Table 2.

Coprimary and key secondary efficacy outcomes at day 183

Ravulizumab (N = 125)Eculizumab (N = 121)Statistic for comparisonTreatment effectNoninferiority marginConclusion
Coprimary end points
 Transfusion avoidance rate, % (95% CI)73.6 (65.87, 81.33)66.1 (57.68, 74.55)Difference in rate6.8 (−4.66, 18.14)−20%Noninferior
 LDH normalization, % (95% CI)53.6 (45.9, 61.2)49.4 (41.7, 57.0)OR1.19 (0.80, 1.77)0.39Noninferior
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 baseline0.67 (−1.21, 2.55)−5.0Noninferior
 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 rate2.9 (−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.