SCIENTIFIC SESSION 7 – Side effects of replacement therapy
Chairs: Frits R. Rosendaal, Giovanni Di Minno
09.00 Randomisation or real world data?
Frits R. Rosendaal, the Netherlands
09.30 Current prophylaxis regimens are delaying but not preventing onset of joint arthropathy
Johannes B. Oldenburg, Germany
10.00 Predictors of outcome in immune tolerance induction (ITI): the Italian Registry
Giovanni Di Minno, Italy
10.30 Do we need to monitor for neurological adverse events in hemophilia?
Michael Makris, UK
11.00 Break & Poster Session
11.30 Oral communications: Translational Science
Chairs: Karen Vanhoorelbeke, Sebastien Lacroix-Desmazes. Embargoed until 14th Sept.
Towards a clinically relevant hybrid adenovirus-Sleeping Beauty transposon vector for gene therapy for von Willebrand disease
Irina Portier (Belgium)
Treatment of hemophilia A by injection of FVIII-encoding mRNA
Jules Russick (France)
Application of combined gene and cell therapy within an implantable therapeutic device for the treatment of severe haemophilia A
Chiara Borsotti (Italy)
Pharmacokinetic profile of rFVIIIFc-VWF-XTEN (BIVV001) protein in cynomolgus monkey generated from a large-scale manufacturing stable cell line
Joe Salas (USA)
Recombinant factor VIII Fc fusion protein exhibits immunomodulatory effects on antigen presenting cells
Katalin Kis-Toth (USA)
Scavenger-receptor stabilin-2 is a major regulator of mouse VWF propeptide clearance
Orla Rawley (Canada)
An open conformation of ADAMTS13 is a hallmark of acute acquired thrombotic thrombocytopenic purpura
Elien Roose (Belgium)
Profiling of anti-ADAMTS13 antibodies derived from patients with acquired thrombotic thrombocytopenic purpura
Nuno Graca (the Netherlands)
The role of von Willebrand factor in experimental malaria-associated acute respiratory distress syndrome
Sirima Kraisin (Belgium)
13.00 Lunch & Poster Session
14.00 Oral communications: Clinical Science
Chairs: Jenny Goudemand, Reinhard Schneppenheim. Embargoed until 14th Sept.
Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A or B with and without inhibitors: Interim results from a Phase 2 extension study
Pratima Chowdary (UK)
“FEIBA Global Outcome study (FEIBA GO)” first data read-out: Real world bleeding frequency in inhibitors patients on prophylaxis with APCC
Roberto Crea (Austria)
A cumulative review on four decades of thrombo-embolic events reported with the use of activated prothrombin complex concentrate (APCC) in congenital hemophilia
Alessandro Gringeri (Austria)
Influence of variant VWF multimer patterns on the diagnosis of VWD type 2B
Reinhard Schneppenheim (Germany)
Comparing platelet-dependent von Willebrand factor activity assays in 661 patients with von Willebrand disease – from the WiN Study
Johan Boender (the Netherlands)
Is type 3 VWD an homogeneous group?
Jenny Goudemand (France)
SCIENTIFIC SESSION 8 – Thrombotic thrombocytopenic purpura
Chairs: Dominique Meyer, Flora Peyvandi
15.00 Emerging concepts in acquired thrombotic thrombocytopenic purpura
Karen Vanhoorelbeke, Belgium
15.30 Immune recognition of ADAMTS13 in acquired thrombotic thrombocytopenic purpura (TTP)
Jan Voorberg, the Netherlands
16.00 Thrombotic microangiopathies
Paul Coppo, France
16.30 Break & Poster Session
SCIENTIFIC SESSION 9 – Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome
Chairs: Pier Mannuccio Mannucci, Giuseppe Remuzzi
17.00 The most recent advances in atypical hemolytic uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP) including complement and von Willebrand factor (VWF) complement interactions
Marina Noris and Giuseppe Remuzzi, Italy
17.30 Treatment monitoring in the atypical hemolytic uremic syndrome (HUS)
Massimo Cugno, Italy