Coprimary and key secondary efficacy outcomes at day 183
| . | Ravulizumab (N = 125) . | Eculizumab (N = 121) . | Statistic for comparison . | Treatment effect . | Noninferiority margin . | Conclusion . |
|---|---|---|---|---|---|---|
| Coprimary end points | ||||||
| Transfusion avoidance rate, % (95% CI) | 73.6 (65.87, 81.33) | 66.1 (57.68, 74.55) | Difference in rate | 6.8 (−4.66, 18.14) | −20% | Noninferior |
| LDH normalization, % (95% CI) | 53.6 (45.9, 61.2) | 49.4 (41.7, 57.0) | OR | 1.19 (0.80, 1.77) | 0.39 | Noninferior |
| Key secondary efficacy end points | ||||||
| LDH, least squares mean % change (95% CI) | −76.84 (−79.96, −73.73) | −76.02 (−79.20, −72.83) | Difference in % change from baseline | −0.83 (−5.21, 3.56) | 20% | Noninferior |
| FACIT-Fatigue score, least squares mean change (95% CI) | 7.07 (5.55, 8.60) | 6.40 (4.85, 7.96) | Difference in change from baseline | 0.67 (−1.21, 2.55) | −5.0 | Noninferior |
| Breakthrough hemolysis rate, % (95% CI) | 4.0 (0.56, 7.44) | 10.7 (5.23, 16.26) | Difference in rate | −6.7 (−14.21, 0.18) | 20% | Noninferior |
| Hemoglobin stabilization rate, % (95% CI) | 68.0 (59.82, 76.18) | 64.5 (55.93, 72.99) | Difference in rate | 2.9 (−8.80, 14.64) | −20% | Noninferior |
| . | Ravulizumab (N = 125) . | Eculizumab (N = 121) . | Statistic for comparison . | Treatment effect . | Noninferiority margin . | Conclusion . |
|---|---|---|---|---|---|---|
| Coprimary end points | ||||||
| Transfusion avoidance rate, % (95% CI) | 73.6 (65.87, 81.33) | 66.1 (57.68, 74.55) | Difference in rate | 6.8 (−4.66, 18.14) | −20% | Noninferior |
| LDH normalization, % (95% CI) | 53.6 (45.9, 61.2) | 49.4 (41.7, 57.0) | OR | 1.19 (0.80, 1.77) | 0.39 | Noninferior |
| Key secondary efficacy end points | ||||||
| LDH, least squares mean % change (95% CI) | −76.84 (−79.96, −73.73) | −76.02 (−79.20, −72.83) | Difference in % change from baseline | −0.83 (−5.21, 3.56) | 20% | Noninferior |
| FACIT-Fatigue score, least squares mean change (95% CI) | 7.07 (5.55, 8.60) | 6.40 (4.85, 7.96) | Difference in change from baseline | 0.67 (−1.21, 2.55) | −5.0 | Noninferior |
| Breakthrough hemolysis rate, % (95% CI) | 4.0 (0.56, 7.44) | 10.7 (5.23, 16.26) | Difference in rate | −6.7 (−14.21, 0.18) | 20% | Noninferior |
| Hemoglobin stabilization rate, % (95% CI) | 68.0 (59.82, 76.18) | 64.5 (55.93, 72.99) | Difference in rate | 2.9 (−8.80, 14.64) | −20% | Noninferior |
For the transfusion avoidance end point, treatment differences (95% CIs) are based on estimated differences in percent with 95% CI. For the LDH-N end point, the adjusted prevalence within each treatment is displayed. Testing of the noninferiority hypothesis is assessed by comparing the bolded limit of the 95% CI to the noninferiority margin.