Wolfgang Miesbach: Caplacizumab as Frontline Therapy for iTTP
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:
“Caplacizumab as Frontline Therapy for iTTP: Is Plasmapheresis Still Needed? Priv.-Doz. Dr.med. Linus Völker presented at ASH compelling evidence that caplacizumab can work without plasma exchange in selected patients.
Systematic evidence: >100 plasma-free iTTP episodes
- First case reports and small series: ~10 episodes
- Kühne et al., Blood 2024 (Austria/Germany): 42 episodes with 90.5% clinical response without TPE and a median time to platelet normalization: 3 days (vs. 4 days with TPE, p=0.31), however, 4 patients required rescue TPE
- Knöbl, ISTH 2025: 34 episodes
- MAYARI Trial, ISTH 2025: 46 episodes and 93.5% remission without TPE (43/46 patients), 95.7% remission overall (44/46 patients)
This strategy is off-label and requires institutional capabilities:
- Extensive iTTP treatment experience at specialized centers
- 24/7 ADAMTS13 activity measurement (turnaround in hours, not days)
- Immediate caplacizumab access via emergency pharmacy
- Plasma exchange instantly available (safety net, not routine)
- No contraindications to caplacizumab therapy
Crucial to identify the right patient
There are clinical characteristics favoring a plasma-free approach
- Immediate platelet recovery after the first caplacizumab dose
- Clinically stable condition at presentation
- Lower LDH levels at onset
- Less severe organ involvement (lower ICU admission rates)
A fascinating glimpse into how frontline caplacizumab, in the right setting with the right patient, may allow truly plasma-free management for selected iTTP cases.”
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