Table 1.
Patient demographics and baseline characteristics
| FAS (N = 115) | PPAS (N = 95) | |||
|---|---|---|---|---|
| FVIII trough level 1% to 3% (n = 57) | FVIII trough level 8% to 12% (n = 58) | FVIII trough level 1% to 3% (n = 52) | FVIII trough level 8% to 12% (n = 43) | |
| Age, y | ||||
| Mean (SD) | 31.1 (13.8) | 31.2 (12.2) | 31.0 (13.6) | 31.6 (12.9) |
| Median (range) | 29.0 (12-61) | 30.5 (13-61) | 28.5 (12-61) | 31.0 (13-61) |
| Sex, n (%) | ||||
| Male | 57 (100) | 58 (100) | 52 (100) | 43 (100) |
| Race, n (%) | ||||
| White | 40 (70.2) | 36 (62.1) | 36 (69.2) | 25 (58.1) |
| Asian | 14 (24.6) | 18 (31.0) | 13 (25.0) | 15 (34.9) |
| Other | 3 (5.3) | 4 (6.9) | 3 (5.8) | 3 (7.0) |
| Previous use of FVIII replacement therapy, n (%) | ||||
| Prophylaxis | 43 (75.4) | 42 (72.4) | 39 (75.0) | 30 (69.8) |
| On-demand | 14 (24.6) | 16 (27.6) | 13 (25.0) | 13 (30.2) |
| Number of joints with ≥4 spontaneous bleeds within 6 mo before screening, n (%) | ||||
| 0 | 17 (29.8) | 14 (24.1) | 16 (30.8) | 10 (23.3) |
| 1 | 15 (26.3) | 17 (29.3) | 14 (26.9) | 15 (34.9) |
| 2 | 15 (26.3) | 13 (22.4) | 12 (23.1) | 7 (16.3) |
| 3 | 5 (8.8) | 4 (6.9) | 5 (9.6) | 2 (4.7) |
| ≥4 | 5 (8.8) | 10 (17.2) | 5 (9.6) | 9 (20.9) |
| Hemophilic arthropathy present at screening, n (%)* | 7 (12.3) | 15 (25.9) | 7 (13.5) | 12 (27.9) |
| HCV antibody present, n (%)† | 30 (52.6) | 26 (44.8) | 27 (51.9) | 21 (48.8) |
| HIV-1/2 antibody present, n (%) | 2 (3.5) | 6 (10.3) | 2 (3.8) | 4 (9.3) |
| ABR (all bleeds) within 12 mo before enrollment ‡ | ||||
| Mean (SD) | 13.3 (15.9) | 13.3 (17.4) | 11.8 (14.0) | 14.3 (18.5) |
| Median (Q1 to Q3) | 6.0 (3.0-14.0) | 5.0 (2.0-16.0) | 6.0 (3.0-13.0) | 7.0 (2.0-20.0) |
*
Status based on the patient’s medical history (not a determination made by the investigator on the basis of their clinical evaluation of the patient). No other testing was done in this protocol to establish arthropathy.
†
HCV antibody-positive patients underwent polymerase chain reaction testing to confirm that no active infection was present.
‡
For newly recruited PwHA, ABRs were assessed on the basis of documented and treated bleeds within 12 mo before enrollment, using patients’ medical records.