Characteristics of 19 children and 36 adult patients with aHUS who discontinued eculizumab and were included in study
| . | n (%) . | ||
|---|---|---|---|
| Age <18 y (n = 19)* . | Age ≥18 y (n = 36) . | All (n = 55) . | |
| Sex | |||
| Female | 7 (37) | 17 (47) | 24 (44) |
| Male | 12 (63) | 19 (53) | 31 (56) |
| Complement gene variants | 8 (42) | 20 (55) | 28 (51) |
| CFH | 1 (5) | 5 (14) | 6 (11) |
| MCP | 5 (26)† | 7 (19) | 12 (22) |
| CFI | 0 (0) | 6 (17) | 6 (11) |
| C3 | 0 (0) | 2 (6) | 2 (5) |
| Combined | 2 (11) | 0 (3) | 2 (4) |
| No variant/positive anti–factor H antibodies | 4 (21) | 0 (0) | 4 (7) |
| No variant /no anti–factor H antibodies | 7 (37) | 16 (44) | 23 (42) |
| >1 aHUS episode before inclusion in study‡ | 4 (21) | 4 (11) | 9 (16) |
| At aHUS onset§ | |||
| Serum creatinine, μmol/L | |||
| Mean | 361‖ | 454¶ | 421 |
| Range | 54-1920 | 91-1660 | 54-1920] |
| Requirement for dialysis | 8 (42)‖ | 16 (44%)‖ | 24 (43%) |
| Extrarenal manifestation | 10 (52) | 14 (40) | 24 (43%)# |
| Neurological manifestation | 4 (21) | 7 (20) | 11 (20) |
| Cardiac manifestation | 6 (31.5) | 4 (11) | 10 (18.5) |
| Other | 6 (31.5)** | 8 (23%)†† | 14 (25) |
| At eculizumab discontinuation (inclusion) | |||
| Duration of eculizumab treatment, mo | |||
| Mean | 13.9 | 17.9 | 16.5 |
| Range | 0.95-57.4 | 4.2-59.3 | 0.95-59 |
| Serum creatinine, μmol/L | |||
| Mean | 50 | 124 | 97 |
| Range | 26-134 | 61-305 | 26-305 |
| eGFR, mL/min/1.73 m2 | |||
| Mean | 112 | 62 | 80 |
| Range | 55-169 | 19-129 | 19-169 |
| eGFR, 30-60 mL/min/1.73 m2 | 1 (5%) | 16 (44) | 17 (30) |
| eGFR, 15-29 mL/min/1.73 m2 | 0 | 4 (11) | 4 (7) |
| Urinary protein/creatinine ratio, g/mmol | |||
| Mean | 0.18 | 0.06# | 0.10 |
| Range | 0-3 | 0-0.38 | 0-3 |
| Plasma C3 level <660 mg/L | 0 | 5/35 (14)‖ | 5/53 (9) |
| sC5b-9 ≥300 ng/mL | 11/18 (61)‖ | 23/35 (66) | 34/54 (63) |
| During follow-up | |||
| Duration of follow-up after eculizumab discontinuation, mo | |||
| Mean | 19.5 | 20 | 19.8 |
| Range | 5.4-24 | 1.6-24 | 5.4-24 |
| Patients with aHUS relapse | 6 (30)‡‡ | 7 (19) | 13 (23) |
| Time between eculizumab discontinuation and aHUS relapse, mo | |||
| Mean | 12.3 | 8.1 | 10.2 |
| Range | 5.4-20.6 | 1.6-22.1 | 1.6-22.1 |
| At last follow-up | |||
| Serum creatinine, μmol/L | |||
| Mean | 52 | 147 | 113 |
| Range | 25-144 | 58-881 | 25-881 |
| eGFR, mL/min/1.73 m2 | |||
| Mean | 123 | 58 | 81 |
| Range | 43-199 | 6-128 | 6-199 |
| eGFR, 30-60 mL/min/1.73 m2 | 1 (5%) | 17 (47%) | 18 (32%) |
| eGFR, 15-29 mL/min/1.73 m2 | 0 | 4 (11%) | 4 (7%) |
| eGFR, <15 mL/min/1.73 m2 | 0 | 1 (3%) | 1 (2%) |
| Urinary protein/creatinine ratio, g/mmol | |||
| Mean | 0.10 | 0.05 | 0.07 |
| Range | 0-1.60 | 0-0.44 | 0-1.60 |
| . | n (%) . | ||
|---|---|---|---|
| Age <18 y (n = 19)* . | Age ≥18 y (n = 36) . | All (n = 55) . | |
| Sex | |||
| Female | 7 (37) | 17 (47) | 24 (44) |
| Male | 12 (63) | 19 (53) | 31 (56) |
| Complement gene variants | 8 (42) | 20 (55) | 28 (51) |
| CFH | 1 (5) | 5 (14) | 6 (11) |
| MCP | 5 (26)† | 7 (19) | 12 (22) |
| CFI | 0 (0) | 6 (17) | 6 (11) |
| C3 | 0 (0) | 2 (6) | 2 (5) |
| Combined | 2 (11) | 0 (3) | 2 (4) |
| No variant/positive anti–factor H antibodies | 4 (21) | 0 (0) | 4 (7) |
| No variant /no anti–factor H antibodies | 7 (37) | 16 (44) | 23 (42) |
| >1 aHUS episode before inclusion in study‡ | 4 (21) | 4 (11) | 9 (16) |
| At aHUS onset§ | |||
| Serum creatinine, μmol/L | |||
| Mean | 361‖ | 454¶ | 421 |
| Range | 54-1920 | 91-1660 | 54-1920] |
| Requirement for dialysis | 8 (42)‖ | 16 (44%)‖ | 24 (43%) |
| Extrarenal manifestation | 10 (52) | 14 (40) | 24 (43%)# |
| Neurological manifestation | 4 (21) | 7 (20) | 11 (20) |
| Cardiac manifestation | 6 (31.5) | 4 (11) | 10 (18.5) |
| Other | 6 (31.5)** | 8 (23%)†† | 14 (25) |
| At eculizumab discontinuation (inclusion) | |||
| Duration of eculizumab treatment, mo | |||
| Mean | 13.9 | 17.9 | 16.5 |
| Range | 0.95-57.4 | 4.2-59.3 | 0.95-59 |
| Serum creatinine, μmol/L | |||
| Mean | 50 | 124 | 97 |
| Range | 26-134 | 61-305 | 26-305 |
| eGFR, mL/min/1.73 m2 | |||
| Mean | 112 | 62 | 80 |
| Range | 55-169 | 19-129 | 19-169 |
| eGFR, 30-60 mL/min/1.73 m2 | 1 (5%) | 16 (44) | 17 (30) |
| eGFR, 15-29 mL/min/1.73 m2 | 0 | 4 (11) | 4 (7) |
| Urinary protein/creatinine ratio, g/mmol | |||
| Mean | 0.18 | 0.06# | 0.10 |
| Range | 0-3 | 0-0.38 | 0-3 |
| Plasma C3 level <660 mg/L | 0 | 5/35 (14)‖ | 5/53 (9) |
| sC5b-9 ≥300 ng/mL | 11/18 (61)‖ | 23/35 (66) | 34/54 (63) |
| During follow-up | |||
| Duration of follow-up after eculizumab discontinuation, mo | |||
| Mean | 19.5 | 20 | 19.8 |
| Range | 5.4-24 | 1.6-24 | 5.4-24 |
| Patients with aHUS relapse | 6 (30)‡‡ | 7 (19) | 13 (23) |
| Time between eculizumab discontinuation and aHUS relapse, mo | |||
| Mean | 12.3 | 8.1 | 10.2 |
| Range | 5.4-20.6 | 1.6-22.1 | 1.6-22.1 |
| At last follow-up | |||
| Serum creatinine, μmol/L | |||
| Mean | 52 | 147 | 113 |
| Range | 25-144 | 58-881 | 25-881 |
| eGFR, mL/min/1.73 m2 | |||
| Mean | 123 | 58 | 81 |
| Range | 43-199 | 6-128 | 6-199 |
| eGFR, 30-60 mL/min/1.73 m2 | 1 (5%) | 17 (47%) | 18 (32%) |
| eGFR, 15-29 mL/min/1.73 m2 | 0 | 4 (11%) | 4 (7%) |
| eGFR, <15 mL/min/1.73 m2 | 0 | 1 (3%) | 1 (2%) |
| Urinary protein/creatinine ratio, g/mmol | |||
| Mean | 0.10 | 0.05 | 0.07 |
| Range | 0-1.60 | 0-0.44 | 0-1.60 |
Normal values: urinary protein/creatinine ratio, <0.05 g/mmol; plasma C3, 660-1250 mg/L; sC5b-9, <300 ng/mL.
One child was included twice in the study during 2 distinct periods of eculizumab treatment.
Two patients (including child with familial aHUS) had persistently low CD46 expression on granulocytes without genetic molecular mechanism identified.
aHUS episode outside a period of eculizmab treatment.
At onset of the last aHUS episode before eculizumab discontinuation.
Data missing for 1 patient.
Data missing for 3 patients.
Data missing for 2 patients.
Six patients had 7 extrarenal manifestations: digestive (n = 4), cutaneous (n = 2), and vascular (n = 1).
Eight patients had 13 extrarenal manifestations: digestive (n = 6), cutaneous (n = 3), vascular (n = 2), hepatic (n = 1), and hematological (n = 1).
Child included twice experienced 2 aHUS relapses.