10TH BIC INTERNATIONAL CONFERENCE

10TH BIC INTERNATIONAL CONFERENCE

AUSPICES

COMUNE DI GENOVA

ORGANIZED BY

SCIENTIFIC COMMITTEE

CHAIRED BY: P.M. MANNUCCI, F. PEYVANDI, Italy N. CIAVARELLA, Italy A.B. FEDERICI, Italy S. LACROIX-DESMAZES, France P.J. LENTING, France D. LILLICRAP, Canada J. VOORBERG, The Netherlands 100% 08:30

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BIC_hemophilia T BIC INTERNATIONAL CONFERENCE

The major innovations that were presented at the most recent events suggest that 2019 is a year of continuous development in treatment approaches and new drugs. Data from basic science research and clinical trials are outlining new scenarios, not only in gene therapy but also in the progress of replacement and non-replacement therapies with new drugs appearing in the market.

This joint meeting is meant to take the best of BIC & INHIBITORS International Conferences and feature only the most novel advances of basic science and clinical research in hemophilia, rare inherited coagulation disorders, von Willebrand factor and von Willebrand disease, gene therapy, the thrombotic microangiopathies (TTP, HUS and aHUS) and, of course, factor VIII inhibitors.

The most distinguished experts in the field will present their very latest data and offer their wisdom and insight into the current science developments, during a 3-day programme. We wish to welcome clinical and basic science researchers to the Joint 10th BIC & 3rd Inhibitors in Hemophilia International Conference and look forward to interacting with them throughout the whole 3-day period.

The Scientific Committee P.M. Mannucci, F. Peyvandi N. Ciavarella, A.B. Federici, S. Lacroix-Desmazes, P. Lenting, D. Lillicrap, J. Voorberg 6TH SEPTEMBER

Replacement therapies in hemophilia 12:00 European pharmacovigilance of new A and B - Efficacy of extended half-life products (with special emphasis on products pegylated products) Chairs: David Lillicrap (Canada), Anneliese Hilger (EMA, Germany) Pier Mannuccio Mannucci (Italy) 12:30 Immunogenicity of subcutaneous and 08:30 Real life data: real benefits. What intravenous drugs have we achieved? David Lillicrap (Canada) Pratima Chowdari (UK) 13:00 Lunch Satellite Symposium: 09:00 How to optimize the use of extended Evolution by design: advancing the half-life products (PK analysis) standard of care in hemophilia A Alfonso Iorio (Canada) Sponsored by Bayer (not accredited for CME) Safety of replacement therapies Chair: Erik Berntorp (Sweden) (standard and novel) (Part I) Chairs: Nicola Ciavarella (Italy), How evolution leads to revolution in Maria Elisa Mancuso (Italy) hemophilia care Erik Berntorp (Sweden) 09:30 Which bleeding phenotype can we expect in severe hemophilia A Advances driven by evolution: previously untreated patients treated investigating the role of factor VIII with non replacement therapies? (FVIII) in coagulation and beyond Karin Fijn van Draat (The Netherlands) Hermann Eichler (Germany)

10:00 How safety of novel anti-hemophilic Advances driven by design: using real- drugs should be approached. Known life practice to guide clinical studies and unknown adverse effects (results from PROTECT VIII) Flora Peyvandi (Italy) Elena Santagostino (Italy)

10:30 Are Fc - factor VIII (FVIII) products Q&A less immunogenic? New clinical data? Christoph Königs (Germany) 14:00 Satellite Symposium: ADAMTS13 and von Willebrand factor 11:00 Break (VWF): progressing patient care through protein innovation Safety of replacement therapies Sponsored by Takeda (standard and novel) (Part II) (not accredited for CME) Chairs: Giovanni Di Minno (Italy), Chair: Flora Peyvandi (Italy) Mauro Biffoni (Italy) Welcome and introduction 11:30 Long-term side-effects of pegylated Flora Peyvandi (Italy) products: facts or fancies? Silvio Garattini (Italy) The ADAMTS13-von Willebrand factor 16:00 Keynote Lecture “Choice of assays for (VWF) interaction: clinical implications novel anti-hemophilic therapies” & applications Armando Tripodi (Italy) Peter Lenting (France) Chair: Wolfgang Schramm (Germany)

Congenital thrombotic The role of von Willebrand thrombocytopenic purpura (cTTP) as factor (VWF) in factor VIII (FVIII) a chronic disease: what is the long- immunogenicity term view to achieve more optimal Chairs: Giancarlo Castaman (Italy), outcomes? Augusto B. Federici (Italy) Paul Coppo (France) 16:30 Factor VIII (FVIII) peptide The role of von Willebrand factor (VWF) presentation to the immune system; in individualized care for patients with modulation by von Willebrand factor von Willebrand disease (VWD) (VWF) Andreas Tiede (Germany) Jan Voorberg (The Netherlands)

Panel discussion 17:00 High von Willebrand factor (VWF) Facilitated by Flora Peyvandi (Italy) affinity factor VIII (FVIII) and immunogenicity Meeting summary and close Peter Lenting (France) Flora Peyvandi (Italy) 17:30 Walking Poster Session 15:00 Satellite Lecture: See ePoster programme for details Von Willebrand factor (VWF) and factor VIII (FVIII): an unending love affair Sponsored by Kedrion (not accredited for CME) Chair: Prasad Mathew (USA)

Setting the stage Prasad Mathew (USA)

Clinical implications of this love story for patients with hemophilia A Maria Elisa Mancuso (Italy)

Q&A

15:30 Break

Scientific session Oral communications and poster session Satellite symposium/lecture 7TH SEPTEMBER

Non-replacement therapies in the Subcutaneous prophylaxis with hemophilias: update concizumab in patients with Chairs: Peter Lenting (France), haemophilia A and haemophilia A/B Jan Voorberg (The Netherlands) with inhibitors: Phase 2 trial results Giancarlo Castaman (Italy) 09:00 Fitusiran Steven Pipe (USA) Q&A

09:30 APC serpin 12:30 Satellite Lecture: James Huntington (UK) From trials to clinical practice: emergent treatments to overcome challenges in 10:00 Anti-TFPI people with haemophilia A Alan Mast (USA) Sponsored by Roche (not accredited for CME) 10:30 Thrombotic microangiopathy Chair: Flora Peyvandi (Italy) associated with emicizumab: views Speaker: Amy Shapiro (USA) from an insider Paul Coppo (France) 13:00 Lunch Satellite Symposium: Can we improve inhibitor management 11:00 Break in patients with haemophilia A? Clinical approaches to inhibitor Novel drugs prevention and elimination Chair: Paul Coppo (France) Sponsored by Octapharma (not accredited for CME) 11:30 Caplacizumab: results of phase II/ Chair: Jan Astermark (Sweden) phase III studies Marie A. Scully (UK) The challenge of inhibitors in haemophilia A - Introduction 12:00 Satellite Lecture: Jan Astermark (Sweden) Treatment of bleeding episodes in the era of non-factor replacement Immune tolerance induction therapies in haemophilia A - challenges, Sponsored by Novo Nordisk experience and future approaches (not accredited for CME) Carmen Escuriola (Germany) Chair: Pratima Chowdary (UK) ITI in the context of new therapies Welcome & introduction for haemophilia A: the Atlanta and Pratima Chowdary (UK) MOTIVATE studies Robert Sidonio (USA) Concomitant use of rFVIIa and emicizumab in congenital haemophilia Final results from the NuProtect study A with inhibitors: safety data from the of Nuwiq® in previously untreated HAVEN clinical programme patients Stephanie Seremetis (USA) Ri Liesner (UK)

Q&A Gene Therapy Towards the transplacental delivery Chairs: Steven Pipe (USA), of maternal FVIII to FVIII-deficient Amy Shapiro (USA) progeny for induction of active immune tolerance to therapeutic FVIII 14:00 Phagocytosis-shielded lentiviral Angelina Mimoun (France) vectors for hemophilia gene therapy Alessio Cantore (Italy) 16:00 Break

14:30 Liver toxicity in gene therapy Immunotolerance induction in Edward Tuddenham (UK) hemophilia (Part I) Chairs: Maria Elisa Mancuso (Italy), 15:00 Oral Communications - Hemophilia Kathleen Pratt (USA)

Targeting of hepatocyte 16:30 How to maintain tolerance in subpopulation contributing to liver hemophilia A post-natal growth is crucial for Sébastien Lacroix-Desmazes (France) maintenance of transgene expression in liver-directed gene therapy 17:00 How to induce Treg responses in Michela Milani (Italy) experimental hemophilia A Naro Biswas (USA) Transplantation of fetal liver cells into newborn hemophilic mice for the 17:30 Is it important to induce immune treatment of hemophilia A without tolerance in the era of non- inhibitors formation replacement therapies? Simone Merlin (Italy) Elena Santagostino (Italy)

B-AMAZE, a Phase 1/2 trial of a novel 18:00 Specific Treg therapy for hemophilia investigational adeno associated inhibitors: CARs versus BARs? virus (AAV) gene therapy (FLT180a) David Scott (USA) in subjects with severe or moderately severe hemophilia B (HB) 18:30 Walking Poster Session Pratima Chowdary (UK) See ePoster programme for details

Developing a novel coagulation factor VIII with reduced immunogenicity by a direct deimmunization approach Karina Winterling (Germany)

Recombinant, patient-derived FVIII- neutralising antibodies: a platform for research, product testing, and ex vivo modelling of haemophilia A Carmen Coxon (UK)

Scientific session Oral communications and poster session Satellite symposium/lecture 8TH SEPTEMBER

Von Willebrand factor (VWF) and 14:00 Oral Communications - Von Willebrand ADAMTS13 in hemostasis and factor (VWF) and ADAMTS 13 thrombosis (Part I) Chairs: Ilaria Mancini (Italy), Von Willebrand factor (VWF) production Karen Vanhoorelbeke (Belgium) and secretion, and endothelial cell characteristics in healthy ECFCs; 09:00 Von Willebrand factor (VWF) self- Generating a valid ex vivo model for association role in hemostasis and von Willebrand disease (VWD) thrombosis Suzan De Boer (The Netherlands) José López (USA) Inhibition of ADAMTS13 prevents 09:30 Interactions of von Willebrand factor the loss of high molecular weight von (VWF)/ADAMTS13 Willebrand factor (VWF) multimers Jim Crawley (UK) in an in vitro left ventricular assist device 10:00 Modulation of ADAMTS13 Shannen Deconinck (Belgium) conformation (open/close) in thrombotic thrombocytopenic Conformation of ADAMTS13 in purpura (TTP) the French cohort of child-onset Karen Vanhoorelbeke (Belgium) thrombotic thrombocytopenic purpura 10:30 Impact of cohort studies on Bérangère Joly (France) therapeutic approaches for von Willebrand disease (VWD) Patient anti-ADAMTS13 Jeroen Eikenboom (The Netherlands) autoantibodies induce an open ADAMTS13 conformation in 11:00 Break immune mediated thrombotic thrombocytopenic purpura Von Willebrand factor (VWF) and Elien Roose (France) ADAMTS13 in hemostasis and thrombosis (Part II) Tilting the balance: A trade- Chairs: Ilaria Mancini (Italy), off between thrombotic Karen Vanhoorelbeke (Belgium) thrombocytopenic purpura (TTP) patients autoantibody binding and 11:30 Update on 3WINTERS proteolytic activity of ADAMTS13 Augusto B. Federici (Italy) variants Nuno Alexandre Gomes Graça 12:00 Acquired von Willebrand disease (VWD) (The Netherlands) Giancarlo Castaman (Italy) In-depth epitope mapping of 12:30 Role of von Willebrand factor (VWF) anti-ADAMTS13 autoantibodies in the vessel wall: interplay with in immune-mediated thrombotic vascular smooth muscle cells thrombocytopenic purpura patients Cécile Denis (France) using a large library of ADAMTS13 fragments 13:00 Lunch Kadri Kangro (Belgium) Other clinical challenges of hemophilia Chairs: Massimo Morfini (Italy), Elena Santagostino (Italy)

15:00 Thrombosis in hemophilia: results from EUHASS registry, including thrombotic microangiopathies Michael Makris (UK)

15:30 Ageing with hemophilia Pier Mannuccio Mannucci (Italy)

16:00 Eradication of HCV in Europe Massimo Colombo (Italy)

16:30 Break

Immunotolerance induction in hemophilia (Part II) Chairs: Sébastien Lacroix-Desmazes (France), Carmen Escuriola (Germany)

17:00 Gene-therapy-mediated tolerance induction Valder R. Arruda (USA)

17:30 A working group on new concepts for ITI Kathleen Pratt (USA)

18:00 End of the Conference

Scientific session Oral communications and poster session Satellite symposium/lecture ePOSTER SESSION BOCCADASSE

Friday, 6TH SEPTEMBER Saturday, 7TH SEPTEMBER

17:30 rFVIIIFc for first-time immune 18:30 In-depth epitope mapping of tolerance induction (ITI) therapy: anti-ADAMTS13 autoantibodies interim results from the global, in immune-mediated thrombotic prospective verITI-8 study thrombocytopenic purpura patients L. Malec (USA) using a large library of ADAMTS13 fragments 17:40 Brazilian registry of persons with K. Kangro (Belgium) hemophilia A receiving emicizumabe (emicizumab cases, EMCase Project) 18:40 APAC, a dual AntiPlatelet and R. Camelo (Brazil) AntiCoagulant, towards a local vascular targeting antithrombotic 17:50 Nature of FVIII-containing immune R. Lassila (Finland) complexes and induction of immune tolerance in patients with hemophilia A 18:50 Investigation of the ADAMTS13 M. Bou Jadeh (France) c.3178C>T mutation in hereditary and acquired thrombotic 18:00 Performance of a clinical risk thrombocytopenic purpura prediction model for inhibitor G. Sinkovits (Hungary) formation in severe hemophilia A S. Hassan (The Netherlands) 19:00 Rare bleeding disorders in the pediatric population of Northern 18:10 Real-World efficacy and safety data Greece: a 10-year single center of patients with hemophilia and experience inhibitors treated with aPCC: “FEIBA A. Adramerina (Greece) global outcome Study (FEIBA-GO)”, results from >12 months follow-up 19:10 Successful treatment and durable V. Cano (Switzerland) remission of refractory thrombotic thrombocytopenic purpura (TTP) with 18:20 The timing of initial exposures to FVIII belimumab treatment in nonsevere hemophilia A R. Woods (USA) A. Abdi (The Netherlands) ePOSTER SESSION CATHEDRAL OF SAN LORENZO

Friday, 6TH SEPTEMBER Saturday, 7TH SEPTEMBER

17:30 Immune tolerance induction rescue 18:30 The presence of anti-ADAMTS13 (ITI-R) with human-cl rhFVIII in autoantibodies in immune-mediated hemophilia A patients and high-titre thrombotic thrombocytopenic inhibitors purpura patients does not hamper S. Pasca (Italy) correct determination of ADAMTS13 antigen levels 17:40 Usefulness of global haemostasis C. Dekimpe (Belgium) assays in hemophilia A patients with discrepant bleeding phenotype 18:40 Single-sample detection of anti- M. Milos (Croatia) ADAMTS13 autoantibodies using fiber-optic surface plasmon resonance 17:50 Visual inspection of the aPTT reaction technology curve as a reliable screening tool for C. Dekimpe (Belgium) detecting the presence of FVIII and FIX inhibitors 18:50 BMI is an important determinant of J. Pavic (Bosnia and Herzegovina) VWF and FVIII levels and bleeding phenotype in patients with von 18:00 Comparison of the effect of cationic Willebrand disease and anionic polyamidoamine F. Atiq (The Netherlands) dendrimers on components of coagulation system 19:00 Analytical performance validation L. Mukhametova (Russia) of a new screening assay for the deficiency of ADAMTS-13 activity 18:10 Prospective study of the N. Binder (Austria) immunological response to factor VIII in severe hemophilia A patients 19:10 Feasibility of a new fully automated during immune tolerance induction ADAMTS-13 activity assay treatment N. Binder (Austria) S. Rossini (Italy)

18:20 Accurate factor VIII concentrate dosing by ideal body weight in overweight and obese hemophilia A patients T. Goedhart (The Netherlands) ePOSTER SESSION THE ROYAL PALACE

Friday, 6TH SEPTEMBER Saturday, 7TH SEPTEMBER

17:30 Baseline patient characteristics in 18:30 Type 1 VWD caused by a novel ReITIrate - a prospective study of dominant p.Thr274Pro mutation rescue ITI with recombinant factor localized in VWF propeptide VIIIFc (rFVIIIFc) in patients who have L. Baronciani (Italy) failed previous ITI attempts C. Königs (Germany) 18:40 Evaluation of the von Willebrand factor (VWF) inhibitor in a large 17:40 Transplantation of fetal liver cells cohort of European and Iranian into newborn hemophilic mice for the patients previously diagnosed with treatment of hemophilia A without type 3 von Willebrand disease (VWD) inhibitors formation enrolled into the 3WINTER-IPS Project S. Merlin (Italy) L. Baronciani (Italy)

17:50 LIFE-ACTIVE: observational study 18:50 Current view on the safety of evaluating the physical activity in PEGylated biologics in hemophilia a subset of damoctocog alfa pegol treatment treated hemophilia A patients who A. Baumann (Germany) are enrolled in the HEM-POWR study E. Musi (Switzerland) 19:00 The presence of inhibitors against von Willebrand factor and factor VIII in 18:00 Observational immune tolerance the same patient: a case report induction study (ObsITI): Immune D. Coen Herak (Croatia) tolerance induction with a single factor VIII/von Willebrand factor 19:10 Evaluation of platelet-dependent concentrate for treatment of VWF activity on a heterogeneous hemophilia A patients with inhibitors group of von Willebrand disease C. Escuriola (Germany) patients using four different methods: two VWF:GPIbM and two VWF:RCo 18:10 MOTIVATE: MOdern Treatment of assays Inhibitor-positiVe pATiEnts with P. Colpani (Italy) hemophilia A - An international observational study C. Escuriola (Germany)

18:20 Structural Evidence of an Anti-Factor C1 Domain Antibody Bound to B Domain-Deleted factor VIII P.C. Spiegel (USA) ePOSTER SESSION VILLA DURAZZO PALLAVICINI

Friday, 6TH SEPTEMBER Saturday, 7TH SEPTEMBER

17:30 Management of acquired hemophilia A: 18:30 Prognostic value of polymorphisms a case series of the CYP2C19 gene in the S. Varvello (Italy) development of stent thrombosis A. Zhunuspekova (Croatia) 17:40 Immune tolerance induction with Nuwiq® (Simoctocog Alfa) in nine 18:40 Six years of experience with plasma patients with severe hemophilia A and fractionation industries at a private inhibitors to FVIII super speciality hospital in south R. Liesner (UK) india: renaissance in usage of excess plasma in blood banks 17:50 Final results from the NuProtect B.B. Poluru Mranikrinda (India) study of Nuwiq® (simoctocog alfa) treatment in previously untreated 18:50 Evaluation of the chromogenic assay patients with severe hemophilia A “Biophen factor IX” on Sysmex CS- R. Liesner (UK) 2400 analyzer C. Novembrino (Italy) 18:00 Combination of emicizumab with simoctocog alfa for prophylaxis 19:00 Testing clot growth and thrombin in previously untreated/minimally wave patterns in hemophilia patients treated hemophilia A patients, and for C. Kluft (The Netherlands) managing inhibitor patients R. Sidonio (USA) 19:10 Overview of efficacy and safety of a plasma-derived human von Willebrand 18:10 Population pharmacokinetic model factor concentrate for perioperative for recombinant factor VIII Fc fusion management and delivery in patients protein (rFVIIIFc) validated and with hereditary VWF deficiency, optimized for use in children unresponsive to dDAVP L. Bukkems (The Netherlands) F. Bridey (France)

18:20 The combination of plasma-derived FVIII/VWF with emicizumab has non-additive effects on thrombin generation assay independently of the presence of inhibitors in hemophilia A plasma A. Pérez (Spain) ePOSTER SESSION PORTA SOPRANA

Friday, 6TH SEPTEMBER Saturday, 7TH SEPTEMBER

17:30 Recombinant, patient-derived FVIII- 18:30 Thrombin and plasmin generation neutralising antibodies: a platform for in patients with plasminogen or research, product testing, and ex vivo plasminogen activator inhibitor type 1 modelling of hemophilia A deficiency C. Coxon (UK) J. Saes (The Netherlands)

17:40 Use of chromogenic factor VIII 18:40 Quantification of VWF propeptide activity determination in hemophilia A release before and after plasma of patients under emicizumab desmopressin in von Willebrand treatment disease and hemophilia A N. Binder (Austria) L. Bukkems (The Netherlands)

17:50 Performance evaluation of a new 18:50 In vitro and in vivo modulation of von fully automated thrombin generation Willebrand factor gene mutations instrument for the measurement of with dominant-negative effect TGA in hemophilia samples C. Casari (Italy) N. Binder (Austria) 19:00 Long-term neuropsychological 18:00 ADAMTS13 Inhibitor assessment sequelae, emotional wellbeing with the hemosil Acustar ADAMTS13 and quality of life in patients activity assay with acquired thrombotic C. Valsecchi (Italy) thrombocytopenic purpura I. Mancini (Italy)

19:10 Non-specific non-antiphospholipid inhibitor in a 26-years-old woman: a case report with few answers and many questions N. Ciavarella (Italy) THURSDAY 5TH SEPTEMBER Welcome dinner Inner city stories - A dinner in denim

We are pleased to invite you to the Welcome Dinner on Thursday, September 5th, in a superb medioeval historical location in the heart of Genova: the Museo Diocesano Cloister. An al-fresco soirée whose main theme is the art of textiles. The Welcome Dinner is a seated buffet meal, ongoing until 10:30 pm. Show up when you wish and taste our right-out-of-the-oven focaccia and the other delicacies that are prepared on the spot. During the dinner it will be also possible to visit the museum’s room dedicated to one peculiar type of textile that was created in : denim.

DRESS CODE: Denim

FRIDAY 6TH SEPTEMBER Conference dinner The Countryside - A Zero-KM dinner

On Friday, September 6th, we are hiking to all the way to the mountainside behind Genoa for a charming dinner with a country flavour, at Agriturismo E-Reixe (Farmhouse restaurant The Roots), located in the village of Sant’Olcese. An old-fashioned torpedone will take us up there, and you will get to taste the delicacies of , hand made (and home grown) in the Agriturismo.

DRESS CODE: Smart casual, flat shoes preferred

SATURDAY 7TH SEPTEMBER Arrivederci dinner Vitamin Sea

On Saturday, September 7th, we will say Arrivederci with a chic dinner on the seaside, in the village of Boccadasse, one of the most peculiar fishing villages of the Genoa Area. The restaurant boasts a set of spectacular glass windows facing right onto the sea, where we will taste a fish-based menu while listening to the waves crashing by.

DRESS CODE: Laid-back chic

Please reserve your dinners at the Registration Desk FACULTY

V.R. ARRUDA ( USA ) J. CRAWLEY ( UK ) University of Pennsylvania - Perelman School of Medicine - The Centre for Haematology, Imperial College London, London, UK Children’s Hospital of Philadelphia, Philadelphia, USA

C. DENIS ( FRANCE ) M. BIFFONI ( ITALY ) Institut National de la Santé et de la Recherche Médicale National Institute of Health, Rome, Italy (INSERM) U1176, Le Kremlin-Bicetre, France

N. BISWAS ( USA ) G. DI MINNO ( ITALY ) Gene and Cell Therapy Group - Wells Center for Pediatric Clinical and Experimental Medicine, University of Naples Research, Indianapolis, USA Federico II, Naples, Italy

A. CANTORE ( ITALY ) J. EIKENBOOM ( THE NETHERLANDS ) Vita-Salute San Raffaele University, Milan, Italy Leiden University Medical Center, Leiden, The Netherlands

G. CASTAMAN ( ITALY ) C. ESCURIOLA ( GERMANY ) Center for Bleeding Disorders and Coagulation, Careggi Haemophilia Centre Rhein Main - (HZRM), Frankfurt, Germany University Hospital, , Italy

A.B. FEDERICI ( ITALY ) P. CHOWDARY ( UK ) University of Milan, Hospital L. Sacco, University ASST KD Haemophilia and Thrombosis Centre, Royal Free London Fatebenefratelli-Sacco, Milan, Italy NHS Foundation Trust, London, UK

K. FIJN VAN DRAAT ( THE NETHERLANDS ) N. CIAVARELLA ( ITALY ) University of Amsterdam, Amsterdam, The Netherlands Hemophilia and Thrombosis Center of the Bari University Hospital; Technical Panel on Thrombosis of the Apulia Region, Bari, Italy S. GARATTINI ( ITALY ) Institute “Mario Negri” - IRCCS, Milan, Italy

M. COLOMBO ( ITALY ) Humanitas Research Hospital, Milan, Italy A. HILGER ( GERMANY ) Federal Institute for Vaccines and Biomedicines, Langen, Germany

P. COPPO ( FRANCE ) French reference Centre for Thrombotic Microangiopathies, J. HUNTINGTON ( UK ) Saint-Antoine Hospital, Paris, France University of Cambridge, Cambridge Institute for Medical Research, Cambridge, United Kingdom A. IORIO ( CANADA ) F. PEYVANDI ( ITALY ) Mc Master University, Hamilton, Canada Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy C. KÖNIGS ( GERMANY ) Klinische und molekulare Hämostaseologie, Klinik für Kinder- und Jugendmedizin, Universitätsklinikum Frankfurt, Frankfurt, S. PIPE ( USA ) Germany University of Michigan, Special Coagulation Laboratory, Michigan, USA

S. LACROIX-DESMAZES ( FRANCE ) Institut National de la Santé et de la Recherche Médicale K. PRATT ( USA ) (INSERM), UMRS 1138, Centre de Recherche des Cordeliers Uniformed Services University of the Health Sciences, Maryland, USA Paris, France

E. SANTAGOSTINO ( ITALY ) P.J. LENTING ( FRANCE ) President of Italian Association of Hemophilia Centres (AICE), Inserm U770, Le Kremlin-Bicêtre, France Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy D. LILLICRAP ( CANADA ) Richardson Laboratory, Queen’s University, Kingston, Canada D. SCOTT ( USA ) Uniformed Services University of Health Sciences, Bethesda, USA J.A. LÓPEZ ( USA ) Bloodworks Northwest Research Institute, University of Washington, School of Medicine, Seattle, USA W. SCHRAMM ( GERMANY ) Universität München - Rudolf Marx Stiftung, München, Germany

M. MAKRIS ( UK ) University of Sheffield, Sheffield Haemophilia and Thrombosis M. SCULLY ( UK ) Centre, Sheffield, UK University College Hospital, London, UK

I. MANCINI ( ITALY ) A. SHAPIRO ( USA ) University of Milan and Luigi Villa Foundation, Milan, Italy Indiana Hemophilia & Thrombosis Center, Indianapolis, USA

P.M. MANNUCCI ( ITALY ) A. TRIPODI ( ITALY ) Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Humanitas University, Angelo Bianchi Bonomi Hemophilia and Milan, Italy Thrombosis Center, IRCCS Maggiore Hospital Foundation, Milan, Italy

M.E. MANCUSO ( ITALY ) Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, E. TUDDENHAM ( UK ) Milan, Italy University College London, Royal Free Hospital Trust, London, UK

A. MAST ( USA ) K. VANHOORELBEKE ( BELGIUM ) Medical College of Winsconsis, Wauwuatosa, USA Campus Kulak Kortrijk, KU Leuven Kortrijk, Belgium

M. MORFINI ( ITALY ) J. VOORBERG ( THE NETHERLANDS ) Associazione Italiana Centri Emofilia (AICE), Florence, Italy Sanquin Research and University of Amsterdam, Amsterdam, The Netherlands Conference Venue: Palazzo Ducale Piazza Giacomo Matteotti, 9 - Genoa (Preferred entrance: Piazza De Ferrari)

CME accreditation: Italian Continuing Medical Education The Conference has been accredited for Italian CME: event n. 266836 (20 credits). Professions involved: Physician (Allergology, Hematology, Genetics, Internal Medicine, Nephrology, Orthopedics, Transfusion Medicine, Clinical Pathology), Biologist (Biologist). EACCME Accreditation The Conference has been accredited by the European Accreditation Council for Continuing Medical Education (EACCME®) with 18 European CME credits (ECMEC®s). Each medical specialist should claim only those hours of credit that he/she actually spent in the educational activity. Through an agreement between the Union Européenne des Médecins Spécialistes and the American Medical Association, physicians may convert EACCME® credits to an equivalent number of AMA PRA Category 1 CreditsTM. Information on the process to convert EACCME® credit to AMA credit can be found at www.ama-assn.org/education/earn-credit-participation- international-activities. Live educational activities, occurring outside of Canada, recognised by the UEMS-EACCME® for ECMEC®s are deemed to be Accredited Group Learning Activities (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada.

EthicalMedTech Compliance MedTech Europe Code of Ethical Business Practice: Compliant. Reference number: EMT18465

Italian CME accreditation managed by:

EMS GROUP Euro Medical Service S.r.l. Provider ECM ID 3765 Email: [email protected] - www.emsgroup.it

International Accreditation and EthicalMedTech Compliance managed by:

SMC Media srl Via Giovenale, 7 - Milan - Italy [email protected] - www.smc-media.com

On-site secretariat is open at:

Thurdsay 5 Sept. Friday 6 Sept. Saturday 7 Sept. Sunday 8 Sept. 18:00-20:00 7:30-18:30 8:30-19:00 8:30-18:00

Staff may be available beyond these hours and dates for urgent matters 10TH BIC INTERNATIONAL CONFERENCE

10TH BIC INTERNATIONAL CONFERENCE

WITH THE CONTRIBUTION OF

MAJOR SPONSORS

SPONSORS

ORGANIZING SECRETARIAT: SMC Media Srl Phone: +39 02 8341 9430/1 Email: [email protected] - [email protected]

Liberate Life builds on our dedication to opening up opportunities for people living with haemophilia by individualising treatment for increased protection. Liberate Life is about living life beyond haemophilia, and celebrating life without compromise.

At Sobi, we are transforming the lives of people a ected by rare diseases. We have a wealth of experience in haemophilia and are dedicated to positive change and long-term commitment to transforming the lives of people a ected by it.

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Sobi is a trademark of Swedish Orphan Biovitrum AB (publ) © 2019 Swedish Orphan Biovitrum AB (publ) – All rights reserved Swedish Orphan Biovitrum AB (publ) SE-112 76 Stockholm, Sweden +46(0)8 697 20 00 Date of preparation: August 2019. NP-8414