Table III.
Effect of eculizumab on transfusion requirements in patients with paroxysmal nocturnal haemoglobinuria by pretreatment transfusion strata.
| TRIUMPH |
SHEPHERD |
|||||||
|---|---|---|---|---|---|---|---|---|
| Median (mean ± SE) |
Median (mean ± SE) |
|||||||
| Pretreatment transfusion strata | 6-month pretreatment‡ |
Week 26 |
||||||
| Placebo | Eculizumab | Placebo | Eculizumab | P-value* | 1-year Pretreatment | Week 52 Eculizumab | P-value† | |
| Overall | 8·5 (9·9 ± 0·7) | 9·0 (9·5 ± 0·6) | 10·0 (11·0 ± 0·83) | 0·0 (3·0 ± 0·67) | <0·001 | 8·0 (12·3 ± 1·25) | 0·0 (5·9 ± 1·06 | <0·001 |
| <4 units | – | – | – | – | – | 2·0 (1·6 ± 0·31) | 0·0 (1·5 ± 0·69) | 0·42 |
| 4–14 units | 6·5 (6·2 ± 0·4) | 5·5 (5·4 ± 0·3) | 6·0 (6·7 ± 0·72) | 0·0 (0·4 ± 0·29) | <0·001 | 8·0 (7·6 ± 0·44) | 0·0 (4·9 ± 1·39) | 0·002 |
| 15–25 units | 8·8 (8·9 ± 0·5) | 9·5 (9·3 ± 0·3) | 10·0 (10·8 ± 1·17) | 2·0 (4·2 ± 1·14) | <0·001 | 17·0 (19·3 ± 1·01) | 4·0 (9·5 ± 3·10) | 0·008 |
| >25 units | 15·0 (16·3 ± 0·9) | 16·5 (15·6 ± 0·7) | 18·0 (17·0 ± 1·04) | 3·0 (4·5 ± 1·59) | <0·001 | 33·5 (36·6 ± 2·78) | 7·5 (11·8 ± 4·02) | <0·001 |
P-values are based on a comparison of the median values.
*
Wilcoxon’s rank sum test.
†
Signed rank test.
‡
Transfusion data obtained during 12 months before treatment were normalized to a value equivalent to the value for a 6-month period.