Baseline characteristics of patients with PNH from parent trials
| Characteristics . | Pilot, n = 11 . | TRIUMPH, n = 87* . | SHEPHERD, n = 97 . |
|---|---|---|---|
| Sex, no. (%) | |||
| Male | 6 (55) | 35 (40) | 48 (49) |
| Female | 5 (45) | 52 (60) | 49 (51) |
| Median age, y (range) | 48 (21-67) | 38 (18-85) | 41 (18-78) |
| Median disease duration, y (range) | 8.7 (1.7-37.9) | 6.5 (0.5-38.5) | 4.9 (0.1-31.4) |
| Reticulocyte count, × 1012/L (range) | 0.14 (0.07-0.37) | 0.21 (0.04-0.57) | 0.14 (0.04-0.76) |
| Platelet count, × 109/L (range) | 182 (36-466) | 162 (59-547) | 136 (23-355) |
| Median PNH granulocyte population size, % (range) | 97.0 (47.8-99.8) | 95.3 (82.6-99.5)† | 96.0 (1.1-99.9)‡ |
| Median PNH type III red cell population size, % (range) | 37.0 (10.4-79.6) | 29.5 (2.4-88.0) | 33.5 (7.7-98.8) |
| History of aplastic anemia or myelodysplastic syndromes, no. (%) | 8 (73) | 20 (23) | 31 (32) |
| History of TE events, no. (%) | 3 (27) | 17 (19) | 42 (43) |
| Use of antithrombotic agents, no. (%) | 6 (55) | 44 (51) | 59 (61) |
| Use of steroids, no. (%) | 0 (0) | 28 (32) | 36 (37) |
| Characteristics . | Pilot, n = 11 . | TRIUMPH, n = 87* . | SHEPHERD, n = 97 . |
|---|---|---|---|
| Sex, no. (%) | |||
| Male | 6 (55) | 35 (40) | 48 (49) |
| Female | 5 (45) | 52 (60) | 49 (51) |
| Median age, y (range) | 48 (21-67) | 38 (18-85) | 41 (18-78) |
| Median disease duration, y (range) | 8.7 (1.7-37.9) | 6.5 (0.5-38.5) | 4.9 (0.1-31.4) |
| Reticulocyte count, × 1012/L (range) | 0.14 (0.07-0.37) | 0.21 (0.04-0.57) | 0.14 (0.04-0.76) |
| Platelet count, × 109/L (range) | 182 (36-466) | 162 (59-547) | 136 (23-355) |
| Median PNH granulocyte population size, % (range) | 97.0 (47.8-99.8) | 95.3 (82.6-99.5)† | 96.0 (1.1-99.9)‡ |
| Median PNH type III red cell population size, % (range) | 37.0 (10.4-79.6) | 29.5 (2.4-88.0) | 33.5 (7.7-98.8) |
| History of aplastic anemia or myelodysplastic syndromes, no. (%) | 8 (73) | 20 (23) | 31 (32) |
| History of TE events, no. (%) | 3 (27) | 17 (19) | 42 (43) |
| Use of antithrombotic agents, no. (%) | 6 (55) | 44 (51) | 59 (61) |
| Use of steroids, no. (%) | 0 (0) | 28 (32) | 36 (37) |