Patient demographics and clinical characteristics at baseline in the 302 study
| Variable . | Eculizumab (n = 98) . | Ravulizumab (n = 97) . | Total (N = 195) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 48 (49.0) | 50 (51.5) | 98 (50.3) |
| Female | 50 (51.0) | 47 (48.5) | 97 (49.7) |
| Age at PNH diagnosis, mean ± SD (range), y | 36.8 ± 14.14 (11-74) | 34.1 ± 14.41 (6-73) | 35.5 ± 14.30 (6-74) |
| Age at first eculizumab infusion, mean ± SD (range), y | 43.2 ± 13.93 (14-74) | 40.7 ± 14.30 (14-74) | 42.0 ± 14.14 (14-74) |
| Race, n (%) | |||
| White | 61 (62.2) | 50 (51.5) | 111 (56.9) |
| Asian | 19 (19.4) | 23 (23.7) | 42 (21.5) |
| African American | 3 (3.1) | 5 (5.2) | 8 (4.1) |
| Not reported | 13 (13.3) | 13 (13.4) | 26 (13.3) |
| Other | 1 (1.0) | 2 (2.1) | 3 (1.5) |
| Unknown | 1 (1.0) | 3 (3.1) | 4 (2.1) |
| Ethnicity, n (%) | |||
| Not Hispanic or Latino | 77 (78.6) | 76 (78.4) | 153 (78.5) |
| Hispanic or Latino | 4 (4.1) | 3 (3.1) | 7 (3.6) |
| Not reported | 17 (17.3) | 15 (15.5) | 32 (16.4) |
| Missing/unknown | 0 | 3 (3.1) | 3 (1.5) |
| Baseline weight, mean ± SD (range), kg | 73.4 ± 14.6 (44-104) | 72.4 ± 16.8 (45-129) | 72.9 ± 15.7 (44-129) |
| LDH, mean ± SD (range), U/L | 235.2 ± 49.7 (100.0-365.5) | 228.0 ± 48.7 (135.0-383.5) | 231.64 ± 49.2 (100.0-383.5) |
| Hemoglobin, mean ± SD (range), g/dL | 10.9 ± 1.8 (4.8-15.5) | 11.1 ± 1.8 (7.5-16.1) | 11.0 ± 1.8 (4.8-16.1) |
| PNH clone size, mean ± SD (range) | |||
| RBC type II | 16.3 ± 23.6 (0.1-91.9) | 14.9 ± 19.6 (0.1-80.9) | 15.6 ± 21.6 (0.1-80.9) |
| RBC type III | 43.5 ± 29.7 (0.9-99.4) | 44.6 ± 30.5 (0.0-98.8) | 44.0 ± 30.0 (0.0-99.4) |
| Total RBC | 59.5 ± 31.4 (1.5-99.6) | 60.7 ± 32.5 (0.1-99.8) | 60.1 ± 31.9 (0.1-99.8) |
| Granulocytes | 84.0 ± 21.4 (3.3-99.8) | 82.6 ± 23.6 (7.4-99.9) | 83.3 ± 22.5 (3.3-99.9) |
| Monocytes | 86.1 ± 19.7 (12.1-99.9) | 85.6 ± 20.5 (11.2-99.9) | 85.9 ± 20.0 (11.2-99.9) |
| Patients with a history of pRBC/whole blood transfusion, n (%)∗ | 12 (12.2) | 13 (13.4) | 25 (12.8) |
| Number of pRBC/whole blood transfusions, total, mean ± SD (range)∗ | 30 2.5 ± 2.3 (1-8) | 64 4.9 ± 5.5 (1-17) | 94 3.8 ± 4.4 (1-17) |
| Units of pRBC/whole blood transfusion, total, mean ± SD (range) | 50 4.2 ± 3.8 (2.0-15.0) | 103 7.9 ± 8.8 (1.0-32.0) | 153 6.1 ± 7.0 (1-32) |
| FACIT-F score, mean ± SD (range) | 40.7 ± 9.5 (16.0-52.0) | 42.5 ± 9.4 (1.0-52.0) | 41.6 ± 9.5 (1.0-52.0) |
| History of PNH-associated conditions, n (%) | |||
| Aplastic anemia | 39 (39.8) | 34 (35.1) | 73 (37.4) |
| Myelodysplastic syndrome | 6 (6.1) | 3 (3.1) | 9 (4.6) |
| Variable . | Eculizumab (n = 98) . | Ravulizumab (n = 97) . | Total (N = 195) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 48 (49.0) | 50 (51.5) | 98 (50.3) |
| Female | 50 (51.0) | 47 (48.5) | 97 (49.7) |
| Age at PNH diagnosis, mean ± SD (range), y | 36.8 ± 14.14 (11-74) | 34.1 ± 14.41 (6-73) | 35.5 ± 14.30 (6-74) |
| Age at first eculizumab infusion, mean ± SD (range), y | 43.2 ± 13.93 (14-74) | 40.7 ± 14.30 (14-74) | 42.0 ± 14.14 (14-74) |
| Race, n (%) | |||
| White | 61 (62.2) | 50 (51.5) | 111 (56.9) |
| Asian | 19 (19.4) | 23 (23.7) | 42 (21.5) |
| African American | 3 (3.1) | 5 (5.2) | 8 (4.1) |
| Not reported | 13 (13.3) | 13 (13.4) | 26 (13.3) |
| Other | 1 (1.0) | 2 (2.1) | 3 (1.5) |
| Unknown | 1 (1.0) | 3 (3.1) | 4 (2.1) |
| Ethnicity, n (%) | |||
| Not Hispanic or Latino | 77 (78.6) | 76 (78.4) | 153 (78.5) |
| Hispanic or Latino | 4 (4.1) | 3 (3.1) | 7 (3.6) |
| Not reported | 17 (17.3) | 15 (15.5) | 32 (16.4) |
| Missing/unknown | 0 | 3 (3.1) | 3 (1.5) |
| Baseline weight, mean ± SD (range), kg | 73.4 ± 14.6 (44-104) | 72.4 ± 16.8 (45-129) | 72.9 ± 15.7 (44-129) |
| LDH, mean ± SD (range), U/L | 235.2 ± 49.7 (100.0-365.5) | 228.0 ± 48.7 (135.0-383.5) | 231.64 ± 49.2 (100.0-383.5) |
| Hemoglobin, mean ± SD (range), g/dL | 10.9 ± 1.8 (4.8-15.5) | 11.1 ± 1.8 (7.5-16.1) | 11.0 ± 1.8 (4.8-16.1) |
| PNH clone size, mean ± SD (range) | |||
| RBC type II | 16.3 ± 23.6 (0.1-91.9) | 14.9 ± 19.6 (0.1-80.9) | 15.6 ± 21.6 (0.1-80.9) |
| RBC type III | 43.5 ± 29.7 (0.9-99.4) | 44.6 ± 30.5 (0.0-98.8) | 44.0 ± 30.0 (0.0-99.4) |
| Total RBC | 59.5 ± 31.4 (1.5-99.6) | 60.7 ± 32.5 (0.1-99.8) | 60.1 ± 31.9 (0.1-99.8) |
| Granulocytes | 84.0 ± 21.4 (3.3-99.8) | 82.6 ± 23.6 (7.4-99.9) | 83.3 ± 22.5 (3.3-99.9) |
| Monocytes | 86.1 ± 19.7 (12.1-99.9) | 85.6 ± 20.5 (11.2-99.9) | 85.9 ± 20.0 (11.2-99.9) |
| Patients with a history of pRBC/whole blood transfusion, n (%)∗ | 12 (12.2) | 13 (13.4) | 25 (12.8) |
| Number of pRBC/whole blood transfusions, total, mean ± SD (range)∗ | 30 2.5 ± 2.3 (1-8) | 64 4.9 ± 5.5 (1-17) | 94 3.8 ± 4.4 (1-17) |
| Units of pRBC/whole blood transfusion, total, mean ± SD (range) | 50 4.2 ± 3.8 (2.0-15.0) | 103 7.9 ± 8.8 (1.0-32.0) | 153 6.1 ± 7.0 (1-32) |
| FACIT-F score, mean ± SD (range) | 40.7 ± 9.5 (16.0-52.0) | 42.5 ± 9.4 (1.0-52.0) | 41.6 ± 9.5 (1.0-52.0) |
| History of PNH-associated conditions, n (%) | |||
| Aplastic anemia | 39 (39.8) | 34 (35.1) | 73 (37.4) |
| Myelodysplastic syndrome | 6 (6.1) | 3 (3.1) | 9 (4.6) |
pRBC, packed RBC; SD, standard deviation.
In the 12 months before the first study dose of ravulizumab or eculizumab.