Primary and key secondary efficacy outcomes at day 183
| . | Ravulizumab (n = 97) . | Eculizumab (n = 98) . | Statistic for comparison . | Treatment effect . | Noninferiority margin . | Conclusion* . |
|---|---|---|---|---|---|---|
| Primary end point | ||||||
| LDH, least squares mean % change (95% CI) | −0.82 (−7.8, 6.1) | 8.4 (1.5, 15.3) | Difference in percentage change from baseline | 9.2 (−0.42 to 18.8) | −15% | Noninferior |
| Key secondary efficacy end points | ||||||
| Breakthrough hemolysis rate, % (95% CI) | 0 (0 to 3.7) | 5.1 (1.7 to 11.5) | Difference in rate | 5.1 (−8.9 to 19.0) | −20% | Noninferior |
| FACIT-Fatigue score, least squares mean change (95% CI) | 2.0 (0.6 to 3.4) | 0.54 (−0.8 to 1.9) | Difference in change from baseline | 1.5 (−0.2 to 3.2) | −3.0 | Noninferior |
| Transfusion avoidance rate, % (95% CI) | 87.6 (81.1 to 94.2) | 82.7 (75.2 to 90.2) | Difference in rate | 5.5 (−4.3 to 15.7) | −20% | Noninferior |
| Stabilized hemoglobin rate, % (95% CI) | 76.3 (67.8 to 84.8) | 75.5 (67.0 to 84.0) | Difference in rate | 1.4 (−10.4 to 13.3) | −20% | Noninferior |
| . | Ravulizumab (n = 97) . | Eculizumab (n = 98) . | Statistic for comparison . | Treatment effect . | Noninferiority margin . | Conclusion* . |
|---|---|---|---|---|---|---|
| Primary end point | ||||||
| LDH, least squares mean % change (95% CI) | −0.82 (−7.8, 6.1) | 8.4 (1.5, 15.3) | Difference in percentage change from baseline | 9.2 (−0.42 to 18.8) | −15% | Noninferior |
| Key secondary efficacy end points | ||||||
| Breakthrough hemolysis rate, % (95% CI) | 0 (0 to 3.7) | 5.1 (1.7 to 11.5) | Difference in rate | 5.1 (−8.9 to 19.0) | −20% | Noninferior |
| FACIT-Fatigue score, least squares mean change (95% CI) | 2.0 (0.6 to 3.4) | 0.54 (−0.8 to 1.9) | Difference in change from baseline | 1.5 (−0.2 to 3.2) | −3.0 | Noninferior |
| Transfusion avoidance rate, % (95% CI) | 87.6 (81.1 to 94.2) | 82.7 (75.2 to 90.2) | Difference in rate | 5.5 (−4.3 to 15.7) | −20% | Noninferior |
| Stabilized hemoglobin rate, % (95% CI) | 76.3 (67.8 to 84.8) | 75.5 (67.0 to 84.0) | Difference in rate | 1.4 (−10.4 to 13.3) | −20% | Noninferior |
Testing of the noninferiority hypothesis is assessed by comparing the bolded limit of the 95% CI to the noninferiority margin.
A conclusion of noninferiority indicates that the noninferiority margin is larger or smaller than the lower or upper bound of the 95% CI indicated in boldface.