Table 3.
Effectiveness of eculizumab in paediatric patients with aHUS
| Effectiveness endpoint | Total number of patients | Number of patients reporting the effectiveness outcome | Percentage of patients reporting the effectiveness outcome |
95% CI |
|---|---|---|---|---|
| Complete TMA response | 30 | 22 | 73.3 | 54.1–87.7 |
| Haematologic normalization | 30 | 22 | 73.3 | 54.1–87.7 |
| PLT count normalization | 34 | 26 | 76.5 | 58.8–89.3 |
| LDH normalization (<1.5 times the ULN) | 33 | 25 | 75.8 | 57.7–88.9 |
| sCr level improvement | 40 | 28 | 70.0 | 53.5–83.4 |
| eGFR improvement | 23 | 18 | 78.3 | 56.3–92.5 |
| TMA event-free status | 40 | 31 | 77.5 | 61.5–89.2 |