Table 1.
Demographic and clinical characteristics at presentation by eculizumab discontinuation status
| Variable | Total cohort (N = 31) | Continued eculizumab (n = 6) | Stopped eculizumab (n = 25) | P |
|---|---|---|---|---|
| Age, median (IQR), y | 44 (25, 53.5) | 32 (25, 48) | 44 (32, 52) | .408 |
| Female sex | 80.6 | 66.6 | 80 | .483 |
| Race: White | 64.5 | 83.3 | 60 | .283 |
| Race: African American | 35.5 | 16.7 | 40 | |
| Clinical presentation | ||||
| Neurologic | ||||
| Seizure | 12.9 | 0 | 16.0 | .294 |
| Vision loss | 12.9 | 33.3 | 8.0 | .096 |
| Unconsciousness | 6.5 | 0 | 8.0 | .474 |
| Headache | 38.7 | 66.6 | 32.0 | .117 |
| Encephalopathy | 19.4 | 0 | 24.0 | .181 |
| Stroke | 9.7 | 0 | 12.0 | .372 |
| Gastrointestinal | ||||
| Hemorrhage | 3.2 | 0 | 4.0 | .618 |
| Epigastric pain | 12.9 | 33.3 | 8.0 | .096 |
| Pancreatitis | 9.7 | 0 | 12.0 | .372 |
| Transaminitis | 12.9 | 16.7 | 12.0 | .759 |
| Vomiting | 45.2 | 66.7 | 40.0 | .239 |
| Ischemic hepatitis | 3.2 | 0 | 4.0 | .618 |
| Abdominal pain | 38.7 | 33.3 | 40.0 | .763 |
| Respiratory | ||||
| Pulmonary edema | 9.7 | 0 | 12.0 | .372 |
| Pleural effusion | 6.5 | 16.7 | 4.0 | .257 |
| Other respiratory failure | 12.9 | 0 | 16.0 | .294 |
| New or worsening hypertension | 41.9 | 50 | 40 | .655 |
| Laboratory studies at presentation | ||||
| Hemoglobin, median (IQR), mg/dL | 8.8 (7.6, 9.8) | 9.1(8.3, 9.5) | 8.8 (7.4, 10.1) | .673 |
| Platelet count, median (IQR), ×109/L | 42 (18, 73) | 33 (13, 41) | 45 (19, 84) | .267 |
| Serum creatinine, median (IQR), mg/dL | 3.5 (2.8, 6.2) | 2.7 (5.5, 9.0) | 3.5 (2.9, 6.1) | .610 |
| Lactate dehydrogenase, median (IQR), U/L | 1329 (858, 2545) | 5571 (3240, 6870) | 1282 (800, 2501) | .036 |
| Trigger identified* | 67.7 | 66.6 | 68.0 | .950 |
| Complement variant present (n = 22 sequenced), % (n/N) | 63.6 (14/22) | 60 (3/5) | 64.7 (11/17) | .743 |
| Treatments | ||||
| Plasma exchange received | 58.1 | 83.3 | 52.0 | .162 |
| Time to starting eculizumab, median (IQR), d | 2 (2, 5) | 2.5 (2, 5) | 4 (2,5) | .487 |
Data are percentages unless otherwise noted.
*
Triggers included infections (n = 7), autoimmune disease flares (n = 4), pregnancy and cesarean section (n = 1), surgery (n = 1), cancer/chemotherapy (n = 4), and other inflammatory disorders (n = 4).