Patient demographics and clinical characteristics at baseline in the 301 study
| Variable . | Eculizumab (n = 121) . | Ravulizumab (n = 125) . | Total (N = 246) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 69 (57.0) | 65 (52.0) | 134 (54.5) |
| Female | 52 (43.0) | 60 (48.0) | 112 (45.5) |
| Age at PNH diagnosis, mean ± SD (range), y | 39.6 ± 16.7 (13-82) | 37.9 ± 14.9 (15-81) | 38.7 ± 15.8 (13-82) |
| Age at first infusion, mean ± SD (range), y | 46.2 ± 16.24 (18-86) | 44.8 ± 15.16 (18-83) | 45.5 ± 15.69 (18-86) |
| Race, n (%) | |||
| White | 50 (41.3) | 43 (34.4) | 93 (37.8) |
| Asian | 57 (47.1) | 72 (57.6) | 129 (52.4) |
| African American | 4 (3.3) | 2 (1.6) | 6 (2.4) |
| American Indian or Alaska Native | 1 (0.8) | 1 (0.8) | 2 (0.8) |
| Not reported | 5 (4.1) | 3 (2.4) | 8 (3.3) |
| Other | 4 (3.3) | 4 (3.2) | 8 (3.3) |
| Ethnicity, n (%) | |||
| Not Hispanic or Latino | 101 (83.5) | 116 (92.8) | 217 (88.2) |
| Hispanic or Latino | 13 (10.7) | 5 (4.0) | 18 (7.3) |
| Not reported | 4 (3.3) | 2 (1.6) | 6 (2.4) |
| Missing/unknown | 3 (2.5) | 2 (1.6) | 5 (2.0) |
| Baseline weight, mean ± SD (range), kg | 69.2 ± 14.86 (45-113) | 68.2 ± 15.58 (40-115) | 68.7 ± 15.21 (40-115) |
| LDH, mean ± SD (range), U/L | 1578.3 ± 727.1 (423.5-3139.5) | 1633.5 ± 778.8 (378.0-3759.5) | 1606.4 ± 752.7 (378.0-3759.5) |
| PNH clone size, mean ± SD (range) | |||
| RBC type II | 13.7 ± 17.7 (0.1-95.3) | 12.4 ± 20.5 (0.1-99.5) | 13.0 ± 19.2 (0.1-99.5) |
| RBC type III | 25.2 ± 16.9 (0.4-75.6) | 26.3 ± 17.2 (0.1-82.0) | 25.8 ± 17.1 (0.1-82.0) |
| Total RBC | 38.7 ± 23.2 (2.2-98.0) | 38.4 ± 23.7 (3.0-99.6) | 38.6 ± 23.4 (2.2-99.6) |
| Granulocytes | 85.3 ± 19.0 (8.0-100.0) | 84.2 ± 21.0 (4.2-99.9) | 84.7 ± 20.0 (4.2-100.0) |
| Monocytes | 89.2 ± 15.2 (17.0-99.9) | 86.9 ± 18.1 (8.5-99.9) | 88.0 ± 16.7 (8.5-99.9) |
| Patients with a history of pRBC/whole blood transfusion, n (%)∗ | 100 (82.6) | 103 (82.4) | 203 (82.5) |
| Number of pRBC/whole blood transfusions, total, mean ± SD (range)∗ | 572 5.7 ± 5.53 (1-28) | 677 6.6 ± 6.04 (1-28) | 1249 6.2 ± 5.80 (1-28) |
| Units of pRBC/whole blood transfusion, total, mean ± SD (range) | 861 8.6 ± 7.9 (1-32) | 925 9.0 ± 7.7 (1-44) | 1786 8.8 ± 7.8 (1-44) |
| History of PNH-associated conditions, n (%) | |||
| Aplastic anemia | 38 (31.4) | 41 (32.8) | 79 (32.1) |
| Myelodysplastic syndrome | 6 (5.0) | 7 (5.6) | 13 (5.3) |
| Variable . | Eculizumab (n = 121) . | Ravulizumab (n = 125) . | Total (N = 246) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 69 (57.0) | 65 (52.0) | 134 (54.5) |
| Female | 52 (43.0) | 60 (48.0) | 112 (45.5) |
| Age at PNH diagnosis, mean ± SD (range), y | 39.6 ± 16.7 (13-82) | 37.9 ± 14.9 (15-81) | 38.7 ± 15.8 (13-82) |
| Age at first infusion, mean ± SD (range), y | 46.2 ± 16.24 (18-86) | 44.8 ± 15.16 (18-83) | 45.5 ± 15.69 (18-86) |
| Race, n (%) | |||
| White | 50 (41.3) | 43 (34.4) | 93 (37.8) |
| Asian | 57 (47.1) | 72 (57.6) | 129 (52.4) |
| African American | 4 (3.3) | 2 (1.6) | 6 (2.4) |
| American Indian or Alaska Native | 1 (0.8) | 1 (0.8) | 2 (0.8) |
| Not reported | 5 (4.1) | 3 (2.4) | 8 (3.3) |
| Other | 4 (3.3) | 4 (3.2) | 8 (3.3) |
| Ethnicity, n (%) | |||
| Not Hispanic or Latino | 101 (83.5) | 116 (92.8) | 217 (88.2) |
| Hispanic or Latino | 13 (10.7) | 5 (4.0) | 18 (7.3) |
| Not reported | 4 (3.3) | 2 (1.6) | 6 (2.4) |
| Missing/unknown | 3 (2.5) | 2 (1.6) | 5 (2.0) |
| Baseline weight, mean ± SD (range), kg | 69.2 ± 14.86 (45-113) | 68.2 ± 15.58 (40-115) | 68.7 ± 15.21 (40-115) |
| LDH, mean ± SD (range), U/L | 1578.3 ± 727.1 (423.5-3139.5) | 1633.5 ± 778.8 (378.0-3759.5) | 1606.4 ± 752.7 (378.0-3759.5) |
| PNH clone size, mean ± SD (range) | |||
| RBC type II | 13.7 ± 17.7 (0.1-95.3) | 12.4 ± 20.5 (0.1-99.5) | 13.0 ± 19.2 (0.1-99.5) |
| RBC type III | 25.2 ± 16.9 (0.4-75.6) | 26.3 ± 17.2 (0.1-82.0) | 25.8 ± 17.1 (0.1-82.0) |
| Total RBC | 38.7 ± 23.2 (2.2-98.0) | 38.4 ± 23.7 (3.0-99.6) | 38.6 ± 23.4 (2.2-99.6) |
| Granulocytes | 85.3 ± 19.0 (8.0-100.0) | 84.2 ± 21.0 (4.2-99.9) | 84.7 ± 20.0 (4.2-100.0) |
| Monocytes | 89.2 ± 15.2 (17.0-99.9) | 86.9 ± 18.1 (8.5-99.9) | 88.0 ± 16.7 (8.5-99.9) |
| Patients with a history of pRBC/whole blood transfusion, n (%)∗ | 100 (82.6) | 103 (82.4) | 203 (82.5) |
| Number of pRBC/whole blood transfusions, total, mean ± SD (range)∗ | 572 5.7 ± 5.53 (1-28) | 677 6.6 ± 6.04 (1-28) | 1249 6.2 ± 5.80 (1-28) |
| Units of pRBC/whole blood transfusion, total, mean ± SD (range) | 861 8.6 ± 7.9 (1-32) | 925 9.0 ± 7.7 (1-44) | 1786 8.8 ± 7.8 (1-44) |
| History of PNH-associated conditions, n (%) | |||
| Aplastic anemia | 38 (31.4) | 41 (32.8) | 79 (32.1) |
| Myelodysplastic syndrome | 6 (5.0) | 7 (5.6) | 13 (5.3) |
pRBC, packed RBC; SD, standard deviation.
In the 12 months before the first study dose of ravulizumab or eculizumab.