Baseline patient characteristics
| Baseline characteristic . | Caplacizumab cohort (n = 77) . | Non-caplacizumab cohort (n = 78) . | P . |
|---|---|---|---|
| Female, N (%) | 58/77 (75%) | 61/78 (78%) | .6711 |
| Age, mean ± SD, y | 47.1 ± 14 | 46.5 ± 14 | .833 |
| First episode iTTP, N (%) | 50/77 (65%) | 72/78 (92%) | <.001 |
| Symptoms/signs, N (%) | |||
| Anemic syndrome∗ | 41/77 (53.2%) | 36/78 (46.2%) | .3772 |
| Neurologic involvement | 43/77 (55.8%) | 47/78 (60.3%) | .4510 |
| Neurologic focal signs† | 22/43 (51.2%) | 16/47 (34.0%) | |
| Headache | 11/43 (25.6%) | 15/47 (31.9%) | |
| Others | 11/43 (25.6%) | 10/47 (21.3%) | |
| Elevated troponin | 22/44 (50%) | NA | |
| Hemorrhagic diathesis‡ | 45/77 (58.4%) | 23/78 (29.5%) | <.001 |
| Mucocutaneous hemorrhage | 39/45 (86.7%) | 18/23(78.3%) | .010 |
| Menorrhagia | 5/46 (10.9%) | 0/23 (0%) | |
| Others | 1/45 (2.2%) | 5/23 (21.7%) | |
| Renal insufficiency/failure§ | 19/77 (24.7%) | 19/76 (25.0%) | .9629 |
| Laboratory parameters at diagnosis, median (IQR) [range] | |||
| Hemoglobin, g/dL | 8.7 (7.2-11.1) [5.7-16.6] | 8.7 (6.85-10.3) [5.7-14.6] | .219 |
| Platelets, ×109/L | 12 (8-20) [3-94] | 12 (8-17.5) [3-57] | .802 |
| WBC, ×109/L | 8.19 (6.7-11.4) [3.2-23] | 9.1 (7.13-12.09) [3.2-23.3] | .226 |
| Indirect bilirubin, mg/dL | 1.61 (0.995-2.35) [ 0.2-8] | NA | |
| LDH, U/L | 1040 (607-1546) [169-7334] | 1086 (715.5-1550) [291-7334] | .497 |
| Creatinine, mg/dL | 0.95 (0.73-1.2) [0.45-2.78] | 0.92 (0.72-1.3) [0.54-5.49] | .803 |
| ADAMTS13, % | 0 (0-0.5) [0-13] | 0 (0-0.5) [0-19] | .954 |
| ADAMTS13 inhibitor, BU | 3.3 (7-20.93) [0-112.2] | NA | |
| Baseline characteristic . | Caplacizumab cohort (n = 77) . | Non-caplacizumab cohort (n = 78) . | P . |
|---|---|---|---|
| Female, N (%) | 58/77 (75%) | 61/78 (78%) | .6711 |
| Age, mean ± SD, y | 47.1 ± 14 | 46.5 ± 14 | .833 |
| First episode iTTP, N (%) | 50/77 (65%) | 72/78 (92%) | <.001 |
| Symptoms/signs, N (%) | |||
| Anemic syndrome∗ | 41/77 (53.2%) | 36/78 (46.2%) | .3772 |
| Neurologic involvement | 43/77 (55.8%) | 47/78 (60.3%) | .4510 |
| Neurologic focal signs† | 22/43 (51.2%) | 16/47 (34.0%) | |
| Headache | 11/43 (25.6%) | 15/47 (31.9%) | |
| Others | 11/43 (25.6%) | 10/47 (21.3%) | |
| Elevated troponin | 22/44 (50%) | NA | |
| Hemorrhagic diathesis‡ | 45/77 (58.4%) | 23/78 (29.5%) | <.001 |
| Mucocutaneous hemorrhage | 39/45 (86.7%) | 18/23(78.3%) | .010 |
| Menorrhagia | 5/46 (10.9%) | 0/23 (0%) | |
| Others | 1/45 (2.2%) | 5/23 (21.7%) | |
| Renal insufficiency/failure§ | 19/77 (24.7%) | 19/76 (25.0%) | .9629 |
| Laboratory parameters at diagnosis, median (IQR) [range] | |||
| Hemoglobin, g/dL | 8.7 (7.2-11.1) [5.7-16.6] | 8.7 (6.85-10.3) [5.7-14.6] | .219 |
| Platelets, ×109/L | 12 (8-20) [3-94] | 12 (8-17.5) [3-57] | .802 |
| WBC, ×109/L | 8.19 (6.7-11.4) [3.2-23] | 9.1 (7.13-12.09) [3.2-23.3] | .226 |
| Indirect bilirubin, mg/dL | 1.61 (0.995-2.35) [ 0.2-8] | NA | |
| LDH, U/L | 1040 (607-1546) [169-7334] | 1086 (715.5-1550) [291-7334] | .497 |
| Creatinine, mg/dL | 0.95 (0.73-1.2) [0.45-2.78] | 0.92 (0.72-1.3) [0.54-5.49] | .803 |
| ADAMTS13, % | 0 (0-0.5) [0-13] | 0 (0-0.5) [0-19] | .954 |
| ADAMTS13 inhibitor, BU | 3.3 (7-20.93) [0-112.2] | NA | |
BU, Bethesda unit; LDH, lactate dehydrogenase; NA, not available; SD, standard deviation; WBC, white blood cells.
Boldface means statistical significant.
Includes fatigue, weakness, and dizziness symptoms.
Includes stroke, cardiovascular events, and transient ischemic attacks.
Required only 1 red blood cell transfusion and stopping caplacizumab treatment for 48 hours in 1 patient with rectal bleeding due to gut polyposis. The event remitted and did not require vWF or other treatments.
Defined as creatinine level >1.5 mg/dL.