HTRS 2021 Scientific Symposium PRELIMINARY VIRTUAL MEETING AGENDA

HTRS Research Colloquium: March 8-9 FWGBD Colloquium: March 9 Multidisciplinary Pre-Conference: March 10-11 Agenda: Day 1 | Day 2 Agenda Agenda: Day 1 | Day 2 HTRS Scientific Symposium: March 10-12 Agenda: Day 1 | Day 2 | Day 3

MONDAY, MARCH 8, 2021

HTRS Research Colloquium: Day 1 – separate registration required Eastern Time Event 12:30 PM – 1:00 PM ET PRE-MEETING NETWORKING “BRING YOUR OWN LUNCH”

1:00 PM ET OPENING REMARKS Wolfgang Bergmeier, PhD, and Margaret V. Ragni, MD, MPH, 2021 Colloquium Co-Chairs 1:05 PM – 1:50 PM ET PLENARY SESSION: “HOW DO I BEGIN, AND WHO IS GOING TO PAY FOR THIS?” RESEARCH FUNDING TRENDS AND OPPORTUNITIES Moderator: Margaret V. Ragni, MD, MPH

Speaker: Andrei Kindzelski, MD, National Institutes of Health (NIH)

1:50 PM – 2:20 PM ET NETWORKING BREAK -OR- MENTORING SESSIONS

2:20 PM – 3:20 PM ET PANEL PRESENTATION: “GETTING A JOB – A RECRUITER’S PERSPECTIVE” Moderator: Wolfgang Bergmeier, PhD 2:20 PM BASIC SCIENCE PERSPECTIVE James Morrissey, PhD, Professor, University of Michigan 2:35 PM CLINICAL SCIENCE PERSPECTIVE Janis Abkowitz, PhD, Professor, Hematology Division Head, University of Washington

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HTRS Research Colloquium: Day 1 – continued Eastern Time Event 2:50 PM TRANSLATIONAL SCIENCE PERSPECTIVE Jorge Di Paola, MD, Professor/Division Chief, Washington University in St. Louis 3:05 PM INDUSTRY PERSPECTIVE Robert Peters, PhD, Sanofi Genzyme 3:20 PM – 4:00 PM ET ROUNDTABLE DISCUSSIONS • Basic Science Tables • Clinical Science Tables • Translational Science Tables • Population Science Tables 4:00 PM ET DAY ONE PROGRAM ENDS Session resumes Tuesday, March 9 at 12:30 PM ET

TUESDAY, MARCH 9, 2021

FWGBD Colloquium on Uterine Hemostasis – separate registration required Eastern Time Event 9:30 AM – 9:45 AM ET CONVENE AND WELCOME Andra H. James, MD, MPH 9:45 AM – 9:55 AM ET UTERINE HEMOSTASIS OVERVIEW 9:55 AM – 10:15 AM ET WOMEN & GIRLS WITH HEMOPHILIA Sarah H. O’Brien, MD, MSc 10:15 AM – 10:30 AM ET BREAK 10:30 AM – 11:00 AM ET MANAGEMENT OF DELIVERY IN WOMEN WITH HEMOPHILIA Andra H. James, MD, MPH 11:00 AM – 11:30 AM ET RISK OF CLOTTING ON HORMONAL THERAPY Naomi Tepper, MD, MPH 11:30 AM – 12:05 PM ET IS IT HELLP SYNDROME? IS IT TTP? IS IT ATYPICAL HUS? DOES IT MATTER? Gowthami Arepally, MD 12:05 – 12:30 PM ET DISCUSSION AND WRAP-UP

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TUESDAY, MARCH 9, 2021 – continued

HTRS Research Colloquium: Day 2 – separate registration required Eastern Time Event 12:30 PM – 1:00 PM ET PRE-MEETING NETWORKING “BRING YOUR OWN LUNCH” 1:00 PM – 1:05 PM ET DAY TWO - OPENING REMARKS Ming Y. Lim, MD/MBBCH, MSCr, and Joshua Muia, PhD, 2021 Colloquium Planning Committee Members 1:05 PM – 1:50 PM ET PLENARY SESSION: “CRITICAL STEPS FOR A SUCCESSFUL CAREER IN ACADEMIC MEDICINE” Moderator: Mary Cushman, MD, MSc

Speaker: Mark T. Gladwin, MD, Jack D. Myers Distinguished Professor and Chair, Chairman of the Department of Medicine, UPMC and the University of Pittsburgh School of Medicine 1:50 PM – 2:10 PM ET BREAKOUT SESSION 1: “PRACTICAL TOOLS EVERYONE NEEDS TO ADVANCE THEIR CAREERS” BREAKOUT 1 SPEAK UP: GIVING EFFECTIVE PRESENTATIONS Discussion Leader: Alisa S. Wolberg, PhD BREAKOUT 2 MANAGING THE K-TO-R TRANSITION: WHAT TO DO IF YOU DON’T GET A K-AWARD Discussion Leader: Nigel Key, MB, ChB, FRCP BREAKOUT 3 FROM GOOD IDEA TO SUBMITTED GRANT Discussion Leader: Gowthami Arepally, MD BREAKOUT 4 BEGIN AGAIN: JUMP START YOUR RESEARCH CAREER AFTER A BREAK Discussion Leader: Menaka Pai, BSc, MSc, MD, FRCPC 2:10 PM – 2:30 PM ET “GET UP AND STRETCH BREAK’ -OR- MENTORING SESSIONS 2:30 PM – 2:50 PM ET BREAKOUT SESSION 2: “PRACTICAL TOOLS EVERYONE NEEDS TO ADVANCE THEIR CAREERS” BREAKOUT 1 SPEAK UP: GIVING EFFECTIVE PRESENTATIONS Discussion Leader: Alisa S. Wolberg, PhD BREAKOUT 2 MANAGING THE K-TO-R TRANSITION: WHAT TO DO IF YOU DON’T GET A K-AWARD Discussion Leader: Nigel Key, MB, ChB, FRCP BREAKOUT 3 FROM GOOD IDEA TO SUBMITTED GRANT Discussion Leader: Gowthami Arepally, MD BREAKOUT 4 BEGIN AGAIN: JUMP START YOUR RESEARCH CAREER AFTER A BREAK Discussion Leader: Menaka Pai, BSc, MSc, MD, FRCPC

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HTRS Research Colloquium: Day 2 – continued Eastern Time Event 2:50 PM – 3:35 PM ET PANEL PRESENTATION: “OVERCOMING OBSTACLES: MOVING YOUR CAREER FORWARD” Moderator: Rodney Camire, PhD 2:50 PM BASIC/TRANSLATIONAL SCIENCE PERSPECTIVE Veronica H. Flood, MD 3:05 PM CLINICAL SCIENCE PERSPECTIVE Margaret V. Ragni, MD, MPH 3:20 PM INDUSTRY PERSPECTIVE Marcus Carr, MD 3:35 PM – 4:20 PM ET CLOSING RECEPTION “BRNG YOUR OWN SNACKS 4:20 PM ET DAY TWO PROGRAM ENDS

WEDNESDAY, MARCH 10, 2021

Multidisciplinary Pre-Conference: Day 1 – separate registration required Eastern Time Event

10:00 AM – 10:05 AM ET DAY ONE - OPENING REMARKS Loren D’Angelo, RN-BC, BSN, MSN, CPNP-PC, HTRS 2021 Planning Committee

10:05 AM – 11:30 AM ET PRE-CONFERENCE SESSION 1: “NEW THERAPIES” Moderators: Loren D’Angelo, RN-BC, BSN, MSN, CPNP-PC, and Caitlin Moncrieff, NP

10:05 AM DOACS IN THE PEDIATRIC POPULATION Riten Kumar, MD, MSc

10:35 AM MANAGING INHIBITORS IN THE REALM OF NON-FACTOR THERAPIES James Munn, RN-BC, BS, BSN, MS

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Multidisciplinary Pre-Conference: Day 1 – continued 10:55 AM “GENE THERAPY CLINICAL TRIAL EXPERIENCES IN CARE” Darcy Phelan, NP, MSN

11:20AM Live Q&A

11:30 AM DAY ONE PROGRAMMING ENDS Session resumes Thursday, March 11 at 10:00 AM ET

HTRS Scientific Symposium: Day 1 Eastern Time Event

12:00 PM – 1:00 PM ET SATELLITE SYMPOSIA: (2) Concurrent Sessions Sanofi Genzyme: "siRNA Technology in Hemophilia" CSL Behring: “On-Demand Bleed Resolution in Hemophilia B With an Extended Half-Life Factor IX Therapy”

1:00 PM – 1:05 PM ET OPENING REMARKS Jordan Shavit, MD, PhD, HTRS 2021 Planning Committee Chair Ayesha N. Zia, MD, HTRS 2021 Planning Committee Vice Chair

1:05 PM – 2:30 PM ET SYMPOSIUM SESSION 1 DEBATE: “THE FUTURE OF HEMOPHILIA THERAPEUTICS: SUBSTITUTION OR GENE THERAPY (To GT or not to GT, a smackdown)” Moderators: Jordan Shavit, MD, PhD and Yasmina Abajas, MD Speakers: Denise E. Sabatino, PhD, Michael Callaghan, MD, and Shawn Jobe, MD, PhD 1:05 PM – 1:10 PM Case Presentation & Introduction to Audience Response System (“ARS”) Jordan Shavit, MD, PhD and Yasmina Abajas, MD 1:10 PM – 1:20 PM Introduction: “The science behind hemophilia therapeutics” Denise E. Sabatino, PhD

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HTRS Scientific Symposium: Day 1 – continued

SYMPOSIUM SESSION 1 DEBATE: “THE FUTURE OF HEMOPHILIA THERAPEUTICS: SUBSTITUTION OR GENE THERAPY (To GT or not to GT, a smackdown)”

1:20 PM – 1:25 PM Pre-Debate ARS Vote Jordan Shavit, MD, PhD and Yasmina Abajas, MD

1:25 PM – 1:40 PM Argument for Substitution Michael Callaghan, MD

1:40 PM – 1:55 PM Argument for Gene Therapy Shawn Jobe, MD, PhD

1:55 PM – 2:05 PM Rebuttal for Substitution Michael Callaghan, MD

2:05 PM – 2:15 PM Rebuttal for Gene Therapy Shawn Jobe, MD, PhD

2:15 PM – 2:25 PM Questions/Discussion Jordan Shavit, MD, PhD and Yasmina Abajas, MD

2:25 PM – 2:30 PM Post-Debate ARS Vote Jordan Shavit, MD, PhD and Yasmina Abajas, MD

2:30 PM – 3:30 PM ET NETWORKING & EXHIBITOR SPEED BREAK

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HTRS Scientific Symposium: Day 1 – continued 3:30 PM – 4:30 PM ET ORAL ABSTRACT PRESENTATIONS: (2) Concurrent Sessions

vWF/HEMOPHILIA A Novel Mechanisms & Trials Moderators: Christine Kempton, MD, MSc Moderators: A. Valance Washington, PhD and Joshua Muia, PhD and Lauren Amos, MD

3:30 PM “THE IMPORTANCE OF GENETIC TESTING IN DIAGNOSING VON “PLATELETS FACTOR 4 (PF4) BINDING ENHANCES IN VITRO WILLEBRAND'S DISEASE TYPE 2M” NEUTROPHIL EXTRACELLULAR TRAPS (NET) CAPTURE OF SARS- Presenter: Radia Ksayer, MD COV-2: THERAPEUTIC IMPLICATIONS” Presenter: Maki Ishizuka, MD

3:45 PM “VON WILLEBRAND FACTOR (VWF) MULTIPLEX ACTIVITY ASSAY “IN VITRO-GROWN MEGAKARYOCYTES (MKS) TAKE UP THE DIFFERENTIATION OF LOW VWF/TYPE 1 VON WILLEBRAND ECTOPIC PROTEIN UROKINASE (UPA) AND THE RESULTING DISEASE (VWD) AND VARIANT VWD: ANALYSIS FROM THE PLATELETS (PLTS) CAN RELEASE THE UPA AT SITES OF VASCULAR COMPARATIVE EFFECTIVENESS IN THE DIAGNOSIS OF VWD” INJURY” Presenter: Sergei V. Zaitsev, PhD Presenter: Jonathan C. Roberts, MD

4:00 PM Non-CME Presentation: “BROAD PATIENT ELIGIBILITY AND “BLEEDING DIATHESIS IN MICE LACKING JAK2 IN PLATELETS” LONG-TERM TOLERABILITY IN THE FIRST-IN-HUMAN GENE Presenters: Nathan Eaton, BSc THERAPY STUDY OF AAVHU37.HFVIIICO IN SEVERE HEMOPHILIA A” Presenter: Francesca Ferrante, MD

4:15 PM Non-CME Presentation: “REAL-WORLD DATA FROM A US “A RANDOMIZED, PRAGMATIC TRIAL TO ASSESS A PEDIATRIC CLAIMS DATABASE AMONG PATIENTS WITH HEMOPHILIA A REAL-TIME RISK PREDICTION MODEL FOR THROMBOTIC EVENTS” TREATED WITH RECOMBINANT FVIII FC FUSION PROTEIN” Presenters: Shannon C. Walker, MD Presenter: Amanda Wilson, MPH, PhD

4:30 PM - 5:30 PM ET OPEN NETWORKING

5:30 PM - 6:30 PM ET SATELLITE SYMPOSIA: (1) Session Takeda: “ADVATE® [Antihemophilic Factor (Recombinant)] Clinical Presentation”

6:30 PM ET DAY ONE PROGRAMMING ENDS General Session resumes Thursday, March 11 at 12:00 PM ET

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THURSDAY, MARCH 11, 2021

Multidisciplinary Pre-Conference: Day 2 – separate registration required Eastern Time Event

10:00 AM – 10:05 AM ET DAY TWO - OPENING REMARKS Loren D’Angelo, RN-BC, BSN, MSN, CPNP-PC, HTRS 2021 Planning Committee

10:05 AM – 11:30 AM ET PRE-CONFERENCE SESSION 2: INCLUSIVE CARE Moderators: Mary Lesh, PNP, MSN and Caitlin Moncrieff, NP

10:05 AM TRAUMA INFORMED CARE FOR HEMATOLOGY PROVIDERS Amanda Stahl, LICSW

10:35 AM HEMATOLOGIC CONSIDERATIONS IN THE CARE OF TRANSGENDER PATIENTS Meredith Russel, PNP, MSN

11:15 AM Live Q&A

11:30 AM ET HTRS 2021 Multidisciplinary Pre-conference Ends

HTRS Scientific Symposium: Day 2 Eastern Time Event

12:00 PM – 1:00 PM ET SATELLITE SYMPOSIA: (2) CONCURRENT SESSIONS Novo Nordisk: “A rFVIIa with over 30 years of experience with treating hemophilia and other rare bleeding disorders” Bayer: “Patient focused prophylaxis management for patients with hemophilia: a case-based discussion”

1:00 PM – 1:05 PM ET OPENING REMARKS Gowthami Arepally, MD, HTRS 2021 Planning Committee – Abstract Co-Chair Alan Mast, MD, PhD, HTRS 2021 Planning Committee – Abstract Co-Chair

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HTRS Scientific Symposium: Day 2 – continued 1:05 PM – 2:10 PM ET SYMPOSIUM SCIENTIFIC SESSION 2: STATE-OF-THE-ART LECTURE: “COVID-19: THROMBOSIS AND CLINICAL CONSIDERATIONS” Moderator: Jordan A. Shavit, MD, PhD and Andrea Obi, MD, Co-moderator Speakers: Roger Lewis, MD, Robert Campbell, PhD, and Hanny Al-Samkari, MD

2:10 PM - 3:10 PM ET EXCLUSIVE SATELLITE SYMPOSIUM GENENTECH: “SubQ Prophylaxis for Hemophilia A: 3 Experts Discuss the Latest Data From the Pivotal Trials”

3:10 PM – 4:10 PM ET ORAL ABSTRACT PRESENTATIONS: (2) Concurrent Sessions

THROMBOSIS HEMOPHILIA B Moderators: Hervé Falet, PhD Moderators: Lacramioara Ivanciu, PhD and Rachel Rosovsky, MD and Janice Staber, MD

3:10 PM “INVESTIGATING THE REGULATION OF RAP1-INTEGRIN “AMT-060 GENE THERAPY IN ADULTS WITH SEVERE OR SIGNALING PATHWAY USING THE IMMKCL MEGAKARYOCYTE MODERATE-SEVERE HEMOPHILIA B CONFIRM STABLE FIX MODEL” Presenter: Ankita Dhenge, PhD EXPRESSION AND SUSTAINED REDUCTIONS IN BLEEDING AND FACTOR IX USE FOR UP TO 5 YEARS” Presenter: Frank W.G Leebeek, MD, PhD

3:25 PM “FORMATION OF PROTHROMBINASE ON HUMAN ENDOTHELIAL “ETRANACOGENE DEZAPARVOVEC (AAV5-PADUA HFIX CELLS” Presenter: Clay T. Cohen, MD VARIANT), AN ENHANCED VECTOR FOR GENE TRANSFER IN ADULTS WITH SEVERE OR MODERATE-SEVERE HEMOPHILIA B: TWO YEAR DATA FROM A PHASE 2B TRIAL” Presenter: Annette von Drygalski, MD, PharmD

3:40 PM “HEMATOLOGIC ABNORMALITIES AND VENOUS “EFFICACY AND SAFETY OF ETRANACOGENE DEZAPARVOVEC IN THROMBOPROPHYLAXIS IN MIS-C: DATA FROM A MULTI- ADULTS WITH SEVERE OR MODERATE-SEVERE HEMOPHILIA B: DISCIPLINARY WORKING GROUP AT A PEDIATRIC TERTIARY CARE FIRST DATA FROM THE PHASE 3 HOPE-B GENE THERAPY TRIAL” HOSPITAL” Presenter: Lynn M. Malec, MD, MSc Presenter: Steven W. Pipe, MD

3:55 PM “RISK OF VENOUS THROMBOEMBOLISM IN PEDIATRIC Non-CME Presentation: “CLEARANCE OF FIDANACOGENE HOSPITALIZED PATIENTS UNDERGOING NON-CARDIAC SURGERY, ELAPARVOVEC VECTOR DNA IN PATIENTS WITH SEVERE OR A REPORT FROM THE CHILDREN'S HOSPITAL-ACQUIRED MODERATELY SEVERE HEMOPHILIA B (HB)” THROMBOSIS (CHAT) CONSORTIUM” Presenter: Patrick F. Fogarty, MD Presenter: Elizabeth T. Stephens, DO

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HTRS Scientific Symposium: Day 2 – continued

4:10 PM – 4:15 PM ET TRANSITION TO REMO

4:15 PM – 5:15 PM ET POSTER PRESENTATIONS + SPEED SESSIONS • “A Survey of Anti-platelet and Use in Persons with Hemophilia” Presenter: Maissaa Janbain, MD, MSCR • “Bleeding Disorder Referrals to Hematology Clinic: A Single Institution Experience” Presenter: Zuri Hudson, DO • “Catastrophic Thrombotic Syndrome in Systemic Lupus Erythematosus without Antiphospholipid Antibodies” Presenter: Krystal J. Park, MD • “Drive-through Anticoagulation Clinic During COVID-19 Pandemic” Presenter: Vilmarie Rodriguez, MD • “ECMO Blood Product Utilization: The First 24 Hours” Presenter: Anusha Chidharla, MD • “Eculizumab Treatment for Refractory Thrombosis in Antiphospholipid Syndrome” Presenter: Habiba Hussain, MD • “Efficacy and Safety of is Sustained for up to ≥6 Years of Observation in Patients Aged 12– < 18 years at Enrollment into PROTECT VIII” Presenter: Jonathan Ducore, MD, MPH • “Gastrointestinal Bleeding in Patients with von Willebrand Disease: Treatments and Outcomes” Presenter: Jonathan C. Roberts, MD • “Implementation and Analysis of a Venous Thromboembolism Prophylaxis Algorithm in Hospitalized Pediatric Patients” Presenter: Nora M. Gibson, MD • “NHF Builds a Community-Driven National Research Agenda for Inherited Bleeding Disorders” Presenter: Michelle L. Witkop, DNP, FNP-BC • “Perceptions about the use and efficacy of Extended Half-Life (EHL) factor products in persons with hemophilia: A national survey of providers from Hemophilia Treatment Centers in the United States” Presenter: Neeraja Swaminathan, MBBS • “Single Center Experience of Bleeding Evaluation in Hemophilia Carriers using ISTH-BAT” Presenter: Maria Carter Febres, MD • “Successful Implementation of a Pediatric Bleeding Assessment Tool in the Electronic Medical Record in an Academic Clinical Setting” Presenter: Kelly Bajorek, BS • “Surgical Procedures in Patients with Hemophilia: Multistakeholder Perspectives From 2018 Roundtables” Presenter: Miguel A. Escobar, MD • “Telegenetic Counseling for Female My Life, Our Future Participants” Presenter: Maria E. Santaella, RN-BC, MSN, CPHON • “Thromboprophylaxis to Improve Outcomes in COVID-19: An Indian Expert Consensus” Presenter: Agam Vora, MD • “Unprovoked extensive deep vein thrombosis in a pediatric patient with sickle cell disease and duplicated inferior vena cava” Presenter: Ramya Ramakrishnan, MD • “Venous Thromboembolic Disease in Children Associated with COVID-19” Presenter: Shalu Narang, MD MPH • Non-CME Presentation: “Single-dose , Efficacy, and Safety of Concentrate for On-Demand Treatment of Acute Bleeding and Surgical Prophylaxis in Patients with Congenital Fibrinogen Deficiency” Presenter: Bruce Schwartz, PhD

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HTRS Scientific Symposium: Day 2 – continued 5:15 PM – 6:15 PM ET OPEN NETWORKING

6:15 PM – 7:15 PM ET SATELLITE SYMPOSIA: 2 Concurrent Sessions Takeda/PER (CME): “Putting the Patient First: Individualized Strategies for the Treatment and Management of von Willebrand Disease” Novo Nordisk: “Double Feature: Obesity in Hemophilia and a Treatment Option for Hemophilia A”

7:15 PM DAY TWO PROGRAMMING ENDS General Session resumes Friday, March 12 at 12:00 PM ET

FRIDAY, MARCH 12, 2021

HTRS Scientific Symposium: Day 3 Eastern Time Event

12:00 PM – 1:00 PM ET SATELLITE SYMPOSIA: (2) Concurrent Sessions Sanofi Genzyme: "A Breakthrough in aTTP Treatment: Now recommended by the ISTH Guidelines" Alnylam Pharmaceuticals: Introduction to GIVLAARI® (givosiran) for the Treatment of Adults with Acute Hepatic Porphyria - Featuring 18-month data from the ENVISION Open-Label Extension (OLE) Study

1:00 PM – 1:05 PM ET OPENING REMARKS Jordan Shavit, MD, PhD, HTRS 2021 Planning Committee Chair Ayesha N. Zia, MD, HTRS 2021 Planning Committee Vice Chair

1:05 PM – 2:10 PM ET SCIENTIFIC SESSION 3: “BENCH, CLINIC, AND EVERYTHING IN-BETWEEN” (3) Concurrent Sessions

Session 3-A: “Platelets” Moderators: Pavel Davizon-Castillo, MD and Jorge Di Paola, MD, Co-moderator

Speakers: Rachael Grace, MD, Jeanne Hendrickson, MD, Kellie Machlus, PhD

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HTRS Scientific Symposium: Day 3 – continued Session 3-B: “Sickle Cell-Associated Thrombosis” Moderators: Courtney Thornburg, MD and Deirdre S. Nolfi-Donegan, MD, Co-moderator

Speakers: Morey Blinder, MD, Erica Sparkenbaugh, PhD, Huanghe Yang, PhD

Session 3-C: “Post-VTE Functional Syndromes” Moderators: Ayesha N. Zia, MD and Marisol Betensky, MD, MPH, Co-moderator

Speakers: Susan Kahn, MD, MSc, FRCPC, ABIM, Farouc Jaffer, MD, PhD, Peter Henke, MD

2:10 PM – 2:20 PM ET TRANSITION TO REMO

2:20 PM – 3:20 PM ET NETWORKING & EXHIBITOR SPEED BREAK

3:20 PM – 4:20 PM ET POSTER PRESENTATIONS AND SPEED SESSIONS • “ATHN 16: Safety of Factor VIIa (recombinant)-jncw for the Treatment of Bleeding Events in Patients with Hemophilia A or B with Inhibitors with or without Prophylactic Treatment” Presenter: Michael Callaghan • “Clinical Study to Investigate the Efficacy and Safety of a Von Willebrand Factor FVIII Concentrate (VWF/FVIII) during Prophylaxis in Previously Treated Patients with Von Willebrand Disease (VWD)” Presenter: Robert Sidonio • “Community Voices in Research (CVR); A Patient-Centric Approach Moving the Future of Inherited Bleeding Disorders Forward” Presenter: Rosaura Vidal • "Development & Testing of an Asynchronous Hemophilia Learning Module” Presenter: Tiffany Lucas • “Evaluation of the hemostatic efficacy of eptacog beta (recombinant factor VIIa) for the treatment of bleeding in pediatric subjects ( < 12 years) with hemophilia A or B with inhibitors in PERSEPT 2” Presenter: Steven Pipe • “Examination and Validation of a Patient-Centric Joint Metric: “PROBLEM JOINT”; Empirical Evidence from the CHESS US Dataset” Presenter: Tom Burke • “How to Reduce Hypercoagulability Testing in the Inpatient Setting? A Quality Improvement Initiative” Presenter: James Hall • HTRS Student Research Award: “Development of an immunoproteomic pipeline for the characterization of anti-FVIII • molecular and cellular repertoires in hemophilia A inhibitor patients” Presenter: Robert Chen • HTRS Student Research Award: “Risk Factors for Non-Variceal Hemorrhage in Patients with Chronic Liver Disease“ Presenter: Preethi Kesavan • “Magnetically powered microwheel thrombolysis of occlusive thrombi in zebrafish” Presenter: Jordan Shavit • “Management of Recurrent Disease in Acquired Hemophilia A” Presenter: Aaron Boothby • “Passive Infusion Logging System (PILS): A pilot” Presenter: Maria Santaella 12

HTRS Scientific Symposium: Day 3 – continued • HTRS Student Research Award: “Reduced Health-Related Quality of Life is Associated with Depression and Cognitive Impairment Among Survivors of Thrombotic Thrombocytopenic Purpura” Presenter: Sruthi Selvakumar • “SHORT-TERM OUTCOME of NEONATAL VENOUS THROMBOEMBOLISM for ANTICOAGULATED and OBSERVED PATIENTS” Presenter: Kisha Beg • “The Safety of Eptacog Beta in Hemophilia Patients with Inhibitors in Adult, Pediatric, and Surgical Settings” Presenter: Miguel Escobar • “The value of immature platelet fraction of platelet apheresis products in prediction of platelet transfusion response in thrombocytopenic cancer patients” Presenter: Ekrem Turk • “Warfarin-induced skin necrosis in patients with protein C and S hereditary deficiency. Systematic review” Presenter: Rafael Pichardo-Rodriquez • Non-CME Presentation: “Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open Label Extension” Presenter: David Rees • Non-CME Presentation: Clinical Outcomes in Patients with Acute Hepatic Porphyria Treated with Givosiran Who Stopped Hemin Prophylaxis at Study Entry: A Post-hoc Analysis from the Phase 3 ENVISION Study Through Month 12” Presenter: Herbert Bonkovsky

4:20 PM - 4:30 PM ET INTRODUCTION TO CLOSING EXTRAVAGANZA

4:30 PM - 5:30 PM ET CLOSING EXTRAVAGANZA NETWORKING & THE FIRST-EVER “HTRS TALENT SHOW”

5:30 PM ET HTRS 2021 SCIENTIFIC SYMPOSIUM PROGRAMMING ENDS

Mark your calendars for the next HTRS Scientific Symposium:

March 10 - 12, 2023 Renaissance Orlando at SeaWorld Orlando, FL

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