Wolfgang Miesbach’s Top 10 Picks for Bleeding Disorders from ASH 2025
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:
”ASH 2025: My Top 10 Picks for Bleeding Disorders.
With so many excellent abstracts to choose from, here are my personal top 10 selections:
Late-breaking abstracts
TRACTION trial – tranexamic acid goes hospital-wide (LBA-5)
Brett Houston presents a hospital-wide policy of tranexamic acid to reduce transfusion in major non-cardiac surgery.
A practice-changing, system-level intervention with implications for peri-operative bleeding management.
VAYHIT2 – a new era for ITP? (LBA-6) Hanny Al-Samkari reports phase 3 results of ianalumab plus eltrombopag versus placebo plus eltrombopag in primary immune thrombocytopenia after failure of first-line corticosteroids.
Dual targeting may reshape how refractory i is managed in routine practice.
Haemophilia A
Mim8 – FRONTIER2 52-week results
Lentz Steven and colleagues show that weekly Mim8 prophylaxis provides strong efficacy and a reassuring safety profile in adults and adolescents with haemophilia A.
NXT007 – subcutaneous prophylaxis for HA ± inhibitors
Maria Elisa Mancuso et al. present global phase I/II data on NXT007 in people with haemophilia A with or without FVIII inhibitors.
Emicizumab in infants – PedNet real-world data
Marloes de Kovel and the PedNet group report on 80 infants (PUPs and MTPs) with severe haemophilia receiving emicizumab.
Early prophylaxis appears feasible, effective and safe in real-world practice.
Haemophilia B
HOPE-B – 5-year gene therapy milestone
Steve Pipe and colleagues present five-year data for etranacogene dezaparvovec in haemophilia B.
Durable efficacy and a stable safety profile over this time span mark an important milestone for gene therapy.
Von Willebrand disease
VGA039 – subcutaneous Q4W prophylaxis
Allison Wheeler et al. report phase 1/2 results for the novel protein S antibody VGA039 in VWD.
Once-every-four-weeks subcutaneous dosing with substantial bleeding reduction could be a major advance for long-term prophylaxis and quality of life.
Recombinant VWF in paediatric surgery
Tammuella Chrisentery-Singleton et al. present retrospective data showing that recombinant VWF is safe and effective for children with VWD undergoing surgery.
This is relevant now that recombinant VWF has gained approval in the United States for paediatric use.
Rare bleeding disorders
Sutacimig for Glanzmann thrombasthenia
Paul Saultier et al. provide phase 2 data on sutacimig, a bispecific antibody for prophylaxis in Glanzmann thrombasthenia.
This represents a major step beyond platelet transfusions and opens a genuinely new treatment option.
Plasminogen therapy for type 1 deficiency
Karen Thibaudeau et al. report pharmacokinetics and clinical outcomes of plasminogen therapy in women and girls with reproductive tract lesions due to type 1 plasminogen deficiency.
Much-needed data for a small but significantly affected patient group.
Encouraging to see such breadth of innovation across ITP, haemophilia, VWD and rare disorders!”

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