Table I.
Demographics and baseline characteristics.
| TRIUMPH |
SHEPHERD |
||
|---|---|---|---|
| Parameter | Eculizumab (n = 43) | Placebo (n = 44) | (n = 97) |
| Sex, n (%) | |||
| Male | 20 (47) | 15 (34) | 48 (49) |
| Female | 23 (53) | 29 (66) | 49 (51) |
| Median age, years (range) | 41 (20–85) | 35 (18–78) | 41 (18–78) |
| Median duration of PNH, years (range) | 43· (0·9–29·8) | 9·2 (0·5–38·5) | 4·9 (0·1–31·4) |
| Median units packed RBC transfused | 9 (6–12) | 8·5 (7–12·5) | 8·0 (0–66·0) |
| prior to screening,* units (range) | |||
| Units transfused 12 months prior to screening, n (%) | |||
| <4 units | – | – | 21 (22) |
| 4–14 units | 15 (35) | 15 (34) | 47 (48) |
| 15–25 units | 17 (40) | 18 (41) | 15 (15) |
| >25 units | 11 (26) | 11 (25) | 14 (14) |
| Median reticulocyte count, 1012/l (range) | 0·207 (0·040–0·570) | 0·206 (0·045–0·556) | 0·140 (0·036–0·757) |
| Median platelet count, 109/l (range) | 178·0 (72·0–416·0) | 138·5 (59·0–547·0) | 136·0 (23·0–355) |
| Median neutrophil count, 109/l (range) | 2·3 (0·8–11·2) | 2·1 (0·1–11·0) | 2·3 (0·2–7·4) |
| Use of erythropoietin, n (%) | 3 (7) | 0 | 5 (5) |
| Use of folate, iron or B12, n (%) | 5 (12) | 6 (14) | 17 (18) |
| Median haemoglobin set point,* g/l (range) | 77 (61–88) | 77 (62–90) | – |
PNH, paroxysmal nocturnal haemoglobinuria; RBC, red blood cell.
*
Qualifying transfusion. Data for TRIUMPH represents units transfused during 12 months prior to screening normalized to a 6-month equivalent.
Data for SHEPHERD represents units transfused during 12 months prior to screening.