OC 73 - Rare factor deficiencies and treatment approaches in VWD

Moderator(s)

• 

  Ana Boban, MD, PhD

  Professor of Internal Medicine
  Haemophilia Centre, Department of Haematology,
  Department of Internal Medicine, University Hospital Centre Zagreb, Zagreb University School of Medicine
  Zagreb, Zagrebacka, Croatia
• 

  Yasmin Goga, MBBCH, FCPaeds, Cert Clin Haem(Paeds), Msc

  Paediatric haematologist
  Greys. Hospital, Univ of KwaZulu Natal, Durban, South Africa
  Westville, South Africa

Presentations:

• 10:15 – 10:30 ICT

  The genetic spectrum of patients with rare bleeding disorders
  

  Presenting Author: SAMIN MOHSENIAN, PhD – Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy

  Co-Author: Omid Seidizadeh, MSc, PhD – University of Milan, Department of Pathophysiology and Transplantation, Milan, Italy

  Co-Author: Andrea Cairo – Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy

  Co-Author: Roberta Palla – Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy,

  Co-Author: Marzia Menegatti, PhD – Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy

  Co-Author: Flora Peyvandi, MD, PhD – Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation
• 10:30 – 10:45 ICT

  Fitusiran displays efficient pro-hemostatic activity in a mouse model of inducible factor X-deficiency
  

  Presenting Author: Geneviève McCluskey, PhD – INSERM U1176, Université Paris Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Sebastien Verhenne – INSERM U1176, Université Paris Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Hortense Maynadié – INSERM U1176, Université Paris Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Cécile V. Denis – INSERM U1176, Université Paris Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Olivier D. Christophe, PhD – INSERM U1176, Université Paris Saclay, Le Kremlin-Bicêtre, France

  Co-Author: Peter J. Lenting, PhD – INSERM U1176, Université Paris Saclay, Le Kremlin-Bicêtre, France
• 10:45 – 11:00 ICT

  VGA039, a Protein S-targeting monoclonal antibody, demonstrates in a Phase Ia healthy volunteer trial the potential of subcutaneous weekly or less frequent prophylactic dosing for bleeding disorders
  

  Presenting Author: Christian Schoergenhofer, MD, PHD – Department of Clinical Pharmacology, Medical University of Vienna

  Co-Author: Miriam M. Moser – Department of Clinical Pharmacology, Medical University of Vienna

  Co-Author: Christa Firbas – Department of Clinical Pharmacology, Medical University of Vienna

  Co-Author: Timothy S. Leach – Vega Therapeutics, Inc.

  Co-Author: Almudena Tercero – Vega Therapeutics, Inc.

  Co-Author: Kelly Covert – Vega Therapeutics, Inc.

  Co-Author: Graham C. Parry – Vega Therapeutics, Inc.

  Co-Author: Hayley Lane – Vega Therapeutics, Inc.

  Co-Author: Bernd Jilma, MD – Department of Clinical Pharmacology, Medical University Vienna

  Co-Author: Sandip Panicker – Vega Therapeutics, Inc.

  Co-Author: Gary Patou – Vega Therapeutics, Inc.

  Co-Author: Benjamin Kim – Vega Therapeutics Inc.
• 11:00 – 11:15 ICT

  Personalized treatment by pharmacokinetic-guided dosing in von Willebrand disease; A prospective multicenter study, OPTI-CLOT/To WiN study
  

  Co-Author: Wala Al Arashi – Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands

  Co-Author: Michael E. Cloesmeijer – Department of Hospital Pharmacy and Clinical Pharmacology, Amsterdam UMC – location AMC, The Netherlands

  Co-Author: Frank W.G Leebeek, MD PhD – Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands

  Co-Author: Laurens Nieuwenhuizen – Department of Hematology, Maxima Medisch Centrum, Eindhoven, The Netherlands

  Co-Author: Floor Heubel-Moenen – Department of Hematology, Maastricht University Medical Center, Maastricht, The Netherlands

  Co-Author: Karina Meijer – Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands

  Co-Author: Ron A.A. Mathôt – Department of Hospital Pharmacy and Clinical Pharmacology, Amsterdam UMC – location AMC, The Netherlands

  Co-Author: Marjon H. Cnossen – Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands

  Presenting Author: Caroline Mussert, MD – Department of hematology – pediatric hematology, Erasmus Medical Center Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, the Netherlands
• 11:15 – 11:30 ICT

  Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study
  

  Presenting Author: Robert F. Sidonio, Jr., MD, M.Sc. – Emory University