62nd ASH Annual Meeting and Exposition: 322. Disorders of Coagulation or Fibrinolysis: Hemophilia: Treatment and Inhibitors

Oral and Poster Abstracts
Oral

Adult, Hemophilia, Bleeding Disorders, Anemias, Biological, Animal models, Diseases, Bleeding and Clotting, Non-Biological, Hemostasis, Therapies, Genetic Disorders, Pediatric, Adverse Events, Biological Processes, coagulant drugs, Young Adult, Study Population, Clinically relevant, immune mechanism

Moderators:

Tyler W. Buckner, MD, MSc, University of Colorado School of Medicine and Glaivy Batsuli, MD, Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta and Emory University

Disclosures:

Buckner: Spark: Consultancy; Takeda: Consultancy; Kedrion: Consultancy; uniQure: Consultancy; Tremeau Pharmaceuticals: Consultancy; Biomarin: Consultancy; Bayer: Consultancy; CSL Behring: Consultancy; Novo Nordisk: Consultancy; American Thrombosis and Hemostasis Network: Membership on an entity's Board of Directors or advisory committees; Genentech: Consultancy. Batsuli: Octapharma: Other: Advisory board ; Bayer: Other: Advisory board ; Genentech: Other: Advisory board .

7:00 AM

507

Revisiting the “Danger Theory”: Toll-like Receptor 9 Stimulation Triggers Activation of Conventional CD8α⁺ and Plasmacytoid Dendritic Cells En Route to Enhancing FVIII Inhibitor Formation

Radoslaw Kaczmarek^*, Thais Bertolini^* and Roland W Herzog, PhD

Pediatrics, Indiana University School of Medicine, Indianapolis, IN

7:15 AM

508

Disentangling the Effects of HLA DRB1*15:01 and DQB1*06:02 to Establish the True HLA Risk Allele for Inhibitor Development in the Treatment of Hemophilia A

Vincent P. Diego, Ph.D.^1,2^*, Bernadette W. Luu, B.S., M.T. (CLS)^1,2,3^*, Alexis Garza, B.S.²^*, Marco Hofmann, M.S.⁴^*, Marcio A. Almeida, Ph.D.^1,2^*, Jerry S. Powell, M.D.^3,5, Long V. Dinh, Ph.D.³^*, Raja Rajalingam, Ph.D.⁶^*, Juan M Peralta, M.S.^1,2^*, Satish Kumar, Ph.D.^1,2^*, Eli Fine, Ph.D.³^*, Joanne E. Curran, Ph.D.^1,2^*, Miguel A. Escobar, M.D.⁷^*, Sarah Williams-Blangero, Ph.D.^1,2^*, Eugene Maraskovsky, Ph.D.⁸^*, John Blangero, Ph.D.^1,2^* and Tom E. Howard, M.D., Ph.D.^1,2,3,9^*

¹South Texas Diabetes and Obesity Institute, The University of Texas Rio Grande Valley, Brownsville, TX
²Department of Human Genetics, School of Medicine, The University of Texas Rio Grande Valley, Brownsville, TX
³Haplogenics, Brownsville, TX
⁴Research, CSL Behring GmbH, Marburg, Germany
⁵Division of Hematology and Oncology, Department of Internal Medicine, School of Medicine, The University of California at Davis, Davis, CA
⁶Immunogenetics and Transplantation Laboratory, Department of Surgery, School of Medicine, The University of California at San Francisco, San Francisco, CA
⁷University of Texas Health Science Center, Houston, TX
⁸Research, Bio21 Institute, CSL Limited, Melbourne, Australia
⁹Department of Pathology and Laboratory Medicine, VA Valley Coastal Bend Healthcare System, Harlingen, TX

7:30 AM

509

Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients with Haemophilia A

Christoph Koenigs¹^*, Margareth C. Ozelo², Amy Dunn³, Roshni Kulkarni, MD⁴, Beatrice Nolan, FRCPath⁵^*, Simon A. Brown⁶^*, Raina Liesner⁷^*, Michele Schiavulli⁸^*, Sriya Gunawardena⁹^*, Sutirtha Mukhopadhyay¹⁰^*, Deepthi Jayawardene⁹^*, Bent Winding¹¹^* and Manuel Carcao¹²

¹Department of Pediatrics and Adolescent Medicine, University Hospital Frankfurt, Frankfurt, Germany
²University of Campinas, São Paulo, Brazil
³Nationwide Children's Hospital, Columbus, OH
⁴Michigan State University, East Lansing, MI
⁵Children Health Ireland at Crumlin, Dublin, Ireland
⁶Queensland Children’s Hospital, South Brisbane, QLD, Australia
⁷Great Ormond Street Hospital, London, United Kingdom
⁸A.O.R.N. Santobono-Pausilipon, Naples, Italy
⁹Sanofi, Waltham, MA
¹⁰Sanofi, Gent, Belgium
¹¹Swedish Orphan Biovitrum AB (Sobi), Stockholm, Sweden
¹²Hospital for Sick Children, Toronto, Canada

7:45 AM

510

Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B

Tyler W. Buckner, MD, MSc¹, Shannon L Carpenter, MD², Stacy E. Croteau, MD³, Adam Cuker, MD, MS⁴, Nabil Daoud, MA⁵^*, Christine L Kempton, MD, MSc⁶, Lynn M Malec, MD⁷, Thomas W. McLean, MD⁸^*, Leslie J. Raffini, MD⁹, Janice M Staber, MD¹⁰, Michael Wang, MD¹¹, Crystal Watson, BS⁵^*, Ayesha Zia¹² and Michael Recht, MD, PhD^5,13

¹Divisions of Hematology and Pediatric Hematology/Oncology, Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO
²Children's Mercy Hospital, Kansas City, MO
³Children's Hospital Boston, Boston, MA
⁴Department of Medicine and Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA
⁵American Thrombosis and Hemostasis Network, Rochester, NY
⁶Emory University, Atlanta, GA
⁷Blood Research Institute, Versiti, Milwaukee, WI
⁸Wake Forest University School of Medicine, Winston Salem, NC
⁹Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
¹⁰University of Iowa Stead Family Children’s Hospital, Iowa City, IA
¹¹Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia and Thrombosis Center, Aurora, CO
¹²University of Southwestern Medical Center, Dallas, TX
¹³The Hemophilia Center, Oregon Health and Sci. Univ., Portland, OR

8:00 AM

511

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors – Results from the Phase II Study

Steven W. Pipe, MD¹, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH², Toshko Lissitchkov³^*, Margaret V. Ragni, MD, MPH⁴, Claude Négrier⁵, Qifeng Yu⁶^*, Stacey Poloskey⁷^*, Baisong Mei⁸ and Shauna R. Andersson⁸^*

¹Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI
²Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom
³Department of Chemotherapy, Hemotherapy and Hereditary Blood Diseases at Clinical Hematology Clinic, Specialized Hospital for Active Treatment of Hematological Diseases, Sofia, Bulgaria
⁴Department of Medicine - Division of Hematology/Oncology, University of Pittsburgh, Pittsburgh, PA
⁵Hôpital Cardiologique Louis Pradel, Lyon, France
⁶Albireo Pharma, Inc., Boston, MA
⁷Sanofi, Waltham, MA
⁸Sanofi, Cambridge, MA

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^*signifies non-member of ASH

322.O2.6 322. Disorders of Coagulation or Fibrinolysis: Hemophilia: Treatment and Inhibitors