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Recording

View the recording below:

2023 International Prophylaxis Study Group (IPSG) Virtual Symposium was held on April 19th.

Management of Previously Untreated Patients (PUPs) with severe hemophilia in the era of novel hemostatic therapies

Itinerary

  1. Introduction

    Chair: Dr.Guy Young

  2. Interim results of the HAVEN 7 study

    Samantha Gouw, MD PhD

    Clinician - Researcher, Department of Pediatric Hematology at the Amsterdam Netherlands University Medical Center

    Clinician - Researcher, Department of Pediatric Hematology at the Amsterdam Netherlands

  3. The Pros and Cons of using Emicizumab in PUPs

    Prof. Gili Kenet, MD

    Director, National Hemophilia Center, Institute of Thrombosis & Hemostasis Sheba Medical Center; Head Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University Israel

    Director, National Hemophilia Center, Institute of Thrombosis & Hemostasis Sheba Medical Center; Head Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University

  4. To add or not to add FVIII to Emicizumab treatment in PUPs

    Robert F. Sidonio, Jr. MD, MSc.

    Associate Professor of Pediatrics, Division of Hematology/Oncology Associate Director of Hemostasis and Thrombosis Aflac Cancer and Blood Disorders Center Children’s Healthcare of Atlanta/Emory University School of Medicine USA

    Associate Professor of Pediatrics, Division of Hematology/Oncology Associate Director of Hemostasis and Thrombosis Aflac Cancer and Blood Disorders Center Children’s Healthcare of Atlanta/Emory University School of Medicine

  5. Panel Discussion

    Panelists: Gili Kenet, Robert Sidonio, Samantha Gouw, Guy Young

  6. Closing Remarks

    Chair: Dr.Guy Young

Meet Your Speakers

Dr.Guy Young

Co-Chair

Guy Young, MD

Guy Young, MD, received his medical degree from the State University of New York at Stony Brook School of Medicine in Stony Brook, New York. He completed a residency in pediatrics at Schneider Children’s Hospital at Long Island Jewish Medical Center, Albert Einstein College of Medicine in New York, and a fellowship in pediatric hematology/oncology at Children’s National Medical Center, George Washington University School of Medicine in Washington, D.C.

Dr. Young is the Director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles and an Associate Professor of Pediatrics at the University of Southern California’s Keck School of Medicine.

His current research activities encompass clinical trials in hemophilia, the use of novel anticoagulants in children, and the development of the global hemostasis assays to monitor the effects of medications used to treat bleeding in hemophilia.

Dr. Young has published many journal articles, abstracts, and textbook chapters and has delivered lectures, workshops, and presentations at national and international meetings. He is a past Board member and past chair of the program committee for the American Society of Pediatric Hematology/Oncology, and also a past chair of the program committee for the Hemophilia and Thrombosis Research Society. He is the former chair of the Scientific and Standardization Committee on Factor VIII, Factor IX, and Rare Bleeding Disorders of the International Society on Thrombosis and Haemostasis and also serves on the Scientific Subcommittee on Hemostasis for the American Society of Hematology. He has been awarded the National Hemophilia Foundation Physician of the Year Award in 2013.

Samantha Gouw, MD PhD

Speaker

Samantha Gouw, MD, PhD

Samantha C. Gouw, MD, PhD is trained as a pediatric hematologist and clinical epidemiologist. She received her PhD doctorate on the etiology of inhibitor development in children with severe hemophilia A at Utrecht University in 2011. In collaboration with a team of international colleagues, she published several papers in Blood and New England Journal of Medicine on genetic and non-genetic risk factors of inhibitor development. She currently works as a clinician-researcher at the Department of Pediatric Hematology at the Amsterdam University Medical Center. She is the coordinating researcher of a nationwide study and biobank in hemophilia patients at the Department of Clinical Epidemiology at Leiden University Medical center, The Netherlands and is a work package leader in the National Symphony Consortium. Her current research focuses on inhibitor development and immune tolerance induction and the quality of hemophilia care, especially e-health and value based health care.

Gili Kenet, MD

Speaker

Gili Kenet, MD

Director of the Israel National Hemophilia Center and Thrombosis Institute, Sheba Medical Center and Director of Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University (located at the Sheba Medical Center).

Gili Kenet is a Pediatric Hematologist, Professor and former Chair of the Hematology Department at the Sackler Medical School, Tel Aviv University.

Professor Kenet received her Medical Degree from the Hebrew University in Jerusalem. Her research projects have been focused on neonatal hemostasis, childhood stroke and pediatric thrombophilia, as well as the research of new therapy modes applied for people with hemophilia and severe bleeding disorders. She was awarded national grants for pediatric hematology and cancer research, study of rFVIIa mode of action, epidemiology of inhibitor evolution in hemophilia and for personalized tailoring of bypass agent therapy, and applying global hemostasis assays. Prof. Kenet pioneered in gene therapy clinical trials in Israel and novel non replacement drugs in Hemophilia.

Professor Kenet is the author of over 300 peer reviewed scientific publications, review articles and book chapters. An active member of the World Federation of Hemophilia, Israeli Society of Pediatric Hematology and Oncology, and the American Society of Hematology, an active reviewer for high impact thrombosis journals and an editorial board member of the Haemophilia journal. Former Chairperson of the Israeli Society of Thrombosis and Hemostasis, chairperson of the Pediatric and Peri-natal Hemostasis Scientific Subcommittee of ISTH and co-chair of the FVIII/FIX and rare bleeding disorders of the SSC of ISTH.

She was recently appointed as director of the PedNet research foundation, an international pediatric hemophilia registry and data base.

Robert F. Sidonio, Jr. MD, MSc.

Co-Chair, Speaker

Robert F. Sidonio, Jr. MD, MSc.

Dr. Sidonio graduated from UAB medical school, completed his pediatric residency at the University of Louisville and completed his fellowship at the University of Pittsburgh where he also obtained his Masters in Clinical Investigation. Dr. Sidonio has been the Associate Director of Hemostasis and Thrombosis at Emory University since 2014. He is also the co-creator of the Atlanta Protocol which combines Emicizumab and FVIII for ITI. He is the co-PI of the Hemophilia of Georgia VWD project focused on characterization of children with low VWF. He is also the lead PI for the Emicizumab PUP and Nuwiq ITI trial, MOTIVATE study, ATHN 9 study (severe VWD) and FEIBA SAFE study.