Summary of mortality in patients receiving caplacizumab
| . | Patient 1 . | Patient 2 . | Patient 3 . | Patient 4 . | Patient 5 . |
|---|---|---|---|---|---|
| Demographics | |||||
| Age, y | 53 | 35 | 81 | 67 | 33 |
| Sex | Female | Male | Female | Female | Male |
| Ethnicity | White | Asian | White | White | White |
| ADAMTS13 activity on admission, IU/dL | <5 | 0 | <5 | 0 | 0 |
| ADAMTS13 inhibitor on admission, IU/mL | >100 | >94 | >100 | 37 | 92 |
| Intubation during admission | Yes | Yes | Yes | No | Yes |
| Time from initiation of PEX to first-dose caplacizumab, d | 21 | 3 | 19 | 2 | 8 |
| Total duration of PEX, d | 22 | 9 | 31 | 5 | 12 |
| Total duration of caplacizumab, d* | 1 | 2 | 13 | 22 | 36 |
| Outcomes | |||||
| Time to platelet count normalization, d | 18 | N/A† | N/A† | 7 | 9 |
| Recovery of ADAMTS13 activity to >10% prior to death | No | No | Yes | No | No |
| Time from admission to death, d | 22 | 10 | 36 | 29 | 15 |
| Bleeding complications | No | Yes‡ | No | No | No |
| Cause of death§ | Severe progressive TTP | Refractory TTP | Concurrent mixed connective tissue disease and refractory TTP | TTP plus multiple comorbidities, palliative management | Bowel ischemia secondary to refractory TTP |
| . | Patient 1 . | Patient 2 . | Patient 3 . | Patient 4 . | Patient 5 . |
|---|---|---|---|---|---|
| Demographics | |||||
| Age, y | 53 | 35 | 81 | 67 | 33 |
| Sex | Female | Male | Female | Female | Male |
| Ethnicity | White | Asian | White | White | White |
| ADAMTS13 activity on admission, IU/dL | <5 | 0 | <5 | 0 | 0 |
| ADAMTS13 inhibitor on admission, IU/mL | >100 | >94 | >100 | 37 | 92 |
| Intubation during admission | Yes | Yes | Yes | No | Yes |
| Time from initiation of PEX to first-dose caplacizumab, d | 21 | 3 | 19 | 2 | 8 |
| Total duration of PEX, d | 22 | 9 | 31 | 5 | 12 |
| Total duration of caplacizumab, d* | 1 | 2 | 13 | 22 | 36 |
| Outcomes | |||||
| Time to platelet count normalization, d | 18 | N/A† | N/A† | 7 | 9 |
| Recovery of ADAMTS13 activity to >10% prior to death | No | No | Yes | No | No |
| Time from admission to death, d | 22 | 10 | 36 | 29 | 15 |
| Bleeding complications | No | Yes‡ | No | No | No |
| Cause of death§ | Severe progressive TTP | Refractory TTP | Concurrent mixed connective tissue disease and refractory TTP | TTP plus multiple comorbidities, palliative management | Bowel ischemia secondary to refractory TTP |
The duration of caplacizumab was calculated from initiation and until death or discontinuation prior to death.
Platelet count did not normalize prior to death.
Case 2 suffered extensive cerebral infarct 24 hours after admission. Four days later the patient became drowsy, with CT brain imaging showing subarachnoid hemorrhage leading to interruption of caplacizumab. Three days later there was further deterioration of glasgow coma scale (GCS) requiring intubation and repeat imaging. This showed multiple cerebral infarcts, deemed likely due to progressive TTP.
Cause of death for each case was determined based on information provided by the hematology clinician submitting the data.