Table 4 TMA manifestation rates
Patient Group/Subgroup | On-Treatment Period | Off-Treatment Period | Fold Change in Ratea | Percent Decrease on Versus off Treatment |
|---|---|---|---|---|
Overall | ||||
Patients | n = 82 | n = 42 | ||
Patients with TMA, n (%) | 2 (2) | 10 (24) | ||
Manifestations | 3 | 14 | ||
Total patient-years | 292.5 | 103.8 | ||
Rate per 100 patient-years | 1.0 | 13.5 | 13.5 | 93% |
Patients who never discontinued | ||||
Patients | n = 51 | N/A | ||
Patients with TMA, n (%) | 2 (4) | N/A | ||
Manifestations | 3 | N/A | ||
Total patient-years | 218.2 | N/A | ||
Rate per 100 patient-years | 1.4 | N/A | N/A | N/A |
Patients who discontinued eculizumab | ||||
Patients | n = 31 | n = 42 | ||
Patients with TMA, n (%) | 0 (0) | 10 (24) | ||
Manifestations | 0 | 14 | ||
Total patient-years | 74.2 | 103.8 | ||
Rate per 100 patient-years | 0.0 | 13.5 | N/A | 100% |
Genetic or autoimmune complement abnormality status | ||||
Patients with complement abnormality | n = 51 | n = 24 | ||
Patients with TMA, n (%) | 1 (2) | 7 (29) | ||
Manifestations | 2 | 9 | ||
Total patient-years | 188.3 | 50.1 | ||
Rate per 100 patient-years | 1.1 | 18.0 | 16.4 | 94% |
Patients without identified complement abnormality | n = 31 | n = 18 | ||
Patients with TMA, n (%) | 1 (3) | 3 (17) | ||
Manifestations | 1 | 5 | ||
Total patient-years | 104.1 | 53.7 | ||
Rate per 100 patient-years | 1.0 | 9.3 | 9.3 | 89% |
Age at diagnosis | ||||
Adult patients | n = 41 | n = 22 | ||
Patients with TMA, n (%) | 0 (0) | 3 (14) | ||
Manifestations | 0 | 5 | ||
Total patient-years | 140.5 | 56.2 | ||
Rate per 100 patient-years | 0.0 | 8.9 | N/A | 100% |
Pediatric patientsb | n = 41 | n = 20 | ||
Patients with TMA, n (%) | 2 (5) | 7 (35) | ||
Manifestations | 3 | 9 | ||
Total patient-years | 152.0 | 47.6 | ||
Rate per 100 patient-years | 2.0 | 18.9 | 9.5 | 89% |
History of TMA events | ||||
Single TMA | n = 51 | n = 29 | ||
Patients with TMA, n (%) | 2 (4) | 6 (21) | ||
Manifestations | 3 | 8 | ||
Total patient-years | 192.7 | 70.4 | ||
Rate per 100 patient-years | 1.6 | 11.4 | 7.1 | 86% |
Multiple TMA prior to initiation of eculizumab | n = 31 | n = 13 | ||
Patients with TMA, n (%) | 1 (3) | 4 (31) | ||
Manifestations | 1 | 6 | ||
Total patient-years | 99.8 | 33.4 | ||
Rate per 100 patient-years | 1.0 | 18.0 | 18.0 | 94% |
Transplant status | ||||
Transplanted kidney | n = 21 | n = 9 | ||
Patients with TMA, n (%) | 0 (0) | 0 (0) | ||
Manifestations | 0 | 0 | ||
Total patient-years | 76.0 | 24.3 | ||
Rate per 100 patient-years | 0.0 | 0.0 | N/A | N/A |
Native kidney | n = 61 | n = 33 | ||
Patients with TMA, n (%) | 2 (3) | 10 (30) | ||
Manifestations | 3 | 14 | ||
Total patient-years | 216.5 | 79.5 | ||
Rate per 100 patient-years | 1.4 | 17.6 | 12.6 | 92% |