Table 2.
Summary of PEGASUS trial outcomes at Week 16 results: intention-to-treat population. Source: Company submission [18]
| Pegcetacoplan (n = 41) | Eculizumab (n = 39) | Risk difference (95% CI) | p value | |
|---|---|---|---|---|
| Transfusion avoidance, n (%)a | 35 (85.4) | 6 (15.4) | 0.63 (0.48–0.77) | < 0.0001 |
| Hb response in absence of transfusion, n (%)b | 31 (75.6) | 0 (0.0) | 0.68 (0.55–0.80) | NR |
| Hb normalisation in absence of transfusion, n (%)c | 14 (34.1) | 0 (0.0) | 0.30 (0.15–0.46) | NR |
| ARC normalisation in absence of transfusion, n (%)d | 32 (78.0) | 1 (2.6) | 0.66 (0.53–0.80) | NR |
| LDH normalisation in absence of transfusion, n (%)e | 29 (70.7) | 6 (15.4) | NR | NR |
ARC absolute reticulocyte count normalisation, CI confidence interval, Hb haemoglobin, LDH lactate dehydrogenase, LLN lower limit of normal, NR not reported, ULN upper limit of normal
aNumber of patients who did not receive a transfusion
bDefined as an increase of ≥ 1 g/dL from baseline Hb level at Week 16 without transfusion
cDefined as Hb level ≥ the gender-specific LLN range (female LLN: 12 g/dL; male LLN: 13.6 g/dL) at Week 16 without transfusion
dDefined as ARC < 120 × 109 cells/L (ULN) at Week 16 without transfusion
eDefined as LDH level < 226 U/L (ULN) at Week 16 without transfusion