International Conference on Inhibitors in Haemophilia and Coagulation Disorders

International Conference on Inhibitors in Haemophilia and Coagulation Disorders.

SESSION 1:
New discoveries on the mechanism of inhibitor development
Chairs: Pier Mannuccio Mannucci (Italy), Donna Di Michele (USA)

 
  Roadmap for improved haemophilia treatment
Flora Peyvandi (Italy)
  Speed bumps on the innovation highway
Donna Di Michele (USA)
  Unexpected guests; Stabilin‐2 as a regulatory factor for inhibitor development
David Lillicrap (Canada)
  IDO1 and inhibitor development in hemophilia A
Davide Matino (Italy)

 

   
11:00 SESSION 1 (continued)
Chairs: Jan Voorberg (The Netherlands), Christoph Königs (Germany)
  Quantification and phenotyping of factor VIII-specific T cells in healthy donors
Bernard Maillère (France)
  Antigen presentation in inhibitor development: new discoveries
Sébastien Lacroix-Desmazes (France)
  To serve and protect: modulating effect of von Willebrand factor on FVIII peptide presentation
Jan Voorberg (The Netherlands)

 

12:30 ORAL COMMUNICATION SESSION
Industry research
Chairs: David Lillicrap (Canada), Elena Santagostino (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 (USA)
  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
   
14:30 SESSION 2:
New developments for tolerance induction
Chairs: Flora Peyvandi (Italy), Federico Mingozzi (France)
  Gene therapy and new approaches for tolerance induction Federico Mingozzi (France)
Federico Mingozzi (France)
  Liver induced tolerance
Percy Knolle (Germany)
   
16:00 SESSION 2 (continued)
Chairs: Luigi Naldini (Italy), David Lillicrap (Canada)
  Engineering regulatory T cells: Driving CARs to BARs
David Scott (USA)
  Perspectives of Immune Tolerance Induction (ITI) in the era of new haemophilia treatment options
Johannes Oldenburg (Germany)
  Alternatives to human VIII – orthologs, ancestors and hybrids thereof
Christopher B. Doering (USA)

 

17:30 WALKING ORAL COMMUNICATIONS SESSION
Foyer in front of the Plenary Hall (with refreshments)
Chairs: Donna Di Michele (USA), Frits 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)