Primary efficacy analysis: comparison of on-study sABRs with historical estimates
| . | Prior on-demand group (n = 13) . | Switch group (n = 10) . |
|---|---|---|
| Historical | ||
| No. of treated spontaneous BEs | 201 | 50 |
| sABR mean (95% CI)* | 6.54 (2.52 to 17.00) | 0.51 (0.04 to 6.31) |
| rVWF prophylaxis (on-study treatment) | ||
| No. of treated spontaneous BEs | 9 | 18 |
| sABR mean (95% CI)* | 0.56 (0.15 to 2.05) | 0.28 (0.02 to 3.85) |
| Comparison (rVWF prophylaxis vs historical sABR) | ||
| sABR rVWF prophylaxis:historical ratio (95% CI) | 0.085 (0.021 to 0.346) | 0.550 (0.086 to 3.523) |
| sABR percentage change from historical (95% CI)† | −91.5% (−97.9% to −65.4%) | −45.0% (−91.4% to 252.3%) |
| . | Prior on-demand group (n = 13) . | Switch group (n = 10) . |
|---|---|---|
| Historical | ||
| No. of treated spontaneous BEs | 201 | 50 |
| sABR mean (95% CI)* | 6.54 (2.52 to 17.00) | 0.51 (0.04 to 6.31) |
| rVWF prophylaxis (on-study treatment) | ||
| No. of treated spontaneous BEs | 9 | 18 |
| sABR mean (95% CI)* | 0.56 (0.15 to 2.05) | 0.28 (0.02 to 3.85) |
| Comparison (rVWF prophylaxis vs historical sABR) | ||
| sABR rVWF prophylaxis:historical ratio (95% CI) | 0.085 (0.021 to 0.346) | 0.550 (0.086 to 3.523) |
| sABR percentage change from historical (95% CI)† | −91.5% (−97.9% to −65.4%) | −45.0% (−91.4% to 252.3%) |
Estimated using a generalized linear mixed-effects model for the full analysis set through month 12. Only BEs treated with VWF infusions are included. Six BEs of unknown cause (4 historical [all in the prior on-demand group] and 2 on-study [switch group]) were counted as spontaneous BEs for this analysis.
Percentage change in sABR was calculated directly from the sABR ratio (RR): 100 × (RR - 1).