(*) Please, note that times and dates of Scientific Sessions are tentative and subject to further changes.
| FRIDAY, OCTOBER 3rd 2014 |
| SESSION 1 • HEMOPHILIA Chairs: D.P. Lillicrap (USA), M. Makris (UK) |
| 8:30 | New insights into the structure and function of factor VIII |
| P.J. Lenting (France) | |
| 9:00 | Factor VIII physiology and von Willebrand factor interaction: consequences for half-life and bioengineering |
| S.W. Pipe (USA) | |
| 9:30 | Personalized medicine in hemophilia: how global assays can support individualization of factor therapy |
| G. A. Young (USA) | |
| 10:00 | Potential interest of global hemostasis assays in the management of hemophilia patients with inhibitors |
| Y. Dargaud (France) | |
| 10:30 | Thrombosis in patients with inherited bleeding disorders |
| M. Makris (UK) |
| 11:00 | Break |
| SESSION 2 • RARE BLEEDING DISORDERS: NO LONGER ORPHAN DISEASES Chairs: P.M. Mannucci (Italy), E. Santagostino (Italy) |
| 11:30 | New insights in the treatment of parahemophilia |
| P. Simioni (Italy) | |
| Factor V deficiency and development of an orphan drug | |
| F. Peyvandi (Italy) | |
| TRENDS OVERVIEW |
| 12:30 | Current and future paradigms in hemophilia |
| E. Santagostino (Italy) |
| 13:00/14:30 | Lunch |
| SATELLITE SYMPOSIA AND LECTURES |
| 14:30 | Satellite Symposium Sponsored by Kedrion From Humans to Humans: building a foundation for the future of haemophilia A management |
| 15:30 | Satellite Lecture Sponsored by CSL Behring Innovation in Recombinant Coagulation Factor |
| 16:00 | BBreak |
| SPECIAL LECTURE • Chair: N. Ciavarella (Italy) |
| 16:30 | How coagulation may differ in hemostasis and thrombosis |
| Z.M. Ruggeri (USA) | |
| SESSION 3 • COMPLEMENT ACTIVATION AND MICROVASCULAR/MACROVASCULAR THROMBOSIS – Chairs: S. Meri (Finland), M. Galbusera (Italy) |
| 17:00 | Disturbances of complement regulation in thrombotic microangiopathies |
| S. Meri (Finland) | |
| 17:30 | The role of complement activation in the diagnosis, treatment, and follow up of thrombotic thrombocytopenic purpura (TTP) and atypical hemolytic-uremic syndrome (aHUS) |
| S.R. Cataland (USA) | |
| 18:00 | Thrombosis and paroxysmal nocturnal hemoglobinuria (PNH) |
| A. Hill (UK) | |
| 18:30 | Complement activation-related diseases: shiga toxin hemolytic-uremic syndrome (Stx-HUS), atypical hemolytic-uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP) |
| M. Galbusera (Italy) |
SATURDAY, OCTOBER 4th 2014 |
| SESSION 4 • VON WILLEBRAND DISEASE Chairs: R.R. Montgomery (USA), Z.M. Ruggeri (USA) |
| 08:30 | The philosophy and reality of VWD |
| I. Peake (UK), A.C. Goodeve (UK) | |
| 09:20 | The role of blood outgrowth endothelial cells in understanding VWD |
| P.D. James (Canada) | |
| 09:50 | Ascertaining and defining variant VWD |
| R.R. Montgomery (USA) |
| 10:20 | Break |
| 11:00 | Clinical evaluation of a novel point of care test to determine different types of VWD in whole blood |
| M. Roest (The Netherlands) | |
| 11:30 | Predictors of bleeding and replacement therapy in VWD |
| A.B. Federici (Italy) |
SPECIAL LECTURE • Chair: K. Vanhoorelbeke (Belgium) |
| 12:00 | Fibrin clot structure and function: mechanisms of clot stability and thrombosis |
| R.A.S. Ariens (UK) |
| 12:30/14:00 | B Lunch |
| SATELLITE SYMPOSIA AND LECTURES |
| 14:00 | Satellite Lecture Sponsored by Grifols Secondary prophylaxis in von Willebrand Disease: Pro.Will Study interim results |
| 14:30 | Satellite Symposium Sponsored by Sobi Future technologies for the improvement of clotting factor pharmacokinetics |
| 15:30 | Satellite Joint Lecture Sponsored by Grifols, Kedrion and LFB State-of-the-Art of the SIPPET Study |
| SESSION 5 • VON WILLEBRAND FACTOR IN HEMOSTASIS AND THROMBOSIS Chairs: P.J. Lenting (France), R. Schneppenheim (Germany) |
| 16:00 | Receptor mediated clearance of von Willebrand factor and factor VIII |
| L.L. Swystun (Canada) | |
| 16:30 | New insights into the age-related pathobiology of von Willebrand factor |
| D.P. Lillicrap (Canada) |
| 17:00 | Break |
| 17:30 | Von Willebrand factor: cigars, strings and beyond |
| H.C.J. Eikenboom (The Netherlands) | |
| 18:00 | Von Willebrand factor control of blood vessel formation |
| A.M. Randi (UK) | |
| 18:30 | Role of von Willebrand factor in thromboembolism |
| R. Schneppenheim (Germany) | |
| 19:00 | Von Willebrand factor and the risk of atherothrombosis |
| F.W.G. Leebeek (The Netherland) | |
| 19:30 | Clinical aspects of active von Willebrand factor |
| M. Roest (The Netherlands) |
SUNDAY, OCTOBER 5th 2014 |
| SESSION 6 • GENE THERAPY IN BLEEDING DISORDERS Chairs: F. Pasinelli (Italy), P.M. Mannucci (Italy) |
| 08:30 | Update on gene therapy for hemophilia |
| A.C. Nathwani (UK) | |
| 09:00 | Overcoming immunogenicity of AAV vectors in gene therapy for inherited bleeding disorders |
| F. Mingozzi (France) | |
| 09:30 | Lentiviral vector based gene therapy of hemophilia |
| L. Naldini (Italy) | |
| 10:00 | Non-viral gene therapy for VWD using the Sleeping-Beauty transposon |
| S.F.M.G. De Meyer (Belgium) | |
| 10:30 | Invited discussant |
| P. Simioni (Italy) | |
| 11:00 B | B Break B |
| 11:30/13:00 B | ORAL COMMUNICATIONS Chairs: M.E. Mancuso (Italy), P.M. Mannucci (Italy) B |
| • Comparison of baseline thromboelastography and thrombin generation assay in Factor VIII deficient patients with and without inhibitors. G.A. Young |
|
| • Hemophilic patients with inhibitors undergoing orthopaedic surgery: the contribution of thrombin generation assay (TGA) as laboratory monitoring tool. M.E. Mancuso |
|
| • Induced Pluripotent Stem Cell (iPSC) – based Strategy to Correct the Bleeding Phenotype in Hemophilia A. C. Olgasi |
|
| • Restoring hemostatic balance in hemophilia by RNAi targeted silencing of antithrombin. B. Sorensen |
|
| • Induction of tolerance to FVIII using nanoparticles in a murine model of hemophilia A. T.K. Kishimoto |
|
| • Distinct cellular VWF phenotypes observed in BOEC from type 3 VWD patients with VWF propeptide mutations compared to non-propeptide mutations. M.L. Bowman |
|
| • ADAMTS13 destabilizes thrombi in a mouse model of thrombotic focal cerebral ischemia. F. Denorme |
|
| • Pregnancy complications in acquired Thrombotic Thrombocytopenic Purpura. B. Ferrari |
|
| • Correction of murine ADAMTS13 deficiency and TTP-like symptoms using the ‘Sleeping Beauty’ transposon system. S. Verhenne |
| 13:00/14:00 B | Lunch B |
| SATELLITE SYMPOSIA AND LECTURES |
| 14:00 | Satellite Symposium Sponsored by Baxter (Detailed program to be announced) Considerations in FVIII Pharmacokinetics, VWF, and Gene Therapy |
| SESSION 7 • VON WILLEBRAND FACTOR AND ADAMTS-13 IN HEALTH AND DISEASE Chairs: D.D. Wagner (USA), J.E. Sadler (USA) |
| 15:00 | Thrombotic microangiopathies: important open research questions |
| B. Lämmle (Switzerland) | |
| Structure, regulation and biological function of ADAMTS-13 | |
| J.E. Sadler (USA) | |
| 16:00 | Interplay between von Willebrand factor and ADAMTS-13 in vitro and in vivo |
| K. Vanhoorelbeke (Belgium) |
| 16:30 | Break |
| 17:00 | Von Willebrand factor and ADAMTS-13 in various disease processes |
| D.D. Wagner (USA) | |
| 17:30 | Novel therapeutics for acquired thrombotic thrombocytopenic purpura (TTP) |
| X. Zheng (USA) | |
| 18:00 | Insights into acquired ADAMTS-13 deficiency |
| J.A. Kremer Hovinga (Switzerland) | |
| 18:30 | Acquired thrombotic thrombocytopenic purpura (TTP): how far to prevent relapses? |
| P. Coppo (France) |
