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Short Talks - Industry Thrombosis - No CME
  Friday, April 5, 2024           10:30 - 11:30 AM
Moderators
Vinai Bhagirath, Amaya Llorente
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Presentations
10:30 SAFETY AND EFFICACY OF LONG-TERM TREATMENT OF TYPE 1 PLASMINOGEN DEFICIENCY PATIENTS WITH INTRAVENOUS PLASMINOGEN REPLACEMENT THERAPY
Amy D Shapiro1, Heather McDaniel2, Robert W Decker3, Jeremy Lorber3, Karen Thibaudeau4, Joseph M Parker5. 1Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA 2Vanderbilt University, Nashville, TN, USA 3Cedars Sinai Medical Center, Los Angeles, CA, USA 4Kedrion, Laval, Canada 5Kedrion, Fort Lee, NJ, USA
10:45 EFFICACY AND SAFETY IS MAINTAINED IN ADULT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA RECEIVING PEGCETACOPLAN FOR UP TO 3 YEARS
Carlos de Castro1, Brian Mulherin2,3, Christopher J. Patriquin4, Veena Selvaratnam5, Raymond Siu Ming Wong6, Richard J. Kelly7, Lisa Tan8,9, Peter Hillmen10, Dale Zhang10, Regis Peffault de Latour11,12. 1Duke University, Durham, NC, USA 2Hematology Oncology of Indiana, Indianapolis, IN, USA 3Ascension St. Vincent Carmel, Carmel, IN, USA 4University Health Network, Toronto, ON, Canada 5Ampang Hospital, Ampang, Malaysia 6The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong, Hong Kong 7Department of Haematology, St. James’s University Hospital, Leeds, United Kingdom 8Swedish Orphan Biovitrum AB, Stockholm, Sweden 9Lisa Tan Pharma Consulting Ltd., Cambridge, United Kingdom 10Apellis Pharmaceuticals, Inc., Waltham, MA, USA 11French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France 12Université Paris Cité, Paris, France
11:00 TOLERABILITY AND PHARMACODYNAMIC EFFECT OF REGN9933, A MONOCLONAL ANTIBODY DIRECTED AGAINST THE FACTOR XI APPLE 2 DOMAIN: RESULTS FROM A FIRST-IN-HUMAN STUDY
Ethan Marin1, Guixian Lin1, Hisham Abdallah1, David Gutstein1, Andrew Kordahi2, Karoline Meagher1, Frederic Cauwberghs3. 1Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA 2Regeneron Pharmaceuticals, Inc. (at time of study), Tarrytown, NY, USA 3SGS (at time of study), Antwerp, Belgium
11:15 SAFETY AND EFFICACY OF VALOCTOCOGENE ROXAPARVOVEC GENE TRANSFER FOR SEVERE HEMOPHILIA A: AN UPDATE FROM 4 YEARS AFTER TREATMENT
Andrew D Leavitt1, Johnny Mahlangu2, Emily Symington3, Doris V Quon4, Adam Giermasz5, Nigel S Key6, Steven W Pipe7, Bella Madan8, Sheng-Chieh Chou9, Robert Klamroth10,11, Jane Mason12, Flora Peyvandi13,14, Hua Yu15, Tara M Robinson15, Margareth C Ozelo16. 1Adult Hemophilia Treatment Center, University of California San Francisco, San Francisco, CA, USA 2Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa 3Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom 4Orthopaedic Hemophilia Treatment Center, Los Angeles, CA, USA 5Hemophilia Treatment Center, University of California Davis, Sacramento, CA, USA 6UNC Blood Research Center, University of North Carolina, Chapel Hill, NC, USA 7Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, USA 8Guy’s and St Thomas’ NHS Foundation Trust, London, United Kingdom 9Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan 10Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Germany 11Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany 12Queensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women’s Hospital, Brisbane and University of Queensland, Brisbane, Australia 13Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy 14Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy 15BioMarin Pharmaceutical Inc., Novato, CA, USA 16Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil