Demographics and baseline clinical characteristics
| Characteristic . | Ravulizumab (N = 125) . | Eculizumab (N = 121) . | Total (N = 246) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 65 (52.0) | 69 (57.0) | 134 (54.5) |
| Female | 60 (48.0) | 52 (43.0) | 112 (45.5) |
| Age at first infusion of study drug, mean (SD), y | 44.8 (15.2) | 46.2 (16.2) | 45.5 (15.7) |
| Race, n (%) | |||
| Asian | 72 (57.6) | 57 (47.1) | 129 (52.4) |
| Japanese | 19 (15.2) | 15 (12.4) | 34 (13.8) |
| White | 43 (34.4) | 51 (42.1) | 94 (38.2) |
| Black or African American | 2 (1.6) | 4 (3.3) | 6 (2.4) |
| American Indian or Alaska Native | 1 (0.8) | 1 (0.8) | 2 (0.8) |
| Other | 4 (3.2) | 4 (3.3) | 8 (3.3) |
| Not reported | 3 (2.4) | 4 (3.3) | 7 (2.8) |
| Weight, mean (SD), kg | 68.2 (15.6) | 69.2 (14.9) | 68.7 (15.2) |
| Height, mean (SD), cm | 166.3 (9.0) | 166.2 (10.7) | 166.2 (9.8) |
| LDH ratio, n (%) | |||
| 1.5 to <3× ULN* | 18 (14.4) | 16 (13.2) | 34 (13.8) |
| ≥3× ULN | 107 (85.6) | 105 (86.8) | 212 (86.2) |
| Packed RBC units received within 1 y before study entry, randomization strata, n (%) | |||
| 0 U | 23 (18.4) | 21 (17.4) | 44 (17.9) |
| 1-14 U | 79 (63.2) | 78 (64.5) | 157 (63.8) |
| >14 U | 23 (18.4) | 22 (18.2) | 45 (18.3) |
| Age at PNH diagnosis, mean (SD), y | 37.9 (14.9)† | 39.6 (16.7)‡ | 38.7 (15.8)§ |
| Number of years from PNH diagnosis to consent, median (minimum, maximum), y | 3.8 (0, 41)† | 3.9 (0, 34)‡ | 3.9 (0, 41)§ |
| LDH, mean (SD), U/L | 1633.5 (778.8) | 1578.3 (727.1) | 1606.4 (752.7) |
| PNH clone size, mean (SD), % | |||
| Type II RBCs | 12.4 (20.5)|| | 13.7 (17.7)¶ | 13.0 (19.2)# |
| Type III RBCs | 26.3 (17.2)|| | 25.2 (16.9)¶ | 25.8 (17.1)# |
| Total RBCs | 38.4 (23.7) | 38.7 (23.2) | 38.6 (23.4) |
| Granulocytes | 84.2 (21.0) | 85.3 (19.0) | 84.7 (20.0) |
| Monocytes | 86.9 (18.1) | 89.2 (15.2) | 88.0 (16.7) |
| History of major adverse vascular events, n (%) | 17 (13.6) | 25 (20.7) | 42 (17.1) |
| Characteristic . | Ravulizumab (N = 125) . | Eculizumab (N = 121) . | Total (N = 246) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 65 (52.0) | 69 (57.0) | 134 (54.5) |
| Female | 60 (48.0) | 52 (43.0) | 112 (45.5) |
| Age at first infusion of study drug, mean (SD), y | 44.8 (15.2) | 46.2 (16.2) | 45.5 (15.7) |
| Race, n (%) | |||
| Asian | 72 (57.6) | 57 (47.1) | 129 (52.4) |
| Japanese | 19 (15.2) | 15 (12.4) | 34 (13.8) |
| White | 43 (34.4) | 51 (42.1) | 94 (38.2) |
| Black or African American | 2 (1.6) | 4 (3.3) | 6 (2.4) |
| American Indian or Alaska Native | 1 (0.8) | 1 (0.8) | 2 (0.8) |
| Other | 4 (3.2) | 4 (3.3) | 8 (3.3) |
| Not reported | 3 (2.4) | 4 (3.3) | 7 (2.8) |
| Weight, mean (SD), kg | 68.2 (15.6) | 69.2 (14.9) | 68.7 (15.2) |
| Height, mean (SD), cm | 166.3 (9.0) | 166.2 (10.7) | 166.2 (9.8) |
| LDH ratio, n (%) | |||
| 1.5 to <3× ULN* | 18 (14.4) | 16 (13.2) | 34 (13.8) |
| ≥3× ULN | 107 (85.6) | 105 (86.8) | 212 (86.2) |
| Packed RBC units received within 1 y before study entry, randomization strata, n (%) | |||
| 0 U | 23 (18.4) | 21 (17.4) | 44 (17.9) |
| 1-14 U | 79 (63.2) | 78 (64.5) | 157 (63.8) |
| >14 U | 23 (18.4) | 22 (18.2) | 45 (18.3) |
| Age at PNH diagnosis, mean (SD), y | 37.9 (14.9)† | 39.6 (16.7)‡ | 38.7 (15.8)§ |
| Number of years from PNH diagnosis to consent, median (minimum, maximum), y | 3.8 (0, 41)† | 3.9 (0, 34)‡ | 3.9 (0, 41)§ |
| LDH, mean (SD), U/L | 1633.5 (778.8) | 1578.3 (727.1) | 1606.4 (752.7) |
| PNH clone size, mean (SD), % | |||
| Type II RBCs | 12.4 (20.5)|| | 13.7 (17.7)¶ | 13.0 (19.2)# |
| Type III RBCs | 26.3 (17.2)|| | 25.2 (16.9)¶ | 25.8 (17.1)# |
| Total RBCs | 38.4 (23.7) | 38.7 (23.2) | 38.6 (23.4) |
| Granulocytes | 84.2 (21.0) | 85.3 (19.0) | 84.7 (20.0) |
| Monocytes | 86.9 (18.1) | 89.2 (15.2) | 88.0 (16.7) |
| History of major adverse vascular events, n (%) | 17 (13.6) | 25 (20.7) | 42 (17.1) |