Wolfgang Miesbach: Sophie Susen’s ”Long‑Term Prophylaxis in VWD” Talk at EAHAD 2026
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:
”Really appreciated Sophie Susen’s EAHAD 2026 talk on long‑term prophylaxis in severe von Willebrand disease.
‘Recombinant von Willebrand factor prophylaxis for severe von Willebrand disease: final results focusing on adults receiving once‑weekly prophylaxis in a phase 3b continuation study.’
Severe VWD often requires frequent VWF infusions; rVWF (vonicog alfa) is usually started at 40–60 IU/kg twice weekly, so safely reducing infusion frequency could meaningfully lower treatment burden for adults.
- Data from the once‑weekly adult subgroup (n=5):
- Over 35–36 months, treated spontaneous bleeding remained low (median sABR ≈1.0) with clear reductions versus historical baselines.
- Once‑weekly rVWF at ~60 IU/kg per infusion maintained good bleed control for most, although 2 of 5 adults required escalation to twice‑weekly dosing after about 2 years because of breakthrough bleeds.
Safety was favourable and all 5 adults completed the study, but these promising findings must be interpreted with caution given the very small number of participants.”
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