Results of primary and key secondary efficacy end points. The model-based ABR during the (A-B) OP and ATP for the primary end point (treated bleeds) and (C-D) key secondary end points in the OD group (A,C) and the RP group (B,D). Ratio estimate, 95% CI and P value are shown for marstacimab vs previous OD therapy; difference estimate and 95% CI are shown for marstacimab vs previous RP (P values not shown because comparisons did not reach significance). The analyses include participants who received ≥1 dose of marstacimab prophylaxis in the ATP. ABR was calculated for time on treatment in each phase. For participants who had a dose escalation, the duration on marstacimab 300 mg was not included. For the OD group, P values are for the null hypothesis that the ABR ratio (marstacimab prophylaxis to OD treatment) equals 0.5. For the RP group, P values are for the null hypothesis that the difference (marstacimab prophylaxis minus RP) equals 0.0. Hierarchical testing precluded superiority testing for the secondary end points in the RP group (panel B) because superiority was not demonstrated in the physical health domain of the Haem-A-QoL.
Figure 2.

Results of primary and key secondary efficacy end points. The model-based ABR during the (A-B) OP and ATP for the primary end point (treated bleeds) and (C-D) key secondary end points in the OD group (A,C) and the RP group (B,D). Ratio estimate, 95% CI and P value are shown for marstacimab vs previous OD therapy; difference estimate and 95% CI are shown for marstacimab vs previous RP (P values not shown because comparisons did not reach significance). The analyses include participants who received ≥1 dose of marstacimab prophylaxis in the ATP. ABR was calculated for time on treatment in each phase. For participants who had a dose escalation, the duration on marstacimab 300 mg was not included. For the OD group, P values are for the null hypothesis that the ABR ratio (marstacimab prophylaxis to OD treatment) equals 0.5. For the RP group, P values are for the null hypothesis that the difference (marstacimab prophylaxis minus RP) equals 0.0. Hierarchical testing precluded superiority testing for the secondary end points in the RP group (panel B) because superiority was not demonstrated in the physical health domain of the Haem-A-QoL.

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