25.0) |
12–17 years | 9 (31.0) | 8 (36.4) | 42 (70.0) |
Body mass index groups | |||
Underweight < 18.5 | 10 (34.5) | 8 (36.4) | 9 (15.0) |
Normal 18.5–24.9 | 9 (31.0) | 8 (36.4) | 40 (66.7) |
Overweight 25–29.9 | 5 (17.2) | 2 (9.1) | 6 (10.0) |
Obese 30+ | 4 (13.8) | 3 (13.6) | 4 (6.7) |
Missing | 1 (3.4) | 1 (4.5) | 1 (1.7) |
Severity | |||
Moderate (1–5 IU/dL) | 2 (6.9) | 2 (9.1) | 12 (20.0) |
Severe (< 1 IU/dL) | 27 (93.1) | 20 (90.9) | 48 (80.0) |
History of inhibitors | |||
0 Never | 23 (79.3) | 17 (77.3) | 46 (76.7) |
1 Once | 6 (20.7) | 5 (22.7) | 10 (16.7) |
More than once—recurrence/relapse | 0 (0) | 0 (0) | 4 (6.70) |
Treatment regimen at initiation | |||
Continuous prophylaxis | 22 (75.9) | 17 (77.3) | 24 (40.0) |
Episodic—on demand—treatment | 6 (20.7) | 3 (13.6) | 32 (53.3) |
Intermittent—periodic—prophylaxis | 1 (3.4) | 2 (9.1) | 4 (6.7) |
Target joints | |||
No | 26 (89.7) | 20 (90.9) | 49 (81.7) |
Yes | 3 (10.3) | 2 (9.1) | 11 (18.3) |
Chronically damaged joints | |||
No | 26 (89.1) | 21 (95.5) | 47 (78.3) |
Yes | 3 (10.3) | 1 (4.5) | 13 (21.7) |
Treatment adherence | |||
Fully adherent: missing < 15% of infusions | 25 (86.2) | 18 (81.8) | 51 (85.0) |
Sub-optimally adherent: missing 15–25% of infusions | 3 (10.3) | 3 (13.6) | 7 (11.7) |
Non-adherent: missing > 25% of infusions | 0 | 0 | 0 (0) |
Not applicable | 1 (3.4) | 1 (4.5) | 2 (3.30) |