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Table 4 TMA manifestation rates

From: Outcomes in patients with atypical hemolytic uremic syndrome treated with eculizumab in a long-term observational study

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%
  1. N/A not applicable; TMA thrombotic microangiopathy
  2. aDuring off-treatment periods compared with on-treatment periods. bDefined as age < 18 years at time of diagnosis