Table 2.
Characteristics of the eculizumab-treated cohort
| Mutation/autoantibody |
|||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| All CaHUS | CFH | CFHR1 | CFI | CD46 | C3 | CFB | FHAA | Combined | Nmd | Not CaHUS | |
| n = 243 | 192 | 33 | 1 | 6 | 23 | 10 | 1 | 11 | 5 | 102 | 51 |
| Incidence rate (per million per y) | 0.41 | 0.06 | 0.003 | 0.01 | 0.04 | 0.02 | 0.003 | 0.02 | 0.008 | 0.24 | NR |
| Age at presentation, y; median (range) | 25 (0-80) | 21 (1-50) | NR 25 |
42 (4-67) | 20 (1-47) | 16 (1-51) | NR 1 |
16 (1-69) | 16 (2-28) | 33 (1-80) | 23 (1-76) |
| Sex (% female) | 61 | 70 | NR (F) | 71 | 52 | 70 | NR (F) | 36 | 100 | 59 | 51 |
| Trigger, n (%) | 59 (31.1) | 13 (39) | 0 | 1 (17) | 7 (30) | 1 (10) | 1 | 6 (55) | 0 | 30 (29) | NR |
| Infection | 29 (15.1) | 4 | 0 | 1 | 4 | 1 | 0 | 5 | 0 | 14 | NR |
| Pregnancy | 18 (9.5) | 5 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 12 | NR |
| Diarrheal prodrome, n (%) | 45 (25) | 8 (24) | 0 | 0 | 1 (4) | 4 (40) | 0 | 1 (9) | 1 (20) | 30 (29) | 12 (27) |
| Extrarenal manifestations, n (%) | 54 (19) | 4 (12) | 0 | 1 (17) | 3 (13) | 1 (10) | 0 | 2 (18) | 0 | 25 (24) | 18 (40) |
| Neurological | 22 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 21 | 13 |
| Cardiac | 8 | 4 | 0 | 1 | 1 | 1 | 0 | 0 | 0 | 1 | 2 |
| Pancreatitis | 4 | 0 | 0 | 0 | 2 | 0 | 0 | 1 | 0 | 1 | 1 |
| Platelet count at presentation, ×109/L; mean (range) | 69 (4-329) | 104 (31-329) | NR 35 |
76 (25-112) | 43 (9-116) | 60 (19-157) | NR 112 |
53 (6-96) | 67 (26-109) | 65 (4-197) | 73 (12-336) |
| Creatinine, μmol/L; mean (range) | 488 (18-2435) | 660 (50-1989) | NR 630 |
366 (86-652) | 470 (79-1377) | 465 (37-1709) | NR 59 |
292 (61-1131) | 625 (94-1172) | 466 (18-2435) | 386 (19-1859) |
| CRP, mg/L; mean (range) | 44 (0.5-309) | 31 (1-246) | NA | 38 (5-160) | 34 (3-182) | 45 (1-136) | NR 13 |
10 (1-34) | 18 (7-28) | 48 (0.5-309) | 83 (1-366) |
| C3, g/L; mean (range) | 0.93 (0.06-2.31) | 0.78 (0.06-2.31) | NR 0.94 |
0.88 (0.63-1.09) | 1.02 (0.55-1.54) | 0.64 (0.08-1.02) | NR 0.85 |
0.75 (0.06-1.23) | 1.07 (0.28-1.67) | 1.0 (0.22-1.99) | 1.0 (0.36-1.79) |
| Adult systolic blood pressure, mmHg; mean (range) | 174 | 180 (117-229) | NR | 194 (157-230) | 165 (110-220) | 168 (145-180) | NR | 152 (144-160) | 147 (140-153) | 176 (137-262) | 167 (165-270) |
| Children; % hypertensive | 59 | 67 | NR | NR | 67 | 40 | NR | 38 | NR | 62 | 70 |
CRP, C-reactive protein; NR, not relevant (missing or insufficient data).