Demographic and baseline disease characteristics of the TPE-free and TPE cohorts treated with frontline caplacizumab
| Parameter . | TPE-free cohort (n = 42) . | TPE cohort (n = 59) . | P value . |
|---|---|---|---|
| Median age (range), y | 43 (20-83) | 47 (20-80) | .43 |
| Female/male sex | .82 | ||
| n (%) | 31 (75.6)/10 (24.4) | 40 (67.8)/17 (28.8) | |
| Data missing, n (%) | 0 (0) | 2 (3.4) | |
| Mean BMI (range), kg/m2 | 29 (17-50) | 28 (20-46) | .62 |
| First iTTP episode/iTTP relapse | .40 | ||
| n (%) | 25 (59.5)/17 (40.5) | 38 (64.4)/18 (30.5) | |
| Data missing, n (%) | 0 (0) | 3 (5.1) | |
| Median platelet count (range), × 109/L | 16 (4-127) | 12 (3-52) | .09 |
| Median LDH (range), U/L | 703 (214-2500) | 1052 (373-3467) | <.01 |
| Initial troponin | .17 | ||
| Elevated, n (%) | 16 (38.1) | 27 (45.8) | |
| Not elevated, n (%) | 11 (26.2) | 8 (13.6) | |
| Data missing, n (%) | 15 (35.7) | 24 (40.7) | |
| Median serum creatinine (range), mg/dL | 0.95 (0.57-5.30) | 1.07 (0.5-3.65) | .07 |
| ADAMTS13 activity <10%, n (%) | 42 (100) | 58 (98.3)∗ | >.99 |
| Glasgow coma scale, n (%) | .17 | ||
| 15 | 38 (90.5) | 43 (72.9) | |
| <15 | 4 (9.5) | 11 (18.6) | |
| Data missing | 0 (0) | 5 (8.5) | |
| Neurological symptoms upon admission, n (%) | |||
| Patients with at least 1 neurological symptom | 24 (57.1) | 26 (44.1) | .23 |
| Aphasia/dysarthria | 4 (9.5) | 8 (13.6) | |
| Blurred vision | 4 (9.5) | 3 (5.1) | |
| Coma | 0 (0) | 2 (3.4) | |
| Headache | 7 (16.7) | 4 (6.8) | |
| Focal deficiency | 4 (9.5) | 5 (8.5) | |
| Paresthesia | 4 (9.5) | 3 (5.1) | |
| Seizure | 1 (2.4) | 3 (5.1) | |
| Confusion | 8 (19.0) | 10 (16.9) | |
| French severity score, n (%) | .90 | ||
| Low (0-1) | 27 (64.3) | 30 (50.8) | |
| Intermediate (2) | 10 (23.8) | 15 (25.4) | |
| High (3-4) | 5 (11.9) | 6 (10.2) | |
| Data missing | 0 (0) | 8 (13.6) |
| Parameter . | TPE-free cohort (n = 42) . | TPE cohort (n = 59) . | P value . |
|---|---|---|---|
| Median age (range), y | 43 (20-83) | 47 (20-80) | .43 |
| Female/male sex | .82 | ||
| n (%) | 31 (75.6)/10 (24.4) | 40 (67.8)/17 (28.8) | |
| Data missing, n (%) | 0 (0) | 2 (3.4) | |
| Mean BMI (range), kg/m2 | 29 (17-50) | 28 (20-46) | .62 |
| First iTTP episode/iTTP relapse | .40 | ||
| n (%) | 25 (59.5)/17 (40.5) | 38 (64.4)/18 (30.5) | |
| Data missing, n (%) | 0 (0) | 3 (5.1) | |
| Median platelet count (range), × 109/L | 16 (4-127) | 12 (3-52) | .09 |
| Median LDH (range), U/L | 703 (214-2500) | 1052 (373-3467) | <.01 |
| Initial troponin | .17 | ||
| Elevated, n (%) | 16 (38.1) | 27 (45.8) | |
| Not elevated, n (%) | 11 (26.2) | 8 (13.6) | |
| Data missing, n (%) | 15 (35.7) | 24 (40.7) | |
| Median serum creatinine (range), mg/dL | 0.95 (0.57-5.30) | 1.07 (0.5-3.65) | .07 |
| ADAMTS13 activity <10%, n (%) | 42 (100) | 58 (98.3)∗ | >.99 |
| Glasgow coma scale, n (%) | .17 | ||
| 15 | 38 (90.5) | 43 (72.9) | |
| <15 | 4 (9.5) | 11 (18.6) | |
| Data missing | 0 (0) | 5 (8.5) | |
| Neurological symptoms upon admission, n (%) | |||
| Patients with at least 1 neurological symptom | 24 (57.1) | 26 (44.1) | .23 |
| Aphasia/dysarthria | 4 (9.5) | 8 (13.6) | |
| Blurred vision | 4 (9.5) | 3 (5.1) | |
| Coma | 0 (0) | 2 (3.4) | |
| Headache | 7 (16.7) | 4 (6.8) | |
| Focal deficiency | 4 (9.5) | 5 (8.5) | |
| Paresthesia | 4 (9.5) | 3 (5.1) | |
| Seizure | 1 (2.4) | 3 (5.1) | |
| Confusion | 8 (19.0) | 10 (16.9) | |
| French severity score, n (%) | .90 | ||
| Low (0-1) | 27 (64.3) | 30 (50.8) | |
| Intermediate (2) | 10 (23.8) | 15 (25.4) | |
| High (3-4) | 5 (11.9) | 6 (10.2) | |
| Data missing | 0 (0) | 8 (13.6) |
All parameters are presented per episode, except for sex. The TPE-free cohort comprised 42 acute iTTP episodes in 41 patients. The TPE cohort comprised 59 acute iTTP episodes in 59 patients.
BMI, body mass index.
One patient from the TPE cohort presented with an acute iTTP relapse, and the diagnosis of iTTP was based on a known history of iTTP, thrombocytopenia, and microangiopathic hemolytic anemia at baseline.