24 / 07 / 2017 SCIENTIFIC COMMITTEE. P.M. Mannucci F. Peyvandi A.B. Federici N. Ciavarella

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1 24 / 07 / 2017 SCIENTIFIC COMMITTEE P.M. Mannucci F. Peyvandi A.B. Federici N. Ciavarella

2 This international conference, that takes place every three years in Italy and has reached the nineth edition, is focused upon the clinical and translational research of a group of rare disorders of the hemostatic system: hemophilia, von Willebrand disease, rare bleeding disorders and the thrombotic microangiopathies (such as: thrombotic thrombocytopenic purpura and hemolitic uremic syndrome). The invited speakers, from Europe, North America and Asia, are among the best experts of these disorders, which are rare but of great relevance and clinical complexity. PRELIMINARY PROGRAMME FRIDAY, 15th September SCIENTIFIC SESSION 1: Hemophilia Chairs: Nicola Ciavarella, David Lillicrap Will severe hemophilia become more common in the future? The effects of population demographics on the incidence of hemophilia Manuel Carcao, Canada Determinants of factor VIII inhibitor risk. Is von Willebrand factor a key factor? David Lillicrap, Canada Endocytosis of factor VIII by antigen-presenting cells: roles of von Willebrand factor and complement Sébastien Lacroix-Desmazes, France Paradigm shift of the treatment of hemophilia A by factor VIII mimicking bispecific antibody Midori Shima, Japan

3 10.30 The challenges of innovative therapeutic interventions in hemophilia Louis M. Aledort, USA Break & Poster Session SCIENTIFIC SESSION 2: Von Willebrand disease Chairs: Giancarlo Castaman, Augusto B. Federici Von Willebrand factor regulation of blood vessel formation and function Anna Maria Randi, UK Gastrointestinal bleeding in von Willebrand disease: an unresolved problem Giancarlo Castaman, Italy The potential use of nanobodies in the treatment of von Willebrand disease and hemophilia Peter Lenting, France Lunch & Poster Session Desmopressin (DDVAP): 40 years after. Oral communications Chairs: Pier Mannuccio Mannucci, Massimo Franchini. Embargoed until 14th Sept. SATELLITE SYMPOSIA / LECTURE Satellite Symposium Break & Poster Session SCIENTIFIC SESSION 3: Thrombotic Microangiopathies Chairs: Flora Peyvandi, Johanna Kremer Hovinga Mechanism of thrombotic microangiopathy: lesson from zebrafish to men X. Long Zheng, USA Differential diagnosis of thrombotic microangiopathies Jeffrey C. Laurence, USA Immune repertoire in acquired thrombotic thrombocytopenic purpura (TTP) Johanna Kremer Hovinga, Switzerland

4 SATURDAY, 16th September SCIENTIFIC SESSION 4: Gene Therapy Chairs: Luigi Naldini, Flora Peyvandi Addressing vector immunogenicity in the development of AAV-based gene therapies for bleeding disorders Federico Mingozzi, France Liver-directed lentiviral gene therapy of hemophilia Luigi Naldini, Italy An update on UCL/St Jude Haemophilia studies; successes and obstacles ahead Amit Nathwani, UK Gene therapy in hemophilia A Liron Walsh, USA Break & Poster Session SCIENTIFIC SESSION 5: Von Willebrand Factor Chairs: Pier Mannuccio Mannucci, Denisa Wagner Von Willebrand factor in ischemic stroke Simon de Meyer, France Interplay between von Willebrand factor, ADAMTS13 and NETs Denisa Wagner, USA SATELLITE SYMPOSIA / LECTURE Satellite Lecture Lunch & Poster Session Satellite Lecture Satellite Symposium Break & Poster Session SCIENTIFIC SESSION 6: Von Willebrand Factor and Disease Chairs: Robert Montgomery, Zaverio M. Ruggeri Old concepts and new functions of von Willebrand factor: 2017 Zaverio M. Ruggeri, USA

5 17.00 Von Willebrand factor regulation and role in disease José Lopez, USA Collagen and VWF interactions and beyond Veronica Flood, USA The genetics of von Willebrand disease Ann Goodeve, UK The many faces of thrombocytopenia in von Willebrand disease type 2B Cécile Denis, France SUNDAY, 17th September SCIENTIFIC SESSION 7: Side effects of replacement therapy Chairs: Frits R. Rosendaal, Giovanni Di Minno Randomisation or real world data? Frits R. Rosendaal, the Netherlands Current prophylaxis regimens are delaying but not preventing onset of joint arthropathy Johannes B. Oldenburg, Germany Predictors of outcome in immune tolerance induction: the Italian Registry Giovanni Di Minno, Italy Do we need to monitor for neurological adverse events in hemophilia? Michael Makris, UK Break & Poster Session ORAL COMMUNICATIONS SESSION Oral communications: Translational Science Chairs: Karen Vanhoorelbeke, Sébastien Lacroix-Desmazes. Embargoed until 14th Sept.

6 13.00 Lunch & Poster Session Oral communications: Clinical Science Chairs: Jenny Goudemand. Embargoed until 14th Sept. SCIENTIFIC SESSION 8: Thrombotic Thrombocytopenic Purpura Chairs: Dominique Meyer, Flora Peyvandi Emerging concepts in acquired thrombotic thrombocytopenic purpura Karen Vanhoorelbeke, Belgium Immune recognition of ADAMTS13 in acquired TTP Jan Voorberg, the Netherlands Thrombotic microangiopathies Paul Coppo, France Break & Poster Session SCIENTIFIC SESSION 9: Thrombotic Thrombocytopenic Purpura and Hemolytic Uremic Syndrome Chairs: Pier Mannuccio Mannucci, Giuseppe Remuzzi The most recent advances in atypical hemolytic uremic syndrome (ahus) and thrombotic thrombocytopenic purpura (TTP) including complement and von Willebrand factor (complement interactions) Marina Noris and Giuseppe Remuzzi, Italy Treatment monitoring in the atypical hemolytic uremic syndrome (HUS) Massimo Cugno, Italy Scientific session Satellite Symposia / Lecture Break & Poster session Oral Communications

7 FACULTY L. M. Aledort New York, USA M. Carcao Toronto, Canada G. Castaman Florence, Italy N. Ciavarella Bari, Italy P. Coppo Paris, France M. Cugno S. De Meyer Kortrijk, Belgium C. Denis Le Kremlin-Bicêtre, France G. Di Minno Naples, Italy A. B. Federici V. Flood Milwaukee, USA M. Franchini Mantova, Italy A. C. Goodeve Sheffield, UK J. Kremer Hovinga Bern, Switzerland S. Lacroix-Desmazes Paris, France J. C. Laurence New York, USA P. J. Lenting Le Kremlin-Bicêtre, France D. Lillicrap Kingston, Canada J. A. López Seattle, USA P. M. Mannucci M. Makris Sheffield, UK D. Meyer Paris, France F. Mingozzi Evry, France R. R. Montgomery Milwaukee, USA L. Naldini A. Nathwani London, UK M. Noris Ranica (BG), Italy J. Oldenburg Bonn, Germany F. Peyvandi A. M. Randi London, UK G. Remuzzi Bergamo, Italy F. R. Rosendaal Leiden, the Netherlands Z. M. Ruggeri La Jolla, USA M. Shima Nara, Japan K. Vanhoorelbeke Kortrijk, Belgium J. Voorberg Amsterdam, the Netherlands D. Wagner Boston, USA L. Walsh Los Angeles, USA X. L. Zheng Birmingham, USA

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