Table 1.

Pooled outcomes from randomized trials of caplacizumaba 

Clinical importance rankingb OutcomeCaplacizumabPlaceboRelative effect: odds ratio (95% CI)Absolute effect: per 1000 iTTP patients (95% CI)
All-cause mortality 1/108 (0.9%) 5/112 (4.5%) 0.27 (0.05-1.34) −32 (−42 to 14) 
All cardiovascular events 4/106 (3.8%) 3/110 (2.7%) 1.39 (0.31-6.23) 10 (−19 to 121) 
Neurologic events 3/106 (2.8%) 4/110 (3.6%) 0.77 (0.17-3.47) −8 (−30 to 79) 
Platelet count recovery 96/108 (88.9%) 92/112 (82.1%) 1.71 (0.8-3.63) −66 (−121 to 35) 
Relapse (at 1 mo) 14/108 (13.0%) 0/112 (0.0%) 9.08 (3.06-26.89) Not estimable
(0 events in placebo) 
Relapse (at 12 mo)c  11/36 (30.6%) 3/37 (8.1%) 4.17 (1.31-13.27) 188 (23 to 458) 
Time to relapse Not reported Not reported N/A N/A 
Acute renal injury/dialysis Not reported Not reported N/A N/A 
Days in hospitald  Mean (95% CI) difference: 4.5 (7.32-1.68) days lower with caplacizumab N/A N/A 
Days of TPE Mean (95% CI) difference: 3.69 (5.35-2.02) days lower with caplacizumab N/A N/A 
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) 
Clinical importance rankingb OutcomeCaplacizumabPlaceboRelative effect: odds ratio (95% CI)Absolute effect: per 1000 iTTP patients (95% CI)
All-cause mortality 1/108 (0.9%) 5/112 (4.5%) 0.27 (0.05-1.34) −32 (−42 to 14) 
All cardiovascular events 4/106 (3.8%) 3/110 (2.7%) 1.39 (0.31-6.23) 10 (−19 to 121) 
Neurologic events 3/106 (2.8%) 4/110 (3.6%) 0.77 (0.17-3.47) −8 (−30 to 79) 
Platelet count recovery 96/108 (88.9%) 92/112 (82.1%) 1.71 (0.8-3.63) −66 (−121 to 35) 
Relapse (at 1 mo) 14/108 (13.0%) 0/112 (0.0%) 9.08 (3.06-26.89) Not estimable
(0 events in placebo) 
Relapse (at 12 mo)c  11/36 (30.6%) 3/37 (8.1%) 4.17 (1.31-13.27) 188 (23 to 458) 
Time to relapse Not reported Not reported N/A N/A 
Acute renal injury/dialysis Not reported Not reported N/A N/A 
Days in hospitald  Mean (95% CI) difference: 4.5 (7.32-1.68) days lower with caplacizumab N/A N/A 
Days of TPE Mean (95% CI) difference: 3.69 (5.35-2.02) days lower with caplacizumab N/A N/A 
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.

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