Characteristics at inclusion and during follow-up of 13 patients who relapsed and 42 patients who did not relapse after eculizumab discontinuation
| . | n (%) . | P . | |
|---|---|---|---|
| Relapsing patients (n = 13)* . | Nonrelapsing patients (n = 42) . | ||
| Age, <18 y | 6 (46) | 13 (31) | .34 |
| Sex | .03 | ||
| Female | 9 (69) | 15 (36) | |
| Male | 4 (31) | 27 (64) | |
| >1 aHUS episode before inclusion in study† | 5 (38) | 3 (7) | .01 |
| Requirement for dialysis during last aHUS episode before eculizumab discontinuation | 2 (15) | 22 (52) | .02 |
| Extrarenal manifestation | 3 (23) | 21 (53)‡ | .06 |
| Duration of eculizumab treatment, mo | .28 | ||
| Mean | 23.3 | 14 | |
| Range | 3.3-59.3 | 0.95-57.4 | |
| Complement gene variants | 12 (92) | 16 (25) | .0009 |
| CFH | 3 (23) | 3 (7) | .1 |
| MCP | 6 (46) | 6 (14) | .02 |
| CFI | 1 (8) | 4 (9) | 1 |
| C3 | 1 (8) | 2 (5) | .5 |
| Combined | 1 (8) | 1 (2) | .4 |
| No variant/positive anti–factor H antibodies | 0 (0) | 4 (9) | .5 |
| No variant/no anti–factor H antibodies | 1 (7)§ | 22 (53) | .004 |
| No. of relapses after eculizumab discontinuation | 14 | — | |
| At eculizumab discontinuation (inclusion) | |||
| Serum creatinine, μmol/L | |||
| All | .12 | ||
| Mean | 82 | 102 | |
| Range | 32-245 | 26-305 | |
| Children | .15 | ||
| Mean | 39 | 56 | |
| Range | 32-52 | 26-134 | |
| Adults | .96 | ||
| Mean | 126 | 123 | |
| Range | 64-245 | 61-305 | |
| eGFR, mL/min/1.73 m2 | |||
| All | .57 | ||
| Mean | 87 | 78 | |
| Range | 27-169 | 19-130 | |
| Children | .19 | ||
| Mean | 123 | 106 | |
| Range | 79-169 | 55-124 | |
| Adults | .25 | ||
| Mean | 52 | 65 | |
| Range | 27-89 | 19-130 | |
| CKD stage 3 | 4 (30) | 13 (31) | .9 |
| CKD stage 4 | 1 (7) | 3 (7) | 1 |
| Urinary protein/creatinine ratio, g/mmol | |||
| All | .10 | ||
| Mean | 0.27 | 0.04‡ | |
| Range | 0.01-3 | 0-0.38 | |
| Children | .39 | ||
| Mean | 0.45 | 0.03 | |
| Range | 0.01-3.00 | 0.00-0.14 | |
| Adults | .07 | ||
| Mean | 0.10 | 0.05 | |
| Range | 0.01-0.22 | 0.00-0.38 | |
| Plasma C3 level <660 mg/L at inclusion | 2/12 (17) | 3/41 (7) | .31 |
| Plasma factor H level <70% at inclusion | 1/10 (10)‖ | 2/40 (5)‖ | .49 |
| sC5b-9 ≥300 ng/ml at inclusion | 11/12 (92)‖ | 23/41 (56)‖ | .04 |
| sC5b-9 at inclusion | .03 | ||
| Mean | 418 | 325 | |
| Range | 363-499 | 234-428 | |
| At last follow-up | |||
| Duration of follow-up, mo | |||
| Before relapse | |||
| All | <.0001 | ||
| Mean | 9.3 | 23 | |
| Range | 1.6-22.1 | 6.7-24 | |
| Children | <.0001 | ||
| Mean | 10.9 | 23.1 | |
| Range | 5.4-17.2 | 18.1-24 | |
| Adults | <.0001 | ||
| Mean | 8 | 22.9 | |
| Range | 1.6-22.1 | 6.7-24 | |
| After relapse | |||
| All | — | ||
| Mean | 13.2 | ||
| Range | 2.7-23.7 | ||
| Children | — | ||
| Mean | 10.9 | ||
| Range | 2.8-18.3 | ||
| Adults | — | ||
| Mean | 14.9 | ||
| Range | 2.7-23.7 | ||
| Serum creatinine, μmol/L | |||
| All | .21 | ||
| Mean | 135 | 107 | |
| Range | 32-881 | 25-287 | |
| Children | .19 | ||
| Mean | 41 | 59 | |
| Range | 32-53 | 25-144 | |
| Adults | .57 | ||
| Mean | 229 | 128 | |
| Range | 64-881 | 58-287 | |
| eGFR, mL/min/1.73 m2 | |||
| All | .70 | ||
| Mean | 89 | 79 | |
| Range | 6-199 | 21-194 | |
| Children | .48 | ||
| Mean | 131 | 118 | |
| Range | 84-199 | 43-194 | |
| Adults | .17 | ||
| Mean | 46 | 61 | |
| Range | 6-89 | 21-128 | |
| eGFR, <30 mL/min/1.73 m2 | |||
| All | 2 (14) | 3 (7) | .59 |
| Children | 0 | 0 | — |
| Adults | 2 (29) | 3 (10) | .24 |
| Urinary protein/creatinine ratio, g/mmol | |||
| All | .004 | ||
| Mean | 0.07 | 0.07 | |
| Range | 0.01-0.26 | 0.00-1.60 | |
| Children | .15 | ||
| Mean | 0.03 | 0.14 | |
| Range | 0.01-0.09 | 0.00-1.60 | |
| Adults | .004 | ||
| Mean | 0.12 | 0.04 | |
| Range | 0.05-0.26 | 0.00-0.44 | |
| . | n (%) . | P . | |
|---|---|---|---|
| Relapsing patients (n = 13)* . | Nonrelapsing patients (n = 42) . | ||
| Age, <18 y | 6 (46) | 13 (31) | .34 |
| Sex | .03 | ||
| Female | 9 (69) | 15 (36) | |
| Male | 4 (31) | 27 (64) | |
| >1 aHUS episode before inclusion in study† | 5 (38) | 3 (7) | .01 |
| Requirement for dialysis during last aHUS episode before eculizumab discontinuation | 2 (15) | 22 (52) | .02 |
| Extrarenal manifestation | 3 (23) | 21 (53)‡ | .06 |
| Duration of eculizumab treatment, mo | .28 | ||
| Mean | 23.3 | 14 | |
| Range | 3.3-59.3 | 0.95-57.4 | |
| Complement gene variants | 12 (92) | 16 (25) | .0009 |
| CFH | 3 (23) | 3 (7) | .1 |
| MCP | 6 (46) | 6 (14) | .02 |
| CFI | 1 (8) | 4 (9) | 1 |
| C3 | 1 (8) | 2 (5) | .5 |
| Combined | 1 (8) | 1 (2) | .4 |
| No variant/positive anti–factor H antibodies | 0 (0) | 4 (9) | .5 |
| No variant/no anti–factor H antibodies | 1 (7)§ | 22 (53) | .004 |
| No. of relapses after eculizumab discontinuation | 14 | — | |
| At eculizumab discontinuation (inclusion) | |||
| Serum creatinine, μmol/L | |||
| All | .12 | ||
| Mean | 82 | 102 | |
| Range | 32-245 | 26-305 | |
| Children | .15 | ||
| Mean | 39 | 56 | |
| Range | 32-52 | 26-134 | |
| Adults | .96 | ||
| Mean | 126 | 123 | |
| Range | 64-245 | 61-305 | |
| eGFR, mL/min/1.73 m2 | |||
| All | .57 | ||
| Mean | 87 | 78 | |
| Range | 27-169 | 19-130 | |
| Children | .19 | ||
| Mean | 123 | 106 | |
| Range | 79-169 | 55-124 | |
| Adults | .25 | ||
| Mean | 52 | 65 | |
| Range | 27-89 | 19-130 | |
| CKD stage 3 | 4 (30) | 13 (31) | .9 |
| CKD stage 4 | 1 (7) | 3 (7) | 1 |
| Urinary protein/creatinine ratio, g/mmol | |||
| All | .10 | ||
| Mean | 0.27 | 0.04‡ | |
| Range | 0.01-3 | 0-0.38 | |
| Children | .39 | ||
| Mean | 0.45 | 0.03 | |
| Range | 0.01-3.00 | 0.00-0.14 | |
| Adults | .07 | ||
| Mean | 0.10 | 0.05 | |
| Range | 0.01-0.22 | 0.00-0.38 | |
| Plasma C3 level <660 mg/L at inclusion | 2/12 (17) | 3/41 (7) | .31 |
| Plasma factor H level <70% at inclusion | 1/10 (10)‖ | 2/40 (5)‖ | .49 |
| sC5b-9 ≥300 ng/ml at inclusion | 11/12 (92)‖ | 23/41 (56)‖ | .04 |
| sC5b-9 at inclusion | .03 | ||
| Mean | 418 | 325 | |
| Range | 363-499 | 234-428 | |
| At last follow-up | |||
| Duration of follow-up, mo | |||
| Before relapse | |||
| All | <.0001 | ||
| Mean | 9.3 | 23 | |
| Range | 1.6-22.1 | 6.7-24 | |
| Children | <.0001 | ||
| Mean | 10.9 | 23.1 | |
| Range | 5.4-17.2 | 18.1-24 | |
| Adults | <.0001 | ||
| Mean | 8 | 22.9 | |
| Range | 1.6-22.1 | 6.7-24 | |
| After relapse | |||
| All | — | ||
| Mean | 13.2 | ||
| Range | 2.7-23.7 | ||
| Children | — | ||
| Mean | 10.9 | ||
| Range | 2.8-18.3 | ||
| Adults | — | ||
| Mean | 14.9 | ||
| Range | 2.7-23.7 | ||
| Serum creatinine, μmol/L | |||
| All | .21 | ||
| Mean | 135 | 107 | |
| Range | 32-881 | 25-287 | |
| Children | .19 | ||
| Mean | 41 | 59 | |
| Range | 32-53 | 25-144 | |
| Adults | .57 | ||
| Mean | 229 | 128 | |
| Range | 64-881 | 58-287 | |
| eGFR, mL/min/1.73 m2 | |||
| All | .70 | ||
| Mean | 89 | 79 | |
| Range | 6-199 | 21-194 | |
| Children | .48 | ||
| Mean | 131 | 118 | |
| Range | 84-199 | 43-194 | |
| Adults | .17 | ||
| Mean | 46 | 61 | |
| Range | 6-89 | 21-128 | |
| eGFR, <30 mL/min/1.73 m2 | |||
| All | 2 (14) | 3 (7) | .59 |
| Children | 0 | 0 | — |
| Adults | 2 (29) | 3 (10) | .24 |
| Urinary protein/creatinine ratio, g/mmol | |||
| All | .004 | ||
| Mean | 0.07 | 0.07 | |
| Range | 0.01-0.26 | 0.00-1.60 | |
| Children | .15 | ||
| Mean | 0.03 | 0.14 | |
| Range | 0.01-0.09 | 0.00-1.60 | |
| Adults | .004 | ||
| Mean | 0.12 | 0.04 | |
| Range | 0.05-0.26 | 0.00-0.44 | |
One child was included twice in study during 2 distinct periods of eculizumab treatment and experienced 2 relapses.
aHUS episode outside period of eculizmab treatment.
Data missing for 2 patients.
Patient was diagnosed with hereditary ADAMTS13 deficiency after completion of the study.
Data missing for 1 patient.