Other secondary efficacy outcomes at day 183
| . | Ravulizumab (N = 125) . | Eculizumab (N = 121)* . | ||
|---|---|---|---|---|
| Patients who received packed RBC transfusions, n (%) | 32 (25.6) | 40 (33.1) | ||
| Total number of packed RBC units transfused, mean (SD) | 4.8 (5.1) | 5.6 (5.9) | ||
| Patients with major adverse vascular events, n (%) | 2 (1.6)† | 1 (0.8)‡ | ||
| Clinical manifestations of paroxysmal nocturnal hemoglobinuria, n (%) | Baseline | Day 183 | Baseline | Day 183 |
| Fatigue | 80 (64.0) | 36 (28.8) | 76 (63.9) | 36 (30.3) |
| Abdominal pain | 17 (13.6) | 6 (4.8) | 15 (12.6) | 6 (5.0) |
| Dyspnea | 42 (33.6) | 18 (14.4) | 38 (31.9) | 17 (14.3) |
| Dysphagia | 13 (10.4) | 3 (2.4) | 16 (13.4) | 1 (0.8) |
| Chest pain | 5 (4.0) | 3 (2.4) | 17 (14.3) | 7 (5.9) |
| Hemoglobinuria§ | 71 (56.8) | 13 (10.4) | 56 (47.5) | 11 (9.3) |
| Erectile dysfunction|| | 16 (12.8) | 10 (8.0) | 21 (17.6) | 5 (4.2) |
| . | Ravulizumab (N = 125) . | Eculizumab (N = 121)* . | ||
|---|---|---|---|---|
| Patients who received packed RBC transfusions, n (%) | 32 (25.6) | 40 (33.1) | ||
| Total number of packed RBC units transfused, mean (SD) | 4.8 (5.1) | 5.6 (5.9) | ||
| Patients with major adverse vascular events, n (%) | 2 (1.6)† | 1 (0.8)‡ | ||
| Clinical manifestations of paroxysmal nocturnal hemoglobinuria, n (%) | Baseline | Day 183 | Baseline | Day 183 |
| Fatigue | 80 (64.0) | 36 (28.8) | 76 (63.9) | 36 (30.3) |
| Abdominal pain | 17 (13.6) | 6 (4.8) | 15 (12.6) | 6 (5.0) |
| Dyspnea | 42 (33.6) | 18 (14.4) | 38 (31.9) | 17 (14.3) |
| Dysphagia | 13 (10.4) | 3 (2.4) | 16 (13.4) | 1 (0.8) |
| Chest pain | 5 (4.0) | 3 (2.4) | 17 (14.3) | 7 (5.9) |
| Hemoglobinuria§ | 71 (56.8) | 13 (10.4) | 56 (47.5) | 11 (9.3) |
| Erectile dysfunction|| | 16 (12.8) | 10 (8.0) | 21 (17.6) | 5 (4.2) |
n = 119 patients were included in the analysis of clinical manifestations of PNH.
One patient, who was taking concomitant oral contraceptive medication, experienced an event of lower leg deep vein thrombosis. The other patient had history of lower leg pain and edema and was taking an oral anticoagulant, which was discontinued after initiation of study drug.
This event of mesenteric venous thrombosis with concurrent neutropenic colitis occurred in a patient who had history of aplastic anemia.
n = 118 patients with evaluable data in the eculizumab group.
n = 65 male patients in the ravulizumab group and n = 68 male patients in the eculizumab group.