Patient demographics, treatment characteristics, health care resource utilization outcomes, and efficacy and safety outcomes for patients with iTTP who had a first diagnosis or relapse episode
| . | First diagnoses (n = 25) . | Relapse episodes (n = 14) . |
|---|---|---|
| Sex, n (%) | ||
| Male | 14 (56.0) | 4 (28.6) |
| Female | 11 (44.0) | 10 (71.4) |
| Time between hospital admission and start of daily TPE, mean (SD), d | 2.3 (4.6) | 0.4 (0.5) |
| Duration of daily TPE, mean (SD), d | 6.1 (1.8) | 5.9 (2.0) |
| Time between TPE initiation and caplacizumab initiation, mean (SD), d | 0.4 (1.0) | 0.0 (0.4) |
| Corticosteroid use, n (%) | ||
| Yes | 25 (100.0) | 14 (100.0) |
| Rituximab use, n (%) | ||
| Yes | 24 (96.0) | 9 (64.3) |
| From hospitalization and request to check ADAMTS13 activity, d | ||
| n | 25 | 13 |
| Number of days, mean (SD) | 1.2 (2.3) | −0.2 (2.1) |
| From ADAMTS13 result to caplacizumab initiation, d | ||
| n | 24 | 11 |
| Number of days, mean (SD) | 0.4 (5.0)∗ | −0.7 (1.6)∗ |
| Caplacizumab treatment duration, d | ||
| Mean (SD) | 39.6 (15.3) | 32.7 (10.1) |
| Median (Q1, Q3) | 37.0 (33.0, 46.0) | 33.5 (28.0, 36.8) |
| Time from initiation of caplacizumab to sustained platelet count, d | ||
| n | 25 | 13 |
| Number of d, mean (SD) | 4.6 (3.3) | 6.0 (5.9) |
| Duration of hospital stay, d | ||
| n | 25 | 13 |
| Number of d, mean (SD) | 16.0 (10.3) | 9.1 (2.9) |
| Duration of intensive care unit stay, d | ||
| n | 15 | 4 |
| Number of d, mean (SD) | 6.2 (4.0) | 3.8 (1.0) |
| Proportion of patients with an exacerbation, n (%) | ||
| Yes | 2 (8.0) | 0 |
| Proportion of patients with a relapse, n (%) | ||
| Yes | 4 (16.0) | 2 (14.3) |
| Proportion of patients with refractory disease, n (%) | ||
| Yes | 0 | 0 |
| Mortality, n (%) | ||
| Yes | 0 | 0 |
| Proportion of patients with a TEE,† n (%) | ||
| Yes | 7 (28.0)‡ | 1 (7.1)§ |
| Proportion of patients with an adverse event, n (%) | 6 (24.0) | 3 (21.4) |
| Related to caplacizumab | 4 (16.0) | 1 (7.1) |
| Proportion of patients with a bleeding event, n (%) | 3 (12.0) | 2 (14.3) |
| Related to caplacizumab | 3 (12.0) | 1 (7.1) |
| . | First diagnoses (n = 25) . | Relapse episodes (n = 14) . |
|---|---|---|
| Sex, n (%) | ||
| Male | 14 (56.0) | 4 (28.6) |
| Female | 11 (44.0) | 10 (71.4) |
| Time between hospital admission and start of daily TPE, mean (SD), d | 2.3 (4.6) | 0.4 (0.5) |
| Duration of daily TPE, mean (SD), d | 6.1 (1.8) | 5.9 (2.0) |
| Time between TPE initiation and caplacizumab initiation, mean (SD), d | 0.4 (1.0) | 0.0 (0.4) |
| Corticosteroid use, n (%) | ||
| Yes | 25 (100.0) | 14 (100.0) |
| Rituximab use, n (%) | ||
| Yes | 24 (96.0) | 9 (64.3) |
| From hospitalization and request to check ADAMTS13 activity, d | ||
| n | 25 | 13 |
| Number of days, mean (SD) | 1.2 (2.3) | −0.2 (2.1) |
| From ADAMTS13 result to caplacizumab initiation, d | ||
| n | 24 | 11 |
| Number of days, mean (SD) | 0.4 (5.0)∗ | −0.7 (1.6)∗ |
| Caplacizumab treatment duration, d | ||
| Mean (SD) | 39.6 (15.3) | 32.7 (10.1) |
| Median (Q1, Q3) | 37.0 (33.0, 46.0) | 33.5 (28.0, 36.8) |
| Time from initiation of caplacizumab to sustained platelet count, d | ||
| n | 25 | 13 |
| Number of d, mean (SD) | 4.6 (3.3) | 6.0 (5.9) |
| Duration of hospital stay, d | ||
| n | 25 | 13 |
| Number of d, mean (SD) | 16.0 (10.3) | 9.1 (2.9) |
| Duration of intensive care unit stay, d | ||
| n | 15 | 4 |
| Number of d, mean (SD) | 6.2 (4.0) | 3.8 (1.0) |
| Proportion of patients with an exacerbation, n (%) | ||
| Yes | 2 (8.0) | 0 |
| Proportion of patients with a relapse, n (%) | ||
| Yes | 4 (16.0) | 2 (14.3) |
| Proportion of patients with refractory disease, n (%) | ||
| Yes | 0 | 0 |
| Mortality, n (%) | ||
| Yes | 0 | 0 |
| Proportion of patients with a TEE,† n (%) | ||
| Yes | 7 (28.0)‡ | 1 (7.1)§ |
| Proportion of patients with an adverse event, n (%) | 6 (24.0) | 3 (21.4) |
| Related to caplacizumab | 4 (16.0) | 1 (7.1) |
| Proportion of patients with a bleeding event, n (%) | 3 (12.0) | 2 (14.3) |
| Related to caplacizumab | 3 (12.0) | 1 (7.1) |
Clinical response was defined as sustained platelet count of ≥150 × 109/L and LDH <1.5 × ULN. Clinical remission was defined as platelet count remains ≥150 × 109/L and LDH <1.5 × ULN for ≥30 days after stopping TPE. Exacerbation was defined as a platelet count reduction to <150 × 109/L and LDH increase within 30 days of stopping TPE, after a clinical response. Relapse was defined as platelet count reduction to <150 × 109/L after a clinical remission. Refractory disease was defined by the lack of doubling of platelet count after 4 days of treatment and an LDH level that remained above ULN.14,15
LDH, lactate dehydrogenase; Q1/3, quartile 1/3; TEE, thromboembolic event; ULN, upper limit of normal.
In 4 patients, caplacizumab treatment was started 1 day before ADAMTS13 test results were available. In 5 patients, treatment was started 3 to 8 days before.
For patients with a first diagnosis, there were on average 2.6 (SD, 4.6) days between the reporting of a TEE and the start of caplacizumab. The TEE reported in the relapse subgroup was reported >2 months after the start of caplacizumab. The majority of patients showed several cardiovascular risk factors probably explaining the thrombotic events. Most of these events were considered as a complication of iTTP, and none were considered as a complication of caplacizumab treatment.
Lung embolism, cerebral thrombosis, myocardial ischemia, jugular vein thrombosis, renal infarction, splenic infarction, and renal vein thrombosis.
Deep vein thrombosis in legs and lung embolism.