Table 2.

Primary and key secondary efficacy outcomes at day 183

Ravulizumab (n = 97)Eculizumab (n = 98)Statistic for comparisonTreatment effectNoninferiority marginConclusion*
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 baseline9.2 (−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 rate5.1 (−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 baseline1.5 (−0.2 to 3.2)−3.0Noninferior
 Transfusion avoidance rate, % (95% CI)87.6 (81.1 to 94.2)82.7 (75.2 to 90.2)Difference in rate5.5 (−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 rate1.4 (−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.