Outcome data
| . | Germany (n = 60) . |
|---|---|
| Mortality, % (n/total) | 1.7 (1/60) |
| Follow-up, median (range), d | 108.5 (5-330) |
| Clinical response, % (n/total)* | |
| Achieved | 93.3 (56/60) |
| Not achieved | 3.3 (2/60) |
| Insufficient follow-up | 3.3 (2/60) |
| Exacerbation, % (n/total) | 38.3 (23/60) |
| While on caplacizumab | 3.3 (2/60) |
| Refractory disease, % (n/total) | 31.7 (19/60) |
| Refractoriness, grade, % (n/total)† | |
| Severe | 68.4 (13/19) |
| Mild | 31.6 (6/19) |
| Relapse, % (n/total) | |
| Relapsing disease | 3.3 (2/60) |
| Insufficient follow-up | 13.3 (8/60) |
| Time to final platelet normalization after diagnosis, median (range), d | 12 (3-152) |
| Reached at end of follow-up, % (n/total) | 95.0 (57/60) |
| Not reached at end of follow-up, % (n/total) | 5.0 (3/60) |
| Time to final platelet normalization after caplacizumab start (n = 64)‡§, d | |
| Median (range) | 3.0 (1-13) |
| Mean (95% CI) | 3.78 (3.19-4.38) |
| Duration of hospital stay (n = 59), d | |
| Median (range) | 18 (5-79) |
| Mean (95% CI) | 21.6 (18.0-25.2) |
| Duration of ICU stay (n = 54), d | |
| Median (range) | 4 (0-46) |
| Mean (95% CI) | 5.8 (3.8-7.7) |
| . | Germany (n = 60) . |
|---|---|
| Mortality, % (n/total) | 1.7 (1/60) |
| Follow-up, median (range), d | 108.5 (5-330) |
| Clinical response, % (n/total)* | |
| Achieved | 93.3 (56/60) |
| Not achieved | 3.3 (2/60) |
| Insufficient follow-up | 3.3 (2/60) |
| Exacerbation, % (n/total) | 38.3 (23/60) |
| While on caplacizumab | 3.3 (2/60) |
| Refractory disease, % (n/total) | 31.7 (19/60) |
| Refractoriness, grade, % (n/total)† | |
| Severe | 68.4 (13/19) |
| Mild | 31.6 (6/19) |
| Relapse, % (n/total) | |
| Relapsing disease | 3.3 (2/60) |
| Insufficient follow-up | 13.3 (8/60) |
| Time to final platelet normalization after diagnosis, median (range), d | 12 (3-152) |
| Reached at end of follow-up, % (n/total) | 95.0 (57/60) |
| Not reached at end of follow-up, % (n/total) | 5.0 (3/60) |
| Time to final platelet normalization after caplacizumab start (n = 64)‡§, d | |
| Median (range) | 3.0 (1-13) |
| Mean (95% CI) | 3.78 (3.19-4.38) |
| Duration of hospital stay (n = 59), d | |
| Median (range) | 18 (5-79) |
| Mean (95% CI) | 21.6 (18.0-25.2) |
| Duration of ICU stay (n = 54), d | |
| Median (range) | 4 (0-46) |
| Mean (95% CI) | 5.8 (3.8-7.7) |
Clinical response as defined in Scully et al7 : a sustained normalization of platelet counts >150 × 109/L and of LDH <1.5 upper limit of normal after cessation of PEX.
Refractoriness grade as defined in Scully et al7 . Refractory TTP is defined as persistent thrombocytopenia <50 × 109/L and persistently raised LDH levels despite 5 PEX and steroid treatments. It is defined as severe if thrombocytopenia remains <30 × 109/L.
Platelets >150 × 109/L. In 3 patients, platelet count did not normalize due to ongoing sepsis or concomitant liver cirrhosis. These cases were excluded from the calculation (patients 10, 49, and 53). One patient (patient 18) was not computed because of aTTP-related death.
Instances (n = 64) rather than patient numbers are reported here reflecting the fact that, in some patients, caplacizumab treatment was stopped and restarted. Every time caplacizumab was restarted, the time to platelet normalization was measured again.