| SCIENTIFIC SESSION 7 – Side effects of replacement therapy Chairs: Frits R. Rosendaal, Giovanni Di Minno |
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| 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 VIEW PAGE |
| ORAL COMMUNICATIONS 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 VIEW PAGE |
| 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 |
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| 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 VIEW PAGE |
| SCIENTIFIC SESSION 9 – Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome Chairs: Pier Mannuccio Mannucci, Giuseppe Remuzzi |
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| 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 |
SCHEDULE
