-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

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322.O2.6 322. Hemophilia A and B: Clinical and Epidemiological: Beyond Efficacy of Hemostatic Agents in Hemophilia: Exploring Pharmacokinetics, Dosing Regimens, and Special Populations

Symposia: Hemophilia A and B: Clinical and Epidemiological Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Maternal Health, Research, Bleeding disorders, Bleeding and Clotting, Adult, Clinical trials, Hemophilia, Clinical Practice (Health Services and Quality), Translational Research, Epidemiology, Clinical Research, Drug-drug interactions, Pediatric, Diversity, Equity, and Inclusion (DEI), Diseases, Real-world evidence, Treatment Considerations, Pregnant, Adverse Events, Study Population, Human, Maternal Health
Saturday, December 7, 2024: 12:00 PM-1:30 PM
Room 29 (San Diego Convention Center)
Moderators:
Ruben Rhoades, MD, Thomas Jefferson University and Sanjay Ahuja, MD, Indiana Hemophilia and Thrombosis Center, Inc.
Disclosures:
Rhoades: Novartis: Consultancy; Spark Therapeutics: Research Funding; Veralox Therapeutics: Research Funding. Ahuja: Sanofi Genzyme: Honoraria, Membership on an entity's Board of Directors or advisory committees; Genentech: Honoraria, Membership on an entity's Board of Directors or advisory committees; BioMarin: Honoraria, Membership on an entity's Board of Directors or advisory committees; CSL Behring: Honoraria, Membership on an entity's Board of Directors or advisory committees; XaTek: Membership on an entity's Board of Directors or advisory committees, Research Funding; Case Western Reserve University: Patents & Royalties: Patent for US 11,408,844 issued; US FDA: Honoraria, Other: Member of the Blood Products Advisory Committee; Governor’s Office, State of Ohio: Other: Membership on Rare Disease Advisory Council for the State of Ohio.

This session will explore how novel therapies may be used in clinical practice and will include data supporting pharmacokinetic based treatment and alternative dosing regimens as well as the use of these therapies in special populations
12:00 PM

Konrad Van Der Zwet1*, Marjon H. Cnossen, MD, PhD2*, Floor CJI Moenen, MD, PhD3*, Saskia EM Schols, MD, PhD4*, Paula F Ypma, MD, PhD5*, Paul L. den Exter, MD, PhD6*, Louise Hooimeijer, MD7*, Michiel Coppens, MD, PhD8,9*, Ron A.A. Mathôt, PhD, MSc10*, Anouk A.M.T. Donners, PhD, MSc11*, Alexander Janssen, MSc12*, Ilmar Kruis, MSc13*, Eduard J. Van Beers, MD, PhD1, Corien L. Eckhardt, MD, PhD1*, Rolf T. Urbanus, PhD, MSc1*, Lize Fie Ditteke Van Vulpen, MD, PhD1*, Roger EG Schutgens, MD, PhD, MSc1* and Kathelijn Fischer, MD, PhD1*

1Center for Benign Hematology, Thrombosis and Hemostasis - Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
2Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, Erasmus University Medical Center, Rotterdam, Netherlands
3Department of Internal Medicine, Hematology, Maastricht University Medical Center+ and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
4Department of Hematology, Radboud University Medical Center, Nijmegen, Netherlands
5Department of Hematology, HagaZiekenhuis, Delft, Netherlands
6Department of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden University, Leiden, Netherlands
7Department of Pediatric Hematology, University Medical Center Groningen, Groningen, Netherlands
8Amsterdam University Medical Centers, Department of Vascular Medicine, University of Amsterdam and Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
9Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
10Department of Clinical Pharmacology, Hospital Pharmacy, Amsterdam University Medical Center, Amsterdam, Netherlands
11Clinical Pharmacy, University Medical Center Utrecht, Utrecht, NLD
12Amsterdam University Medical Center, Amsterdam, Netherlands
13Netherlands Hemophilia Society, Nijkerk, NLD

12:15 PM

Steven W. Pipe, MD1, Kaan Kavakli2*, Tadashi Matsushita, MD3, Huyen Tran, MBBS (Hons), Master Clin Epi, FRACP, FRCPA4, Bulent Zulfikar5*, Laurel Menapace6*, Marja Puurunen, MD, PhD7*, Wenruo Hu8*, Yuqian Shen9*, Chanchala Kaddi6* and Vanessa Salinas10*

1University of Michigan, Ann Arbor, MI
2Division of Hematology, Department of Hematology, Ege University Faculty of Medicine, Izmir, Turkey
3Nagoya University Hospital, Nagoya, Japan
4Department of Clinical Hematology, Alfred Hospital, Melbourne, Australia
5Istanbul University Oncology Institute, Division of Pediatric Hematology-Oncology, Istanbul, Turkey
6Sanofi, Cambridge, MA
7Senior Clinical Research Director, Sanofi, Cambridge, MA
8Sanofi, Beijing, China
9Sanofi, Bridgewater, NJ
10Center for Inherited Blood Disorders, Orange, CA

12:30 PM

Anne de Vaan, MD1*, Marieke JHA Kruip, MD, PhD2*, Jeroen Eikenboom, MD, PhD3, Marieke C Punt, MD, PhD1*, Michiel Coppens, MD, PhD4,5*, Laurens Nieuwenhuizen, MD PhD6*, Saskia EM Schols, MD, PhD7*, Anja BU Mäkelburg, MD, PhD8*, Floor CJI Moenen, MD, PhD9*, Hans JJ Duvekot, Md, PhD10*, Marjolein Peters, MD, PhD11*, Annemieke JM Middeldorp, MD, PhD12*, Kitty WM Bloemenkamp, MD, PhD13*, Roger EG Schutgens, MD, PhD, MSc1*, Titia AT Lely, MD, PhD13* and Karin PM van Galen, MD, PhD1*

1Center for Benign Hematology, Thrombosis and Hemostasis - Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
2Department of Hematology, Erasmus MC, Erasmus University Medical Center Rotterdam, Rotterdam, NLD
3Department of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, Netherlands
4Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
5Amsterdam University Medical Centers, Department of Vascular Medicine, University of Amsterdam and Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
6Department of Internal Medicine, Máxima Medisch Centrum, Eindhoven, Netherlands
7Department of Hematology, Radboud University Medical Center, Nijmegen, Netherlands
8Department of Hematology, University Medical Center Groningen, Groningen, Netherlands
9Department of Internal Medicine, Hematology, Maastricht University Medical Center+ and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
10Department of Gynaecology and Obstetrics, Erasmus MC, Erasmus University Medical Center, Rotterdam, Rotterdam, Netherlands
11Department of Pediatric Hematology, Emma Children's Hospital, University Medical Centers, location AMC, Amsterdam, Amsterdam, Netherlands
12Department of Obstetrics, Leiden University Medical Center, Leiden, Netherlands
13Department of Woman and Baby, University Medical Center Utrecht, Wilhelmina Children's Hospital, Utrecht, Netherlands

12:45 PM

Shalini Vemuru, MD1, Stacey Fedewa, PhD, MPH2* and Christine L Kempton, MD3

1Department of Internal Medicine, Emory University, Johns Creek, GA
2Department of Hematology and Medical Oncology and Hemophilia of Georgia Center for Bleeding & Clotting Disorders at Emory, Emory University, Atlanta, GA
3Emory University, Atlanta, GA

1:00 PM

Maissaa Janbain, MD1*, Miguel A. Escobar, M.D.2*, Manuel Carcao, MD, FRCP(C)3*, Andrew Wilson, MS4*, Jennifer Dumont4, Nathan Ball, BSc5*, Ella Morton, BSc5*, Duygu Bozkaya4*, Anne-Laure Tardy6* and Sylvie Bozzi, MS7*

1Deming Department of Internal Medicine, Section of Hematology and Medical Oncology, Tulane School of Medicine, New Orleans, LA
2Division of Hematology, McGovern Medical School, University of Texas Health Sciences Center, Houston, TX
3Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, Canada
4Sanofi, Cambridge, MA
5Adelphi Real World, Bollington, United Kingdom
6Sanofi, Gentilly, Paris, France
7Sanofi, Gentilly, France

1:15 PM

Sibgha Zaheer, MD, MPH1, Elizabeth Marquez Casas2*, Myew-Ling Toh, MD3* and Guy Young, MD4

1University of Southern California, Keck School of Medicine/Children's Hospital Los Angeles, Los Angeles, CA
2Children's Hospital of Los Angeles, Los Angeles, CA
3Sanofi, Lyon, AL, FRA
4Children's Hospital Los Angeles, Los Angeles, CA

*signifies non-member of ASH