Table 1.

Demographic and baseline clinical characteristics

CharacteristicRavulizumab (n = 97)Eculizumab (n = 98)Total (N = 195)
Sex, no. (%)
 Male50 (51.5)48 (49.0)98 (50.3)
 Female47 (48.5)50 (51.0)97 (49.7)
Age at first infusion of study drug, mean (SD), y46.6 (14.4)48.8 (14.0)47.7 (14.2)
Race, no. (%)
 White50 (51.5)61 (62.2)111 (56.9)
 Asian23 (23.7)19 (19.4)42 (21.5)
 Japanese5 (5.2)7 (7.1)12 (6.2)
 African American5 (5.2)3 (3.1)8 (4.1)
 Other/multiple3 (3.1)1 (1.0)4 (2.1)
 Not reported/unknown16 (16.5)14 (14.3)30 (15.4)
Weight, mean (SD), kg72.4 (16.8)73.4 (14.6)72.9 (15.7)
Height, mean (SD), cm168.3 (10.1)168.8 (9.9)168.5 (10.0)
Years on eculizumab before first study infusion6.0 (3.5)5.6 (3.5)5.8 (3.5)
Patients with packed red blood cells/whole blood transfusions received within 1 y before first dose, no. (%)13 (13.4)12 (12.2)25 (12.8)
Age at PNH diagnosis, mean (SD), y34.1 (14.4)36.8 (14.1)35.5 (14.3)
Time from PNH diagnosis to consent, mean (SD), y12.4 (8.4)11.9 (9.4)12.2 (8.9)
LDH, mean (SD),* U/L228.0 (48.7)235.2 (49.7)231.6 (49.2)
PNH clone size, mean (SD), %
 Type II red blood cells14.9 (19.6)16.3 (23.6)15.6 (21.6)
 Type III red blood cells44.6 (30.5)43.5 (29.7)44.0 (30.0)
 Total red blood cells60.6 (32.5)59.5 (31.4)60.1 (31.9)
 Granulocyte82.6 (23.6)84.0 (21.4)83.3 (22.5)
 Monocyte85.6 (20.5)86.1 (19.7)85.9 (20.0)
Hemoglobin, g/L, mean (SD)110.8 (18.4)109.1 (18.4)Not available
Haptoglobin, g/L, mean (SD)§0.283 (0.235)0.255 (0.174)Not available
History of major adverse vascular events, no. (%)28 (28.9)22 (22.4)50 (25.6)
History of aplastic anemia, no. (%)34 (35.1)39 (39.8)73 (37.4)
  • * Normal range, 120 to 246 U/L.

  • Erythrocytes with complete deficiency in glycosylphosphatidylinositol-anchored proteins, including complement regulatory proteins CD59 and CD55.30

  • Normal range, 11.5 to 16.0 g/dL (women) and 13.0 to 17.5 g/dL (men).

  • § Normal range, 0.4 to 2.4 g/dL.