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) |
| . | 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.