Comparison of presentation characteristics and outcomes in caplacizumab cohort vs a precaplacizumab historical control
| Presentation characteristics and outcomes . | Standard-treatment cohort* (n = 39) . | Caplacizumab cohort (n = 85) . | P† . |
|---|---|---|---|
| Age, mean (range), y | 45 (15-93) | 46 (3-82) | .76 |
| Females | 31 (80) | 56 (66) | .18 |
| Platelet count on admission, median (IQR), ×109/L | 10 (6-20) | 13 (9-21) | .51 |
| Elevated cardiac troponin | 25 (64) | 67 (79) | .12 |
| Elevated serum creatinine | 10 (26) | 35 (41) | .08 |
| Neurological symptoms at presentation | 29 (74) | 56 (66) | .74 |
| ADAMTS13 activity <10 IU/dL | 39 (100) | 84 (99) | >.99 |
| Patients achieving platelet count normalization | 39 (100) | 81 (95) | .31 |
| Time from first PEX to normalization of platelet count, median (IQR), d | 6 (4-10) | 4 (3-8)‡ | .011 |
| Days of PEX, median (IQR) | 9 (8-16) | 7 (5-14) | .007 |
| Adjuvant therapy with rituximab | 34 (87) | 84 (99) | >.99 |
| Patients requiring intubation | 4 (10) | 22 (26) | .06 |
| Length of hospitalization, median (IQR), d | 14 (9-17) | 12 (8-24) | .62 |
| Patients with bleeding post admission | 0 (0) | 15 (18) | .003 |
| Patients with VTE during admission | 2 (5) | 4 (5) | >.99 |
| TTP-related death | 0 (0) | 5 (6) | .32 |
| Presentation characteristics and outcomes . | Standard-treatment cohort* (n = 39) . | Caplacizumab cohort (n = 85) . | P† . |
|---|---|---|---|
| Age, mean (range), y | 45 (15-93) | 46 (3-82) | .76 |
| Females | 31 (80) | 56 (66) | .18 |
| Platelet count on admission, median (IQR), ×109/L | 10 (6-20) | 13 (9-21) | .51 |
| Elevated cardiac troponin | 25 (64) | 67 (79) | .12 |
| Elevated serum creatinine | 10 (26) | 35 (41) | .08 |
| Neurological symptoms at presentation | 29 (74) | 56 (66) | .74 |
| ADAMTS13 activity <10 IU/dL | 39 (100) | 84 (99) | >.99 |
| Patients achieving platelet count normalization | 39 (100) | 81 (95) | .31 |
| Time from first PEX to normalization of platelet count, median (IQR), d | 6 (4-10) | 4 (3-8)‡ | .011 |
| Days of PEX, median (IQR) | 9 (8-16) | 7 (5-14) | .007 |
| Adjuvant therapy with rituximab | 34 (87) | 84 (99) | >.99 |
| Patients requiring intubation | 4 (10) | 22 (26) | .06 |
| Length of hospitalization, median (IQR), d | 14 (9-17) | 12 (8-24) | .62 |
| Patients with bleeding post admission | 0 (0) | 15 (18) | .003 |
| Patients with VTE during admission | 2 (5) | 4 (5) | >.99 |
| TTP-related death | 0 (0) | 5 (6) | .32 |
Unless otherwise noted, data are n (%).
VTE, venous thromboembolism.
Standard treatment cohort is from 39 consecutive cases from unpublished UK TTP Registry data from 2014 to 2018.
P values were calculated using the Student t test/χ2 test for parametric data and the Mann-Whitney U test for nonparametric data.
Based on 81 of 85 patients. Four of 85 patients did not achieve a normal platelet count (by 30 days, post-PEX discontinuation); 1 case due to a concomitant diagnosis of immune thrombocytopenia with a history of chronic low-grade thrombocytopenia, 1 case whose platelets returned to normal levels by day 43, and 2 cases with multiple organ failure leading to death.