Proportion of PwHA who had zero bleeds (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 = 58) . | P* . | FVIII trough level 1% to 3% (n = 52) . | FVIII trough level 8% to 12% (n = 43) . | P* . | |
| Zero total bleeds | ||||||
| Point estimate (95% CI) of proportion of PwHA, % | 42 (29-55) | 62 (49-75) | .055 | 40 (27-55) | 67 (52-81) | .015 |
| Zero spontaneous bleeds | ||||||
| Point estimate (95% CI) of proportion of PwHA, % | 60 (47-72) | 76 (65-88) | .101 | 60 (45-73) | 81 (67-92) | .038 |
| Zero spontaneous joint bleeds | ||||||
| Point estimate (95% CI) of proportion of PwHA, % | 65 (53-77) | 85 (75-95) | .026 | 65 (51-78) | 91 (78-97) | .008 |
| . | FAS (N = 115) . | PPAS (N = 95) . | ||||
|---|---|---|---|---|---|---|
| FVIII trough level 1% to 3% (n = 57) . | FVIII trough level 8% to 12% (n = 58) . | P* . | FVIII trough level 1% to 3% (n = 52) . | FVIII trough level 8% to 12% (n = 43) . | P* . | |
| Zero total bleeds | ||||||
| Point estimate (95% CI) of proportion of PwHA, % | 42 (29-55) | 62 (49-75) | .055 | 40 (27-55) | 67 (52-81) | .015 |
| Zero spontaneous bleeds | ||||||
| Point estimate (95% CI) of proportion of PwHA, % | 60 (47-72) | 76 (65-88) | .101 | 60 (45-73) | 81 (67-92) | .038 |
| Zero spontaneous joint bleeds | ||||||
| Point estimate (95% CI) of proportion of PwHA, % | 65 (53-77) | 85 (75-95) | .026 | 65 (51-78) | 91 (78-97) | .008 |
P < .05 between treatment arms was considered statistically significant.
Zero bleeds rate was based on intent-to-treat principle.
The null hypothesis of independence was tested against a 2-sided alternative by χ2 test with continuity correction.