Program

(*) 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 B Break
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 (USA)
• Hemophilic patients with inhibitors undergoing orthopaedic surgery: the contribution of thrombin generation assay (TGA) as laboratory monitoring tool.
M.E. Mancuso (Italy)
• Induced Pluripotent Stem Cell (iPSC) – based Strategy to Correct the Bleeding Phenotype in Hemophilia A.
C. Olgasi (Italy)
• Restoring hemostatic balance in hemophilia by RNAi targeted silencing of antithrombin.
B. Sorensen (USA)
• Induction of tolerance to FVIII using nanoparticles in a murine model of hemophilia A.
T.K. Kishimoto (USA)
• Distinct cellular VWF phenotypes observed in BOEC from type 3 VWD patients with VWF propeptide mutations compared to non-propeptide mutations.
M.L. Bowman (Canada)
• ADAMTS13 destabilizes thrombi in a mouse model of thrombotic focal cerebral ischemia.
F. Denorme (Belgium)
• Pregnancy complications in acquired Thrombotic Thrombocytopenic Purpura.
B. Ferrari (Italy)
• Correction of murine ADAMTS13 deficiency and TTP-like symptoms using the ‘Sleeping Beauty’ transposon system.
S. Verhenne (Belgium)
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)

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