Comparison of eculizumab and ravulizumab in 3 prospective noncontrolled trials in adults with aHUS
| . | Eculizumab (n = 17) (Legendre et al)5 n, (%) . | Eculizumab (n = 41) (Fakhouri et al)6 n, (%) . | Ravulizumab (n = 58) (Rondeau et al)46 n, (%) . |
|---|---|---|---|
| Age, >65 y | Not available (range, 17-68) | 2 (5) | 8 (14.3) >60 |
| ≥1 variant in complement gene (or anti–factor H antibodies) | 13 (76) | 21 (51) | 8 (20.5) |
| Recurrence of aHUS | 10 (59) | 11 (26.8) | 3 (5.4) |
| Kidney transplant recipients | 7 (41) | 9 (22) | 8 (14.3) |
| Dialysis requirement at inclusion | 6 (35) | 24 (59) | 29 (51.8) |
| “Complete TMA response”∗ | 11 (6%) | 30 (73) | 30 (53.6) |
| “Modified TMA response”† | Not available | 23 (56) | 30 (53.6) |
| Patients requiring dialysis at inclusion and weaned from dialysis at 6 mo | 5/6 (83) | 20 (83) | 17 (58.6) |
| Patients requiring dialysis at 6 mo | 1 (6) | 6 (15) | 18 (32) |
| Patients requiring dialysis at last follow-up | 1 (6) | ||
| Death | 0 | 0 | 4 (7) |
| . | Eculizumab (n = 17) (Legendre et al)5 n, (%) . | Eculizumab (n = 41) (Fakhouri et al)6 n, (%) . | Ravulizumab (n = 58) (Rondeau et al)46 n, (%) . |
|---|---|---|---|
| Age, >65 y | Not available (range, 17-68) | 2 (5) | 8 (14.3) >60 |
| ≥1 variant in complement gene (or anti–factor H antibodies) | 13 (76) | 21 (51) | 8 (20.5) |
| Recurrence of aHUS | 10 (59) | 11 (26.8) | 3 (5.4) |
| Kidney transplant recipients | 7 (41) | 9 (22) | 8 (14.3) |
| Dialysis requirement at inclusion | 6 (35) | 24 (59) | 29 (51.8) |
| “Complete TMA response”∗ | 11 (6%) | 30 (73) | 30 (53.6) |
| “Modified TMA response”† | Not available | 23 (56) | 30 (53.6) |
| Patients requiring dialysis at inclusion and weaned from dialysis at 6 mo | 5/6 (83) | 20 (83) | 17 (58.6) |
| Patients requiring dialysis at 6 mo | 1 (6) | 6 (15) | 18 (32) |
| Patients requiring dialysis at last follow-up | 1 (6) | ||
| Death | 0 | 0 | 4 (7) |
A comparison of the 2 available types of C5 blockers is limited by the significant differences in the characteristics of patients included in 3 large studies with these 2 drugs. Indeed, patients included in the ravulizumab trial may not be fully representative of patients with aHUS (older age, low frequency of complement gene variants), an additional illustration of the importance of the definition of aHUS. Furthermore, end points also differ between studies.
Different definitions in the 3 trials: Legendre et al,5 platelet count and LDH level normalization sustained for at least 2 consecutive measurements over a period of ≥4 weeks; Fakhouri et al,6 platelet count and LDH level normalization, kidney function preservation (<25% increase in serum creatinine); Rondeau et al,46 platelet count and LDH level normalization, kidney function improvement (>25% decrease in serum creatinine).
In Fakhouri et al,6 defined as hematologic response (platelet and LDH as earlier), and kidney function improvement (>25% decrease in creatinine level).