Table 1.
Pooled outcomes from randomized trials of caplacizumaba
| Clinical importance rankingb | Outcome | Caplacizumab | Placebo | Relative effect: odds ratio (95% CI) | Absolute effect: per 1000 iTTP patients (95% CI) |
|---|---|---|---|---|---|
| 1 | All-cause mortality | 1/108 (0.9%) | 5/112 (4.5%) | 0.27 (0.05-1.34) | −32 (−42 to 14) |
| 2 | All cardiovascular events | 4/106 (3.8%) | 3/110 (2.7%) | 1.39 (0.31-6.23) | 10 (−19 to 121) |
| 3 | Neurologic events | 3/106 (2.8%) | 4/110 (3.6%) | 0.77 (0.17-3.47) | −8 (−30 to 79) |
| 4 | Platelet count recovery | 96/108 (88.9%) | 92/112 (82.1%) | 1.71 (0.8-3.63) | −66 (−121 to 35) |
| 5 | Relapse (at 1 mo) | 14/108 (13.0%) | 0/112 (0.0%) | 9.08 (3.06-26.89) | Not estimable (0 events in placebo) |
| 5 | Relapse (at 12 mo)c | 11/36 (30.6%) | 3/37 (8.1%) | 4.17 (1.31-13.27) | 188 (23 to 458) |
| 6 | Time to relapse | Not reported | Not reported | N/A | N/A |
| 7 | Acute renal injury/dialysis | Not reported | Not reported | N/A | N/A |
| 8 | Days in hospitald | Mean (95% CI) difference: 4.5 (7.32-1.68) days lower with caplacizumab | N/A | N/A | |
| 8 | Days of TPE | Mean (95% CI) difference: 3.69 (5.35-2.02) days lower with caplacizumab | N/A | N/A | |
| 9 | Exacerbation | 6/108 (5.6%) | 39/112 (34.8%) | 0.17 (0.09-0.32) | −265 (−302 to −202) |
| 10 | Normal ADAMTS13 level after TPE | Not reported | Not reported | N/A | N/A |
| N/A | Serious adverse events | 36/106 (34.0%) | 24/110 (21.8%) | 1.84 (1.01-3.34) | 121 (2 to 264) |
a
As reported in the 2020 ISTH iTTP Treatment Guidelines supplement.
b
As defined by the 2020 ISTH iTTP Treatment Guidelines.
c
Data only available for TITAN trial.
d
Data only available for HERCULES trial.