Long-term outcomes
| Outcome . | Eculizumab-treated cohort (%) . | Control cohort (%) . | P value . |
|---|---|---|---|
| Five-year ESKD-free survival | |||
| Total CaHUS | 78 | 39.5 | .000 |
| Total CaHUS with mutation or FHAA | 85.5 | 39.5 | .000 |
| CFH mutation | 78 | 17 | .000 |
| CFHR1 hybrid | NR∗ | 15 | na |
| CFI mutation | 83.5 | 16 | .004 |
| CD46 mutation | 95.6 | 87.5 | .274 |
| C3 mutation | 80 | 25 | .007 |
| CFB mutation | NR∗ | NR | na |
| Factor H autoantibody | 100 | 71 | .057 |
| Combined mutation(s)/autoantibody | 75 | 20 | .005 |
| VUS | 79 | NR | na |
| No mutation | 65 | NR | na |
| Not CaHUS | 57 | NR | na |
| Duration of follow-up, median (range) d | 1514 (2-3720) = 4.2 y | ||
| Renal function at last follow-up, number (%) n = 153 CaHUS | |||
| CKD stage G1 | 52 (34) | ||
| CKD stage G2 | 30 (20) | ||
| CKD stage G3a | 14 (9) | ||
| CKD stage G3b | 9 (6) | ||
| CKD stage G4 | 10 (7) | ||
| CKD stage G5 | 3 (2) | ||
| CKD stage G5D | 23 (14) | ||
| Underwent transplantation | 13 (9) | ||
| Preemptive eculizumab | 10 of 13 | ||
| Functioning transplantation | 12 of 13 |
| Outcome . | Eculizumab-treated cohort (%) . | Control cohort (%) . | P value . |
|---|---|---|---|
| Five-year ESKD-free survival | |||
| Total CaHUS | 78 | 39.5 | .000 |
| Total CaHUS with mutation or FHAA | 85.5 | 39.5 | .000 |
| CFH mutation | 78 | 17 | .000 |
| CFHR1 hybrid | NR∗ | 15 | na |
| CFI mutation | 83.5 | 16 | .004 |
| CD46 mutation | 95.6 | 87.5 | .274 |
| C3 mutation | 80 | 25 | .007 |
| CFB mutation | NR∗ | NR | na |
| Factor H autoantibody | 100 | 71 | .057 |
| Combined mutation(s)/autoantibody | 75 | 20 | .005 |
| VUS | 79 | NR | na |
| No mutation | 65 | NR | na |
| Not CaHUS | 57 | NR | na |
| Duration of follow-up, median (range) d | 1514 (2-3720) = 4.2 y | ||
| Renal function at last follow-up, number (%) n = 153 CaHUS | |||
| CKD stage G1 | 52 (34) | ||
| CKD stage G2 | 30 (20) | ||
| CKD stage G3a | 14 (9) | ||
| CKD stage G3b | 9 (6) | ||
| CKD stage G4 | 10 (7) | ||
| CKD stage G5 | 3 (2) | ||
| CKD stage G5D | 23 (14) | ||
| Underwent transplantation | 13 (9) | ||
| Preemptive eculizumab | 10 of 13 | ||
| Functioning transplantation | 12 of 13 |
P value refers to the log-rank (Mantel-Cox) comparison.
CKD based on Kidney Disease Improving Global Outcomes grade (G).
na, not applicable; NR (not relevant; insufficient number).
Single patient free from ESKD.