Participant characteristics
| Characteristic . | Eculizumab-treated cohort (n = 243), n (%) . | Control cohort (n = 279), n (%) . |
|---|---|---|
| Age at first presentation, y | ||
| Median | 25 | 22 |
| Range | 0-80 | 0-79 |
| Sex | ||
| Male | 98 (41) | 135 (48) |
| Female | 145 (59) | 144 (52) |
| Plasma exchange | ||
| Yes | 130 (53) | 114 (38) |
| No | 94 (39) | 67 (23) |
| Data not available | 20 (8) | 116 (39) |
| Complement gene mutation | 90 (37) | 279 (100) |
| CFH | 33 (37∗) | 131 (47) |
| CFHR1:CFH hybrid | 1 (1∗) | 11 (4) |
| CFI | 6 (7∗) | 19 (7) |
| CD46 | 23 (26∗) | 61 (22) |
| C3 | 10 (11∗) | 24 (9) |
| CFB | 1 (1∗) | 0 |
| Factor H autoantibody positive | 11 (12∗) | 29 (10) |
| Combined mutation(s)/autoantibody | 5 (6∗) | 4 (1) |
| No complement mutation/autoantibody | 102 (42); 24 (10) with VUS | 0 |
| Not CaHUS | 51 (21) | NA |
| Eculizumab-treated CaHUS cohort (n = 192), n (%) | ||
| Trigger, n = 189† | 59 (31) | |
| Infection | 31 (16) | |
| Pregnancy | 18 (10) | |
| Other | 12 (6) | |
| None | 128 (68) | |
| Extrarenal manifestations, n = 191† | ||
| Neurological§ | 22 (12) | |
| Cardiac|| | 8 (4) | |
| Pancreatitis | 4 (2) | |
| Other¶ | 2 (1) | |
| None | 155 (81) | |
| Prodromal diarrhea, n = 180† | 45 (25) | |
| Laboratory variables at presentation mean (range) | ||
| Serum creatinine (μmol/L), n = 187† | 488 (18-2435) | |
| eGFR (ml/min per 1.73 m2) | 21 (2-118) | |
| Hemoglobin (g/L), n = 175† | 80 (38-142) | |
| Platelet count (×109/L), n = 185† | 69 (4-329) | |
| Lactate dehydrogenase (IU), n = 177† | 2373 (290-19130) | |
| C-reactive protein (mg/L), n = 140† | 44 (0.5-309) | |
| Urine protein:creatinine ratio (mg/mmol), n = 90† | 1381 (0-17520) | |
| C3 (g/L) n = 182† | 0.93 (0.04-2.31) | |
| Presenting blood pressure | ||
| Adults: systolic blood pressure, mean (range), mmHg, n = 92† | 175 (100-262) (88% hypertensive‡) | |
| Children: % hypertensive, n = 51† | 30 (59%‡) |
| Characteristic . | Eculizumab-treated cohort (n = 243), n (%) . | Control cohort (n = 279), n (%) . |
|---|---|---|
| Age at first presentation, y | ||
| Median | 25 | 22 |
| Range | 0-80 | 0-79 |
| Sex | ||
| Male | 98 (41) | 135 (48) |
| Female | 145 (59) | 144 (52) |
| Plasma exchange | ||
| Yes | 130 (53) | 114 (38) |
| No | 94 (39) | 67 (23) |
| Data not available | 20 (8) | 116 (39) |
| Complement gene mutation | 90 (37) | 279 (100) |
| CFH | 33 (37∗) | 131 (47) |
| CFHR1:CFH hybrid | 1 (1∗) | 11 (4) |
| CFI | 6 (7∗) | 19 (7) |
| CD46 | 23 (26∗) | 61 (22) |
| C3 | 10 (11∗) | 24 (9) |
| CFB | 1 (1∗) | 0 |
| Factor H autoantibody positive | 11 (12∗) | 29 (10) |
| Combined mutation(s)/autoantibody | 5 (6∗) | 4 (1) |
| No complement mutation/autoantibody | 102 (42); 24 (10) with VUS | 0 |
| Not CaHUS | 51 (21) | NA |
| Eculizumab-treated CaHUS cohort (n = 192), n (%) | ||
| Trigger, n = 189† | 59 (31) | |
| Infection | 31 (16) | |
| Pregnancy | 18 (10) | |
| Other | 12 (6) | |
| None | 128 (68) | |
| Extrarenal manifestations, n = 191† | ||
| Neurological§ | 22 (12) | |
| Cardiac|| | 8 (4) | |
| Pancreatitis | 4 (2) | |
| Other¶ | 2 (1) | |
| None | 155 (81) | |
| Prodromal diarrhea, n = 180† | 45 (25) | |
| Laboratory variables at presentation mean (range) | ||
| Serum creatinine (μmol/L), n = 187† | 488 (18-2435) | |
| eGFR (ml/min per 1.73 m2) | 21 (2-118) | |
| Hemoglobin (g/L), n = 175† | 80 (38-142) | |
| Platelet count (×109/L), n = 185† | 69 (4-329) | |
| Lactate dehydrogenase (IU), n = 177† | 2373 (290-19130) | |
| C-reactive protein (mg/L), n = 140† | 44 (0.5-309) | |
| Urine protein:creatinine ratio (mg/mmol), n = 90† | 1381 (0-17520) | |
| C3 (g/L) n = 182† | 0.93 (0.04-2.31) | |
| Presenting blood pressure | ||
| Adults: systolic blood pressure, mean (range), mmHg, n = 92† | 175 (100-262) (88% hypertensive‡) | |
| Children: % hypertensive, n = 51† | 30 (59%‡) |
Percentage of those with CaHUS and a mutation or FHAA.
n refers to number of individuals for whom data were available.
Hypertension defined by >140mmHg systolic in adults and >95th centile in children, according to the fourth report on diagnosis, evaluation, and treatment of high blood pressure in children and adolescents.
Extrarenal manifestations: Neurological manifestations reported were seizures, n = 12; stroke, n = 7; hemiparesis, n = 3; agitation/confusion, n = 3; encephalopathy, n = 2; cortical blindness, n = 1; and reduced conscious level, n = 1 (in some individuals >1 was reported).
Cardiac manifestations were left ventricular systolic dysfunction, n = 5, and cardiomyopathy, n = 2.
Other manifestations reported were liver failure and retinopathy.