Outcome parameters and safety analysis of the TPE-free and TPE cohorts treated with frontline caplacizumab
| Parameter . | TPE-free cohort (n = 42) . | TPE cohort (n = 59) . | P value . |
|---|---|---|---|
| Primary outcome | |||
| Median time to platelet count normalization (range; IQR), d | 3 (1-12; 2-4) | 4 (2-27; 3-5) | .31 |
| Key secondary outcomes | |||
| Patients achieving a clinical response without requiring subsequent TPE in the TPE-free cohort, n (%) | 38 (90.5) | 57 (96.6) | .23 |
| Patients achieving a clinical response (including patients with subsequent TPE in the TPE-free cohort), n (%) | 41 (97.6) | 57 (96.6) | >.99 |
| Patients with a clinical exacerbation, n (%) | 2 (4.8) | 9 (15.3) | .12 |
| Patients refractory to therapy, n (%) | 0 (0) | 1 (1.7) | >.99 |
| TTP-related death, n (%) | 0 (0) | 1 (1.7) | >.99 |
| Median time to platelet count doubling (range; IQR) | 1 (1-7; 1-2) | 1 (1-4; 1-2) | .88 |
| Other secondary outcomes | |||
| Median time to recovery of ADAMTS13 activity to ≥20% after treatment initiation (IQR), d | 25 (13-33) | 37 (19-51) | .01 |
| Patients without confirmed recovery of ADAMTS13 activity to ≥20% at end of follow-up, n (%) | 4 (9.5) | 10 (16.9) | .39 |
| Median days in hospital (IQR) | 11 (5-15) | 14 (9-21) | .02 |
| Patients admitted to ICU, n (%) | 11 (26.2) | 42 (71.2) | <.01 |
| Data missing, n (%) | 5 (11.9) | 8 (13.6) | |
| Median days on ICU, if admitted (IQR) | 3 (2.5-4.5) | 4 (2-6) | .53 |
| Safety | |||
| Patients with at least 1 reported complication during overall follow-up period, n (%) | 11 (26.2) | 16 (27.1) | >.99 |
| Reported complications on TPE, n (%) | 0 (0) | 4 (6.7) | .14 |
| Any bleeding, n (%) | 5 (11.9) | 2 (3.4) | .12 |
| Major bleeding according to ISTH, n (%) | 2 (4.8) | 0 (0) | .17 |
| Parameter . | TPE-free cohort (n = 42) . | TPE cohort (n = 59) . | P value . |
|---|---|---|---|
| Primary outcome | |||
| Median time to platelet count normalization (range; IQR), d | 3 (1-12; 2-4) | 4 (2-27; 3-5) | .31 |
| Key secondary outcomes | |||
| Patients achieving a clinical response without requiring subsequent TPE in the TPE-free cohort, n (%) | 38 (90.5) | 57 (96.6) | .23 |
| Patients achieving a clinical response (including patients with subsequent TPE in the TPE-free cohort), n (%) | 41 (97.6) | 57 (96.6) | >.99 |
| Patients with a clinical exacerbation, n (%) | 2 (4.8) | 9 (15.3) | .12 |
| Patients refractory to therapy, n (%) | 0 (0) | 1 (1.7) | >.99 |
| TTP-related death, n (%) | 0 (0) | 1 (1.7) | >.99 |
| Median time to platelet count doubling (range; IQR) | 1 (1-7; 1-2) | 1 (1-4; 1-2) | .88 |
| Other secondary outcomes | |||
| Median time to recovery of ADAMTS13 activity to ≥20% after treatment initiation (IQR), d | 25 (13-33) | 37 (19-51) | .01 |
| Patients without confirmed recovery of ADAMTS13 activity to ≥20% at end of follow-up, n (%) | 4 (9.5) | 10 (16.9) | .39 |
| Median days in hospital (IQR) | 11 (5-15) | 14 (9-21) | .02 |
| Patients admitted to ICU, n (%) | 11 (26.2) | 42 (71.2) | <.01 |
| Data missing, n (%) | 5 (11.9) | 8 (13.6) | |
| Median days on ICU, if admitted (IQR) | 3 (2.5-4.5) | 4 (2-6) | .53 |
| Safety | |||
| Patients with at least 1 reported complication during overall follow-up period, n (%) | 11 (26.2) | 16 (27.1) | >.99 |
| Reported complications on TPE, n (%) | 0 (0) | 4 (6.7) | .14 |
| Any bleeding, n (%) | 5 (11.9) | 2 (3.4) | .12 |
| Major bleeding according to ISTH, n (%) | 2 (4.8) | 0 (0) | .17 |
All parameters are presented per episode. The TPE-free cohort comprised 42 acute iTTP episodes in 41 patients. The TPE cohort comprised 59 acute iTTP episodes in 59 patients.
ISTH, International Society on Thrombosis and Haemostasis.