Table 2.
Demographic and baseline clinical characteristics in the parent studies
| Characteristic | Never Discontinued (n = 51) | Discontinued (n = 42) | All Patients (N = 93) | |
|---|---|---|---|---|
| Reinitiated (n = 21) | Not Reinitiated (n = 21) | |||
| Age, at first eculizumab dose, median (range), years | 23.0 (0.0, 63.0) | 21.0 (0.0, 65.0) | 30.0 (0.0, 80.0) | 21.0 (0.0, 80.0) |
| Age < 12 years, n (%) | 15 (29) | 5 (24) | 6 (29) | 26 (28) |
| Female, n (%) | 30 (59) | 11 (52) | 16 (76) | 57 (61) |
| Genetic or autoimmune complement abnormality, n (%)a | 31 (61) | 14 (67) | 10 (48) | 55 (59) |
| No. of TMA manifestations prior to first eculizumab dose, n (%) | ||||
| 1 | 30 (59) | 13 (62) | 16 (76) | 59 (63) |
| ≥ 2 | 21 (41) | 8 (38) | 5 (24) | 34 (37) |
| Time from most recent TMA manifestation to the first eculizumab dose, median (range), months | 1.8 (0.0, 47.4) | 0.4 (0.1, 37.8) | 0.6 (0.0, 19.2) | 0.9 (0.0, 47.4) |
| Time from aHUS diagnosis to first eculizumab dose, median (range), months | 18.0 (0.0, 313.3) | 3.0 (0.0, 191.4) | 0.5 (0.0, 178.1) | 4.0 (0.0, 313.3) |
| No. of PE/PI sessions at latest TMA manifestation before the first eculizumab dose, median (range) | 13.0 (0.0, 230.0) | 7.0 (0.0, 121.0) | 7.0 (0.0, 64.0) | 10.5 (0.0, 230.0) |
| Patients with dialysis at baseline of parent study, n (%) | 18 (35) | 11 (52) | 9 (43) | 38 (41) |
| Patients with renal transplant prior to first eculizumab dose, n (%) | 14 (28) | 4 (19) | 5 (24) | 23 (25) |
aHUS atypical hemolytic uremic syndrome; CFB complement factor B; CFH complement factor H; CFI complement factor I; MCP membrane cofactor protein; PE/PI plasma exchange/plasma infusion; TMA thrombotic microangiopathy
aIncludes pathogenic variants in C3, CD46 (MCP), CFB, CFH, and CFI, as well as CFH autoantibodies as determined at enrollment in parent studies