Table 4.
Studies describing the outcome of eculizumab regimen discontinuation in aHUS patients
| Study group | Number of patients | Median duration of eculizumab treatment | Number of patients who discontinued treatment with eculizumab | Median duration of follow-up after stopping treatment | Number of patients who relapsed after discontinuation | Proportion of relapsed patients with mutations and type of mutations | Outcome |
|---|---|---|---|---|---|---|---|
| Ardissino et al84 (2015) | 16 | 4.3 (0.5–14.4) months | 16 | 0.7–40 months | 5/16 (31.2%) | 5/5 (CFH-4, CFI-1) | All patients who relapsed were restarted on eculizumab and had rapid improvement of renal function. |
| Fakhouri et al83(2017) | 108 | 17.5 months | 38 | 22 months | 12/38 (31.5%) | 12/12 (CFH-8, MCP-4) | All patients had rapid remission of TMA after restarting eculizumab with no long-term sequelae. |
| Meril et al47(2017) | 17 | 3 months | 15 | 10.2 months | 3/15 (20%) | 3/3 (CFH-2, ADAMTS 13–1) | 2 of 3 patients restarted eculizumab and renal function returned to baseline. |
| Macia et al82(data from clinical trials) (2017) | 130 | 6.3 months | 61 | 6.3 months | 12/61 (19.6) | 7/12 (CHF-5, others-2) | 3 patients progressed to ESRD with one patient requiring dialysis despite restarting eculizumab |
| Wijnsma et al85 (2017) | 20 | 3.8 months | 17 | 27.4 months | 5/17 (29.4%) | 5/5 (CFH-4, C3-1) | No chronic sequelae after restart of eculizumab were noted. |
| Sheerin et al86 (2016) | 43 | 6 months | 14 | 12 months | 3/14(21%) | 2/3 (CFH-1, CD46-1) | Complete recovery was noted in all three patients after reintroducing eculizumab. |