Table 1 Baseline characteristics of the study participants (N = 13)
Patient characteristics | Statistical values |
|---|---|
No. of participants, N | 13 |
Age in years, mean (SD) | 20.1 (6.8) |
Natural baseline FVIII:C levels, n (%) | |
• < 1 IU/dL | 8 (61.5) |
• 1–3 IU/dL | 5 (38.5) |
ABR while using low-dose PK-guided EHL FVIII prophylaxis before study inclusion, bleeds/year | |
• Mean (SD) | 9.5 (7.1) |
• Median (IQR) | 10 (3–14) |
AJBR while using low-dose PK-guided EHL FVIII prophylaxis before study inclusion, joint bleeds/year | |
• Mean (SD) | 6.7 (7.3) |
• Median (IQR) | 4 (0–12) |
HJHS while using low-dose PK-guided EHL FVIII prophylaxis before study inclusion, marks | |
• Mean (SD) | 16.5 (12.3) |
• Median (IQR) | 14 (10–24) |
No. of target joint(s), n (%) | |
• None | 3 (23.1) |
• 1 joint | 1 (7.7) |
• 2 joints | 2 (15.4) |
• 3 joints | 7 (53.8) |
Annualized FVIII consumption while using low-dose PK-guided EHL FVIII prophylaxis before study inclusion, IU/kg/year | |
• Mean (SD) | 1581.5 (430.6) |
• Median (IQR) | 1724.8 (1235.4–1778.5) |