Demographic and baseline clinical characteristics
| Characteristic . | Ravulizumab (n = 97) . | Eculizumab (n = 98) . | Total (N = 195) . |
|---|---|---|---|
| Sex, no. (%) | |||
| Male | 50 (51.5) | 48 (49.0) | 98 (50.3) |
| Female | 47 (48.5) | 50 (51.0) | 97 (49.7) |
| Age at first infusion of study drug, mean (SD), y | 46.6 (14.4) | 48.8 (14.0) | 47.7 (14.2) |
| Race, no. (%) | |||
| White | 50 (51.5) | 61 (62.2) | 111 (56.9) |
| Asian | 23 (23.7) | 19 (19.4) | 42 (21.5) |
| Japanese | 5 (5.2) | 7 (7.1) | 12 (6.2) |
| African American | 5 (5.2) | 3 (3.1) | 8 (4.1) |
| Other/multiple | 3 (3.1) | 1 (1.0) | 4 (2.1) |
| Not reported/unknown | 16 (16.5) | 14 (14.3) | 30 (15.4) |
| Weight, mean (SD), kg | 72.4 (16.8) | 73.4 (14.6) | 72.9 (15.7) |
| Height, mean (SD), cm | 168.3 (10.1) | 168.8 (9.9) | 168.5 (10.0) |
| Years on eculizumab before first study infusion | 6.0 (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), y | 34.1 (14.4) | 36.8 (14.1) | 35.5 (14.3) |
| Time from PNH diagnosis to consent, mean (SD), y | 12.4 (8.4) | 11.9 (9.4) | 12.2 (8.9) |
| LDH, mean (SD),* U/L | 228.0 (48.7) | 235.2 (49.7) | 231.6 (49.2) |
| PNH clone size, mean (SD), % | |||
| Type II red blood cells | 14.9 (19.6) | 16.3 (23.6) | 15.6 (21.6) |
| Type III red blood cells† | 44.6 (30.5) | 43.5 (29.7) | 44.0 (30.0) |
| Total red blood cells | 60.6 (32.5) | 59.5 (31.4) | 60.1 (31.9) |
| Granulocyte | 82.6 (23.6) | 84.0 (21.4) | 83.3 (22.5) |
| Monocyte | 85.6 (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) |
| Characteristic . | Ravulizumab (n = 97) . | Eculizumab (n = 98) . | Total (N = 195) . |
|---|---|---|---|
| Sex, no. (%) | |||
| Male | 50 (51.5) | 48 (49.0) | 98 (50.3) |
| Female | 47 (48.5) | 50 (51.0) | 97 (49.7) |
| Age at first infusion of study drug, mean (SD), y | 46.6 (14.4) | 48.8 (14.0) | 47.7 (14.2) |
| Race, no. (%) | |||
| White | 50 (51.5) | 61 (62.2) | 111 (56.9) |
| Asian | 23 (23.7) | 19 (19.4) | 42 (21.5) |
| Japanese | 5 (5.2) | 7 (7.1) | 12 (6.2) |
| African American | 5 (5.2) | 3 (3.1) | 8 (4.1) |
| Other/multiple | 3 (3.1) | 1 (1.0) | 4 (2.1) |
| Not reported/unknown | 16 (16.5) | 14 (14.3) | 30 (15.4) |
| Weight, mean (SD), kg | 72.4 (16.8) | 73.4 (14.6) | 72.9 (15.7) |
| Height, mean (SD), cm | 168.3 (10.1) | 168.8 (9.9) | 168.5 (10.0) |
| Years on eculizumab before first study infusion | 6.0 (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), y | 34.1 (14.4) | 36.8 (14.1) | 35.5 (14.3) |
| Time from PNH diagnosis to consent, mean (SD), y | 12.4 (8.4) | 11.9 (9.4) | 12.2 (8.9) |
| LDH, mean (SD),* U/L | 228.0 (48.7) | 235.2 (49.7) | 231.6 (49.2) |
| PNH clone size, mean (SD), % | |||
| Type II red blood cells | 14.9 (19.6) | 16.3 (23.6) | 15.6 (21.6) |
| Type III red blood cells† | 44.6 (30.5) | 43.5 (29.7) | 44.0 (30.0) |
| Total red blood cells | 60.6 (32.5) | 59.5 (31.4) | 60.1 (31.9) |
| Granulocyte | 82.6 (23.6) | 84.0 (21.4) | 83.3 (22.5) |
| Monocyte | 85.6 (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) |