Table 2.
Published series of eculizumab discontinuation in aHUS
| Series | n | Median duration of therapy (range or IQR), mo | Median follow-up (since cessation) (mo) | Relapse rate, n (%) | Outcome of relapses |
|---|---|---|---|---|---|
| Ardissino et al10 (Italy) | 16 | 4.3 (0.5-14.4) | 13.1 (1.2-28.2) | 5 (31.3) | Retreated with eculizumab, no progressive renal injury |
| Sheerin et al9 (UK) | 14 | — | — | 3 (21.4) | Retreated without chronic sequelae |
| Wijnsma et al15 (Netherlands) | 17 | 3.8 (2.8-5.8) | 27.4 (7.8-42) | 5 (29.4) | Retreated with eculizumab, no progressive renal injury |
| Fakhouri et al22 (France) | 38 | 17.5 (2-50) | 22 (5-43) | 12 (31.6) | Eculizumab resumed with no significant change in GFR |
| Menne et al 13 | 42 | 19.6 (0.2-86.9) | — | 11 (26.1)* | 40% had a decline in renal function after discontinuing but eGFR remained >60 mL/min/1.73 m2 |
| Fakhouri et al16 (France) | 55 | Mean 16.5 (0.95, 59) | 24 | 13 (23) | 11 of 13 regained baseline renal function |
| Current study | 25 | 2.4 (IQR 1.1, 9.7) | 27 (IQR 5, 50) | 5 (20) | Four salvaged with prompt eculizumab therapy. One patient died during recurrent TMA (nonadherent with therapy) |
—, not reported.
*
In this study, 21 (50%) restarted therapy for TMA relapse/renal impairment (n = 11), preparation for a kidney transplant (n = 5), short discontinuation period because of change in dose or missed doses (n = 2), administrative reasons (n = 2), and multiple serious adverse effects and a change in dosing (n = 1).