Table 3.
Demographics and disease characteristics at baseline and discontinuation for 61 patients discontinuing eculizumab in clinical trials
| Discontinuation with subsequent TMA event | Discontinuation without subsequent TMA event | All discontinued patients | |
|---|---|---|---|
| (n = 12) | (n = 49) | (n = 61) | |
| Age at parent study baseline (years), median (range) | 19.5 (0.0–80.0) | 27.0 (0.0–68.0) | 26.0 (0.0–80.0) |
| Female, n (%) | 6 (50) | 30 (61) | 36 (59) |
| Identified complement mutation or autoantibody, n (%) | 7 (58) | 24 (49) | 31 (51) |
| Factor H mutation | 5 (42) | 9 (18) | 14 (23) |
| Time from TMA manifestation to start of eculizumab in parent trial (months), median (range) | 0.7 (0.0–19.1) | 0.8 (0.0–36.6) | 0.8 (0.0–36.6) |
| Time from diagnosis to start of eculizumab in parent trial (months), median (range) | 23.5 (0.0–112.5) | 1.0 (0.0–288.0) | 1.4 (0.0–288.0) |
| Duration of eculizumab treatment before discontinuation (weeks), median (range) | 19 (1–116) | 48 (1–231) | 27 (1–231) |
| Time to TMA manifestation after discontinuation (weeks), median (min–max) | 13 (4–127) | – | – |
| Follow-up time after discontinuation (weeks), median (min, max) | 14 (4–151) | 24 (0–145) | 24 (0–151) |
| eGFR (mL/min/1.73 m2), median (range) | |||
| At parent study baseline | 22.8 (10.0–105.5) | 15.7 (5.3–102.0) | 19.1 (5.3–105.5) |
| At discontinuation | 36.5 (10.1–151.3) | 42.9 (6.6–126.7) | 41.5 (6.6–151.3) |
| Dialysis, n (%) | |||
| At parent study baseline | 4 (33) | 20 (41) | 24 (39) |
| At discontinuation | 0 (0) | 12 (25) | 12 (20) |
| Kidney transplant before start of parent study, n (%) | 3 (25) | 13 (27) | 16 (26) |
eGFR, estimated glomerular filtration rate; TMA, thrombotic microangiopathy.