66th ASH Annual Meeting: 322. Hemophilia A and B: Clinical and Epidemiological: Poster II

Symposia: Hemophilia A and B: Clinical and Epidemiological Oral and Poster Abstracts
Poster

Maternal Health, Research, Bleeding and Clotting, Bleeding disorders, Fundamental Science, Clinical trials, Adult, Clinical Practice (Health Services and Quality), Bispecific Antibody Therapy, Hemophilia, Translational Research, Epidemiology, Clinical Research, Diversity, Equity, and Inclusion (DEI), Health disparities research, Pediatric, Diseases, Immune mechanism, Neonatal, Treatment Considerations, Biological therapies, Real-world evidence, Metabolism, Young adult , Biological Processes, Study Population, Human

Halls G-H (San Diego Convention Center)

2582

Cost Comparison of Efanesoctocog Alfa with Existing Factor VIII Replacement Therapies for Major Surgeries in People with Severe Hemophilia a

Janice Staber, MD¹, Alix Arnaud, MS²^*, Ion Agirrezabal, MSc/PhD³^*, Lane Anson, PharmD/MBA/RPh⁴^*, Andrew Wilson, MS⁴^*, Nana Kragh, MSc⁵^*, Doris V. Quon, MD, PhD⁶ and Allison P Wheeler, MD/MSCI⁷

¹Pediatrics, University of Iowa Stead Family Children's Hospital, Iowa City, IA
²Sanofi, Boston, MA
³Sanofi, Barcelona, Spain
⁴Sanofi, Cambridge, MA
⁵Sobi, Stockholm, Sweden
⁶Orthopaedic Institute for Children, Orthopaedic Hemophilia Treatment Center, Los Angeles, CA
⁷University of Washington, Washington Center for Bleeding Disorders, Seattle, WA

2583

Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients with Congenital Hemophilia A with Inhibitors

Midori Shima¹, Katsuyuki Fukutake²^*, Masanori Matsumoto³, Yoshihiko Sugimura⁴^*, Chiaki Sugita⁴^*, Lyu Ji⁴^*, Mika Kawano⁵^* and Tadashi Matsushita, MD⁶

¹Thrombosis and Hemostasis Research Center and Department of Pediatrics, Nara Medical University, Kashihara, Japan
²Department of Blood Coagulation, Ogikubo Hospital, Tokyo, Japan
³Department of Blood Transfusion Medicine and Department of Hematology, Nara Medical University, Kashihara, Japan
⁴Drug Safety Division, Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
⁵Medical Affairs Division, Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
⁶Nagoya University Hospital, Nagoya, Japan

2584

Characterizing an Underdiagnosed Condition: Hemophilia in Females

Valentina Restrepo, MD¹, Stephanie Prozora, MD², Evangelia Louizos³^*, Layla N Van Doren, MD, MBA⁴ and Robert D Bona, MD⁵

¹Section of Hematology, Yale University School of Medicine, New Haven, CT
²Department of Pediatric Hematology & Oncology, Yale University School of Medicine, New Haven, CT
³Hematology Section, Yale School of Medicine, New Haven, CT
⁴Section of Hematology, Division of Medicine, Yale School of Medicine, New Haven, CT
⁵Classical Hematology, Section of Hematology, Yale University School of Medicine, New Haven, CT

2585

Unmet Needs of Patients with Hemophilia a/B with or without Inhibitors: Real-World End-of-Study Results from the explorer6 Non-Interventional Study

Allison P. Wheeler, MD¹, Aby Abraham, MD, DM²^*, Chris Barnes³^*, Renée Brown Frandsen⁴^*, Kingsley Hampton⁵^*, Francisco José López Jaime, MD⁶^*, Camila Martins Mazini Tavares⁴^*, Keiji Nogami⁷^*, Jerzy Windyga, MD, PhD⁸ and Giancarlo Castaman⁹^*

¹Washington Center for Bleeding Disorders, Seattle, WA
²Department of Haematology, Christian Medical College Ranipet Campus, Ranipet, India
³The Royal Children’s Hospital, Melbourne, VIC, Australia
⁴Novo Nordisk A/S, Søborg, Denmark
⁵Department of Cardiovascular Science, University of Sheffield, Sheffield, ENG, GBR
⁶Hemostasis and Thrombosis Unit, Málaga Regional University Hospital, IBIMA, Málaga, Spain
⁷Department of Pediatrics, Nara Medical University, Nara, Japan
⁸Laboratory of Hemostasis and Metabolic Diseases, Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
⁹Center for Bleeding Disorders, Department of Oncology, Careggi University Hospital, Florence, Italy

2586

Assessment of Bleeding Severity and Prevalence of Iron Deficiency Among Hemophilia B Carriers By Factor IX Activity Levels

Zhexi Lu, MD¹^* and Nicoletta C Machin, DO^2,3

¹Department of Medicine, University of Pittsburgh Medical Center, Pittsburgh, PA
²Division of Classical Hematology, Department of Medicine, University of Pittsburgh, Pittsburgh, PA
³Hemophilia Center of Western Pennsylvania, Pittsburgh, PA

2587

Circulating and Synovial Fluid Levels of YKL-40 Protein in Patients with Hemophilia and Arthropathy: Preliminary Observations

Aikaterini Michalopoulou, MD¹^*, Anna Kouramba, MD²^*, Aimilia Mantzou, PhD³^*, Styliani-Despoina Christidi, MD⁴^*, Athina Dettoraki, MD, PhD¹^*, George Thivaios, MD⁴^*, Zoey Kapsimali, PhD¹^*, Olga Katsarou Fasouli, MD, PhD²^*, Ioannis Papassotiriou, PhD⁵ and Helen Pergantou, MD, PhD¹^*

¹Haemophilia Centre/Haemostasis and Thrombosis Unit - Aghia Sophia Children's Hospital, Athens, Greece
²Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Laiko General Hospital, Athens, GRC
³First Department of Pediatrics, National and Kapodistrian University of Athens, School of Medicine, Athens, Greece
⁴Trauma and Orthopaedics Department, Laiko General Hospital, Athens, Greece
⁵First Department of Pediatrics, National and Kapodistrian University of Athens, School of Medicine, Athens, Attica, Greece

2588

Emicizumab and Immune Tolerance Induction in People with Hemophilia A and Factor VIII Inhibitors: 96-Week Interim Analysis of the AKATSUKI Study

Azusa Nagao¹^*, Keiji Nogami²^*, Nobuaki Suzuki³^*, Chiai Nagae⁴^*, Masahiko Ando⁵^*, Yachiyo Kuwatsuka, MD PhD⁵^*, Daisuke Nosaka⁶^*, Yui Kyogoku⁶^*, Tomomi Shimura⁶^* and Tadashi Matsushita, MD⁵

¹Department of Pediatrics, Ogikubo Hospital, Tokyo, Japan
²Nara Medical University, Nara, Japan
³Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan
⁴Department of Pediatrics, St Marianna University School of Medicine, Kanagawa, Japan
⁵Nagoya University Hospital, Nagoya, Japan
⁶Chugai Pharmaceutical Co., Ltd., Tokyo, Japan

2589

FVIII Exposure Analysis from the HAVEN 7 Study of Emicizumab in Infants with Severe Hemophilia A

Guy Young, MD¹, Katherine Eakle²^*, Elise Lim²^* and Michael Wang³^*

¹Children's Hospital Los Angeles, Los Angeles, CA
²Genentech, Inc., South San Francisco, CA
³University of Colorado Anschutz Medical Campus, Aurora, CO

2590

Mechanistic Modeling to Support Hemostatic Equivalency of Antithrombin Lowering in People with Hemophilia A or B

Randolph Leiser, PhD¹^*, Chanchala Kaddi²^*, Mengdi Tao, MS¹^*, Ane Salvador, PhD³^*, Ekta Seth Chhabra, PhD, MSc⁴^* and Susana Zaph, PhD¹^*

¹Translational Disease Modelling, Data and Data Science, Sanofi, Bridgewater, United States, Bridgewater
²Sanofi, Cambridge, MA
³Rare and Neurologic Diseases, Sanofi, Cambridge, United States, Cambridge
⁴Global Research Lead, Rare and Neurologic Diseases, Sanofi, Cambridge

2591

Patient and Caregiver Preferences for Hemophilia Prophylactic Treatments: A Discrete Choice Experiment

Hui Lu, PhD¹^*, Jenny Whitty, PhD¹^*, Travis Gould, PhD², Carmine Colavecchia, PhD, PharmD²^*, Josh Coulter, MA²^*, Nicola Dunn, BCA, Psych Dip³^*, Pratima Chowdary, MD, MRCP, FRCPath⁴^*, Lisa J Wilcox, PhD⁵^*, Joseph C. Cappelleri, PhD⁶^*, Stephanie A Christopher, MA²^*, Alexis Sohn, PharmD, MPH, MS²^* and Brett Hauber, PhD²^*

¹Evidera, London, United Kingdom
²Pfizer Inc, New York, NY
³Independent Consultant, London, United Kingdom
⁴Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, United Kingdom
⁵Senior Director – Global Medical Affairs at Pfizer, Pfizer, Toronto, ON, Canada
⁶Pfizer Inc, Groton, CT

2592

Indirect Comparison of the Efficacy and Therapy-Related Costs of a Pharmacokinetic and Individualized Prophylaxis Regimen with Simoctocog Alfa Versus Other Extended-Half Life Factor VIII Concentrates

Craig Kessler, MD¹, Fernando F Corrales-Medina, MD², Pier Mannuccio Mannucci³^* and Michael D Tarantino, MD⁴

¹Lombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, Washington, DC
²Division of Pediatric Hematology-Oncology, University of Miami-Miller School of Medicine-Sylvester Cancer Center, Miami, FL
³Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi, Hemophilia and Thrombosis Center, IRCCS Ca' Granda Foundation, Milano, Italy
⁴Bleeding and Clotting Disorders Institute, Peoria, IL

2593

Real-World Data on Hemophilia a Patient Usage of Bay 81-8973 and Bay 94-9027 Stratified By Body Mass Index Categories in the Athndataset

Martin Chandler¹^*, Jessica Charlet, PhD, MSc, BSc²^* and Tammuella Chrisentery-Singleton, MD¹

¹American Thrombosis and Hemostasis Network, Rochester, NY
²Bayer US LLC Pharmaceuticals, Whippany, NJ

2593.1

Become–9: A Phase 1/2 Dose Escalation and Expansion Study of be-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B

Steven W. Pipe, MD¹, Mark T Reding, MD², Bryan Chin³^*, Karen Campbell³^*, Hanlan Liu, PhD³^* and Wing Yen Wong, MD³

¹University of Michigan, Ann Arbor, MI
²Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center, Minneapolis, MN
³Be Biopharma, Cambridge, MA

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^*signifies non-member of ASH