New discoveries on the mechanism of inhibitor development

Chairs: Pier Mannuccio Mannucci (Italy)  and Donna DiMichele (USA)
09:00 Roadmap for improved haemophilia treatment
Flora Peyvandi (Italy)
09:10 Speed bumps on the innovation highway
Donna DiMichele (USA)
09:40 Unexpected guests; Stabilin‐2 as a regulatory factor for inhibitor development
David Lillicrap (Canada)
10:10 IDO1 and inhibitor development in hemophilia A
Davide Matino (Italy)
10:40 Coffee break

Chairs: Jan Voorberg (The Netherlands), Christoph Konigs (Germany)
11:00 Quantification and phenotyping of  factor VIII-specific T cells in healthy donors
Bernard Maillère (France)
11:30 Antigen presentation in inhibitor development: new discoveries
Sébastien Lacroix-Desmazes (France)
12:00 To serve and protect: modulating effect of von Willebrand factor on FVIII peptide presentation
Jan Voorberg (The Netherlands)
(Oral communication of best abstract submitted by the industry)
Chairs: David Lillicrap (Canada), Flora Peyvandi (Italy)

  • Emicizumab prophylaxis in adolescent/adult patients with haemophilia A previously receiving episodic or prophylactic bypassing agent treatment: updated analyses from the HAVEN 1 Study. Maria Elisa Mancuso (Italy)
  • Role of rFVIIIFc in immune tolerance induction in severe hemophilia A patients with Inhibitors. Nisha Jain
  • Efficacy, safety, and pharmacokinetics of once-weekly emicizumab prophylaxis in paediatric persons with haemophilia A (PwHA) with inhibitors two years old or younger in the HAVEN 2 Study. Flora Peyvandi (Italy)
  • Low immunogenicity of human-cell-derived recombinant FVIII in previously untreated patients with non-null F8 mutations. Anna Pavlova (Germany)
  • Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of haemophilia. Torben Colberg (USA)
  • Real-World safety and benefit of early treatment with room temperature-stable recombinant activated Factor VII (rFVIIa) in patients with haemophilia A or B with inhibitors: SMART-7™ study results.  Kaan Kavakli (Turkey), on behalf of the SMART-7™ steering committee
13:30 Lunch
New developments for tolerance induction

Chairs: Luigi Naldini (Italy) and Federico Mingozzi (France)
14:30 Gene therapy and new approaches for tolerance induction
Federico Mingozzi (France)
15:00 Liver induced tolerance
Percy Knolle (Germany)
15:30 Coffee break

Chairs: Luigi Naldini (Italy) and David Lillicrap (Canada)
16:00 Engineering regulatory T cells: Driving CARs to BARs
David Scott (USA)
16:30 Perspectives of Immune Tolerance Induction (ITI) in the era of new haemophilia treatment options
Johannes Oldenburg (Germany)
17:00 Alternatives to human VIII – orthologs, ancestors and hybrids thereof
Christopher B. Doering (USA)
17:30 Young scientist session: Walking oral communication session
Chairs: Donna DiMichele (USA), Frits R. Rosendaal (The Netherlands)

  • Generation and characterization of humanized hemophilic mice expressing HLA-DRB1*0101. Jules Russick (France)
  • Recombinant FVIII product type and the risk of inhibitor development in previously treated patients with hemophilia A: a systematic review and meta-analysis. Shermarke Hassan (The Netherlands)
  • SIPPET effect on Turkisk previously untreated patients (PUP) with severe hemophilia A: National data. Kaan Kavakli (Turkey)
  • Identification and functional characterization of a novel consensus sequence splicing mutation of factor VIII (FVIII) coagulation gene associated with severe hemophilia A and inhibitors development. Rosella Famà (Italy)
  • Single center experience in the treatment of haemophilia A PUPs. Susan Halimeh (Germany)
  • Lot-matching decreases the time for Immune Tolerance Induction (ITI): Preliminary results of the ObsITI study. Jonathan Ducore (USA)
  • Severe acquired von Willebrand syndrome (type 3-like): a case report. Francesca Stufano (Italy)

Discussion and question and answers

19:00 End of sessions


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