Table 3.
Studies describing a restrictive eculizumab regimen in aHUS patients
| Author and year of publication | Number of participants | Age (years) | Duration standard eculizumab therapy (months) | Number of participants with tapered therapy | Number of participants in whom therapy was discontinued | Follow-up period after therapy adjustment (months) | Recurrence, number (%) | Time until recurrence (months) | Outcome |
|---|---|---|---|---|---|---|---|---|---|
| Cugno et al. (2014) [49] | 18 | Mean 21 (range 2–40) | Up till 40 months | 18; interval was extended up to 4 weeks based on CH50 | 0 | Up till 43 months | 0 | NA | No chronic sequelae |
| Ardissino et al. (2014–2015) [50, 51] | 22 | 18 (1–53) | 4.3 months (0.5–14.4) | 0 | 16 | Up till 40 months | 5/16 (31%) | 1.2 months (0.7–16.3) | No chronic sequelae after restart of eculizumab. Serum creatinine and proteinuria returned to baseline values |
| Sheerin et al. (2016) [31] | 43 | Unknown | 6 months (0.5–8.5) | 0 | 12 | 12 months | 3/12 (25%) | 2.5 months (1.5–9) | Full renal recovery was seen in 1 patient. The remaining 2 patient were still dialysis dependent on time of withdrawal and presented with hemolysis and hyperkalaemia which resolved quickly with reintroduction of eculizumab. |
| Fakhouri et al. (2017) [52] | 108 | 30 (2–79) | 17.5 months (2–50) | 0 | 38 | 22 months (5–23) | 12/38 (31%) | 7.5 months (3–29) | No chronic sequelae after restart of eculizumab |
| Merril et al. (2017) [45] | 17 | 46 (19–69) | 3 months (0.5–18.2) | NA | 15 | 10.2 months (1.2–46.3) | 3/15 (20%) | 2 months (1.8–3.3) | 2 patients received eculizumab after which kidney function was restored. 1 patient died during PE for recurrence after non-adherence with antihypertensive drugs. |
| Macia et al. (2017) [53], summary of authors’ case reports | 6 | 37 (16–39) | 6 months (1–14) | 1; patient received 900 mg every 4 weeks | 5 | Unknown | 4/6 (67%) | 3 months (2–12) | Unknown |
| Wijnsma et al. (2017) [54] | 20 | 28 (1–62) | 3.8 months (1.3–14.7) | 5 | 15 | 27.4 months (6–47) | 5/20 (25%) | 7.5 months (1–12) | No chronic sequelae after restart of eculizumab. 1 relapse occurred during tapering eculizumab. |
| Ardissino et al. (2017) [37] | 47 | 25 (0.5–60) | 2.6 months (0.4–24.6) | 38 | 9 | 26.9 months (0.8–80.9) | 0 | NA | No chronic sequelae after restart of eculizumab. |
| Macia et al. (2017) [53]; summary of clinical series | 130 | 26 (0–80) | 6.3 months (0.2–53.7) | 0 | 61 | 5.6 months (0–35.1) | 12/61 (20%) | 3 months (1–29.5) | Limited data available. 1 patient progressed to ESRD despite re-initiation of therapy. |
Numbers are expressed as median (range) unless otherwise specified. The different cohort studies and case reports include patients with atypical hemolytic uremic syndrome (aHUS) after kidney transplantation and patients with aHUS due to auto-antibodies directed against complement component factor H
ESRD end-stage renal disease, NA not applicable, PE plasma exchange