Characteristics and outcomes for pediatric patients treated with caplacizumab
| Cases . | Patient 1 . | Patient 2 . | Patient 3 . | Patient 4 . |
|---|---|---|---|---|
| Demographics | ||||
| Age, y | 12 | 17 | 3 | 13 |
| Sex | Female | Female | Male | Male |
| Ethnicity | Black/Afro-Caribbean | White | Mixed | White |
| ADAMTS13 inhibitor on admission, U/mL | 11 | 30 | 56 | >80 |
| Platelet count on admission, ×109/L | 53 | 21 | 9 | 9 |
| Troponin elevation on admission | No | No | Yes | No |
| Creatinine elevation on admission | No | No | Yes | No |
| Neurological symptoms on admission | No | Yes* | Yes* | No |
| Intubation during admission | No | No | Yes† | No |
| Concurrent treatment | Steroid, rituximab | Steroid, rituximab | Steroid, rituximab | Steroid, rituximab, MMF |
| Time from initiation of PEX to first dose of caplacizumab, d | 0 | 8 | 1 | 7 |
| Total duration of PEX, d | 3 | 4 | 16 | 8 |
| Total duration of caplacizumab, d | 24 | 36 | 27 | 61 |
| ADAMTS13 activity at discontinuation of caplacizumab, IU/dL | 82.1 | 23.9 | 49.9 | 8.0 |
| Outcomes | ||||
| Time to platelet count normalization, d | 3 | 4 | 25 | 3 |
| Recurrence of TTP | No | No | No | No |
| Recovery of creatinine | N/A | N/A | Yes | N/A |
| Resolution of neurological symptoms | N/A | No‡ | Yes | N/A |
| Length of stay, d | 10 | 14 | 28 | 13 |
| Bleeding complications | No | No | No§ | No |
| Outcome | Survived | Survived | Survived | Survived |
| Cases . | Patient 1 . | Patient 2 . | Patient 3 . | Patient 4 . |
|---|---|---|---|---|
| Demographics | ||||
| Age, y | 12 | 17 | 3 | 13 |
| Sex | Female | Female | Male | Male |
| Ethnicity | Black/Afro-Caribbean | White | Mixed | White |
| ADAMTS13 inhibitor on admission, U/mL | 11 | 30 | 56 | >80 |
| Platelet count on admission, ×109/L | 53 | 21 | 9 | 9 |
| Troponin elevation on admission | No | No | Yes | No |
| Creatinine elevation on admission | No | No | Yes | No |
| Neurological symptoms on admission | No | Yes* | Yes* | No |
| Intubation during admission | No | No | Yes† | No |
| Concurrent treatment | Steroid, rituximab | Steroid, rituximab | Steroid, rituximab | Steroid, rituximab, MMF |
| Time from initiation of PEX to first dose of caplacizumab, d | 0 | 8 | 1 | 7 |
| Total duration of PEX, d | 3 | 4 | 16 | 8 |
| Total duration of caplacizumab, d | 24 | 36 | 27 | 61 |
| ADAMTS13 activity at discontinuation of caplacizumab, IU/dL | 82.1 | 23.9 | 49.9 | 8.0 |
| Outcomes | ||||
| Time to platelet count normalization, d | 3 | 4 | 25 | 3 |
| Recurrence of TTP | No | No | No | No |
| Recovery of creatinine | N/A | N/A | Yes | N/A |
| Resolution of neurological symptoms | N/A | No‡ | Yes | N/A |
| Length of stay, d | 10 | 14 | 28 | 13 |
| Bleeding complications | No | No | No§ | No |
| Outcome | Survived | Survived | Survived | Survived |
MMF, mycophenolate mofetil.
The pediatric TTP cohort was defined as patients younger than 18 years of age at the time of receiving the first dose of caplacizumab.
Patient 2 had headaches, forgetfulness, limb weakness, and paresthesia; patient 3 had agitation and encephalopathy.
The indication for intubation was agitation and encephalopathy.
Case 2 was reported to have residual anxiety and depression.
Case 3 had caplacizumab interrupted as a precaution for 24 hours because of concerns of bleeding risk with removal of Vascath line. No bleeding occurred.