ABRs (second 6-mo study period; FAS and PPAS)
| . | FAS (N = 115) . | PPAS (N = 95) . | ||
|---|---|---|---|---|
| FVIII trough level 1% to 3% (n = 57) . | FVIII trough level 8% to 12% (n = 53)* . | FVIII trough level 1% to 3% (n = 52) . | FVIII trough level 8% to 12% (n = 43) . | |
| Total ABR | ||||
| Mean (SD) | 3.6 (7.5) | 1.6 (3.4) | 2.8 (3.0) | 1.2 (2.4) |
| Median (Q1 to Q3) | 2.0 (0.0-4.0) | 0.0 (0.0-2.0) | 2.0 (0.0-4.0) | 0.0 (0.0-2.0) |
| Spontaneous ABR | ||||
| Mean (SD) | 2.5 (6.6) | 0.7 (1.7) | 1.7 (2.5) | 0.6 (1.5) |
| Median (Q1 to Q3) | 0.0 (0.0-4.0) | 0.0 (0.0-0.0) | 0.0 (0.0-4.0) | 0.0 (0.0-0.0) |
| Spontaneous joint ABR | ||||
| Mean (SD) | 2.0 (6.4) | 0.5 (1.7) | 1.2 (2.0) | 0.4 (1.4) |
| Median (Q1 to Q3) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) |
| Joint ABR | ||||
| Mean (SD) | 2.6 (7.4) | 1.1 (2.6) | 1.8 (2.2) | 0.8 (2.3) |
| Median (Q1 to Q3) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) | 1.0 (0.0-3.0) | 0.0 (0.0-0.0) |
| ABR of joints with ≥4 spontaneous bleeds in 6 consecutive months | ||||
| Mean (SD) | 1.0 (6.8) | 0.4 (1.5) | 0.1 (0.6) | 0.2 (1.3) |
| Median (Q1 to Q3) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) |
| Injury-related ABR | ||||
| Mean (SD) | 1.1 (2.0) | 0.9 (2.6) | 1.1 (1.9) | 0.7 (1.7) |
| Median (Q1 to Q3) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) |
| . | FAS (N = 115) . | PPAS (N = 95) . | ||
|---|---|---|---|---|
| FVIII trough level 1% to 3% (n = 57) . | FVIII trough level 8% to 12% (n = 53)* . | FVIII trough level 1% to 3% (n = 52) . | FVIII trough level 8% to 12% (n = 43) . | |
| Total ABR | ||||
| Mean (SD) | 3.6 (7.5) | 1.6 (3.4) | 2.8 (3.0) | 1.2 (2.4) |
| Median (Q1 to Q3) | 2.0 (0.0-4.0) | 0.0 (0.0-2.0) | 2.0 (0.0-4.0) | 0.0 (0.0-2.0) |
| Spontaneous ABR | ||||
| Mean (SD) | 2.5 (6.6) | 0.7 (1.7) | 1.7 (2.5) | 0.6 (1.5) |
| Median (Q1 to Q3) | 0.0 (0.0-4.0) | 0.0 (0.0-0.0) | 0.0 (0.0-4.0) | 0.0 (0.0-0.0) |
| Spontaneous joint ABR | ||||
| Mean (SD) | 2.0 (6.4) | 0.5 (1.7) | 1.2 (2.0) | 0.4 (1.4) |
| Median (Q1 to Q3) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) |
| Joint ABR | ||||
| Mean (SD) | 2.6 (7.4) | 1.1 (2.6) | 1.8 (2.2) | 0.8 (2.3) |
| Median (Q1 to Q3) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) | 1.0 (0.0-3.0) | 0.0 (0.0-0.0) |
| ABR of joints with ≥4 spontaneous bleeds in 6 consecutive months | ||||
| Mean (SD) | 1.0 (6.8) | 0.4 (1.5) | 0.1 (0.6) | 0.2 (1.3) |
| Median (Q1 to Q3) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) | 0.0 (0.0-0.0) |
| Injury-related ABR | ||||
| Mean (SD) | 1.1 (2.0) | 0.9 (2.6) | 1.1 (1.9) | 0.7 (1.7) |
| Median (Q1 to Q3) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) | 0.0 (0.0-2.0) | 0.0 (0.0-0.0) |
ABR based on 53 PwHA; data not available from 5 randomized PwHA who discontinued the study before the second 6-mo period.