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ISTH Webinar: Non-Factor Therapies in Hemophilia
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ISTH Webinar: Non-Factor Therapies in Hemophilia

When: Wednesday, March 28, 2018
19:00 UTC

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Non-Factor therapies in hemophilia 

ISTH Clinical Core Curriculum Competencies: 6.1, 6.4, 6.7, 6.12, 6.13, 6.18, 6.20

March 28, 2018 19:00 - 20:00 UTC (15:00 EDT)


Steven Pipe "Basic science aspects of non-factor therapies in hemophilia"

  • This presentation will review current landscape of available treatment options for hemophilia with and without inhibitors and unmet needs.  It will particularly focus on newly approved and emerging therapeutics which do not rely on traditional factor replacement or bypassing activity. Key preclinical findings and clinical trial results including safety and efficacy data will be reviewed as well as consensus recommendations on their clinical implementation.

Guy Young "Clinical aspects of non-factor therapies in hemophilia"

  • This presentation will review the results of clinical trials for emicizumab, fitusiran and concizumab briefly and then engage in a clinical discussion regarding how such drugs will be implemented in patients with hemophilia. Important aspects of efficacy, safety, and actual clinical implementation will be discussed in detail.

Moderator: Pedro Las Casas




Guy Young

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 a 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 current 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 current 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.

Steven Pipe

Dr Steven Pipe is a Professor and the Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases, as well as Professor of Pathology at the University of Michigan, Ann Arbor, Michigan, USA.  He is the medical director of the Pediatric Hemophilia and Coagulation Disorders Program and medical director of the Special Coagulation Laboratory at the University of Michigan. His clinical interests include bleeding and thrombotic disorders and congenital vascular anomalies. Dr Pipe also directs a basic research lab investigating coagulation factor VIII and the molecular mechanisms of hemophilia A. 

Dr Pipe completed his undergraduate medical training at the University of Toronto, Ontario, Canada, and his pediatric training at McMaster University in Hamilton, Ontario. His pediatric hematology and oncology training was completed at the University of Michigan.  He was a fellow of the Pediatric Scientist Development Program

Dr Pipe is certified by the American Board of Pediatrics in pediatric hematology/oncology. He was awarded a Young Investigator Award from both the American Society of Pediatric Hematology and Oncology and the International Society on Thrombosis and Haemostasis, and a Faculty Scholar Award from the American Society of Hematology.  He was the 2015 recipient of the Leadership in Research Award from the National Hemophilia Foundation.  He has served on the Board of Directors for the Hemostasis and Thrombosis Research Society, as Chair of the Board of Directors for the American Thrombosis and Hemostasis Network and currently the Chair of the Medical and Scientific Advisory Committee to the National Hemophilia Foundation. 

Pedro Las Casas

Pedro Las Casas is a medical doctor and Hematology and Hemotherapy fellow at Universidade Federal de Minas Gerais, in Belo Horizonte, Brazil. He has created a network in Latin America among early career professionals to improve the exchange of knowledge in several ways. His areas of research interest include thromboprophylaxis in oncology patients and the incidence of venous thromboembolism, and the role of immune tolerance induction in hemophilia patients. He has been a member of the ISTH Early Career Task Force since the ISTH Congress in Toronto, 2015, and is proud to be part of the Task Force. He hopes to contribute by reaching and involving many other early career professionals in the field of thrombosis and hemostasis, particularly in Latin America, with the ISTH and its activities.

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