Table 2.
Clinical endpoints of effectiveness for patients with aHUS
| TMA event-free status, n | 27 |
| n (%) | 23 (85.2) |
| 95%CI | 66.3–95.8 |
| Complete TMA response, n | 22 |
| n (%) | 8 (36.4) |
| 95% CI | 17.2–59.3 |
| Hematologic outcome | |
| Hematologic normalization, n | 22 |
| n (%) | 9 (40.9) |
| 95% CI | 20.7–63.6 |
| Platelet count normalization, n | 23 |
| n (%) | 18 (78.3) |
| 95% CI | 56.3–92.5 |
| LDH normalization, n | 25 |
| n (%) | 12 (48.0) |
| 95% CI | 27.8–68.7 |
| Hemoglobin improvement ≥ 2 g/dl, n | 27 |
| n (%) | 17 (63.0) |
| 95% CI | 42.4–80.6 |
| Renal outcome | |
| Serum creatinine level decrease by ≥ 25%, n | 27 |
| n (%) | 18 (66.7) |
| 95% CI | 46.0-83.5 |
| eGFR improvement by ≥ 15 ml/min/1.73 m2, n | 12 |
| n (%) | 9 (75.0) |
| 95% CI | 42.8–94.5 |
eGFR was calculated for pediatric patients aged 2 years through 18 years
CI confidence interval, eGFR estimated glomerular filtration rate, LDH lactate dehydrogenase, TMA thrombotic microangiopathy