62nd ASH Annual Meeting and Exposition: 311. Disorders of Platelet Number or Function: Poster I

Oral and Poster Abstracts
Poster

Adult, anticoagulant drugs, aplasia, autoimmune disorders, Biological, Bleeding Disorders, Follicular Lymphoma, HSCs, Leukemia, multiple myeloma, sickle cell disease, aplastic anemia, Bleeding and Clotting, cell division, Diseases, Hemostasis, iPSCs, Non-Biological, smoldering myeloma, biopsy, Combinations, Diamond Blackfan Anemia, GVHD, HIT, Hodgkin Lymphoma, platelets, Therapies, Adverse Events, Genetic Disorders, ITP, Non-Hodgkin Lymphoma, Pediatric, red blood cells, Biological Processes, DLBCL, DNA damage, Pregnancy, DNA repair, Immune Disorders, Platelet Disorders, Technology and Procedures, Young Adult, Cell Lineage, epigenetics, Lymphocytopenia, infusion, Neutropenia, Study Population, Clinically relevant, Thrombocytopenias, hematopoiesis, imaging, Thrombotic Disorders, immune mechanism, TTP, inflammation, integrative -omics, VWD, iron transport, NGS, multi-systemic interactions, pathogenesis

835

Consistent Efficacy Demonstrated By Avatrombopag in Immune Thrombocytopenia (ITP) Regardless of the Number of Lines of Prior ITP Treatment

Morey Blinder, MD¹^*, Michael Vredenburg, PhD²^*, Wei Tian, PhD²^*, Brian Jamieson, MD, BA² and Keith R. McCrae, MD³

¹Washington University, Saint Louis, MO
²Dova Pharmaceuticals, Durham, NC
³Taussig Cancer Institute, Cleveland Clinic Lerner College of Medicine, Cleveland Clinic, Cleveland, OH

836

Chronic Thrombocytopenia in Children: What Could It Hide?

Sara Mohamed, MD¹^*, Martina Di Palma, MD¹^*, Michela Faleschini²^*, Daniela De Benedittis, MD¹^*, Maria Luisa Moleti, MD³^*, Luisa Cardarelli, MD³^*, Anna Maria Testi, MD^1,3^*, Giovanna Palumbo, MD³^*, Anna Savoia^2,4^* and Fiorina Giona, MD^1,3

¹Department of Translational and Precision Medicine - Hematology Division, Sapienza University of Rome, Rome, Italy
²Institute for Maternal and Child Health – IRCCS Burlo Garofolo, University of Trieste, Trieste, TS, Italy
³AOU Policlinico Umberto I, Rome, Italy
⁴Department of Medical Sciences, University of Trieste, Trieste, Italy

837

Comprehensive Description of Monoallelic GP1BA Variants Associated with Thrombocytopenia

Rachel C Peck, BSc, MRCP¹^*, Sarah Westbury, MBBChir, MRCP²^*, Lucy Fitzgibbon³^*, Neil V Morgan⁴^*, Jose Rivera, PhD⁵^*, Walter H. Kahr, MD, PhD, FRCPC⁶, Nihr BioResource⁷^*, Karyn Megy⁸^*, Kate Downes, PhD⁹^*, Daniel Greene¹⁰^*, Ernest Turro, PhD⁹^* and Andrew David Mumford³^*

¹Haematology, University of Bristol, Bristol, ENG, United Kingdom
²School of Clinical Science and School of Cellular and Molecular Medicine, University of Bristol, Bristol, GBR
³University of Bristol, Bristol, United Kingdom
⁴Institute of Cardiovascular Sciences, University of Birmingham, Birmingham, United Kingdom
⁵Hospital Universitario Morales Meseguer, Centro Regional de Hemodonación, Universidad de Murcia, IMIB-Arrixaca, CB15/00055-CIBERER, Murcia, Spain
⁶The Hospital for Sick Children, Toronto, ON, Canada
⁷NIHR BioResource–Rare Diseases Consortium, Cambridge, United Kingdom
⁸National Institute for Health Research (NIHR) BioResource, Cambridge, United Kingdom
⁹Department of Haematology, University of Cambridge, Cambridge, United Kingdom
¹⁰University of Cambridge, Cambridge, GBR

838

Long-Term Safety Profile of the Oral Spleen Tyrosine Kinase Inhibitor Fostamatinib in Immune Thrombocytopenia (ITP) and Other Diseases

Sandra Tong, MD^*, Robert P. Numerof, PhD^*, Jane Datangel, RN^* and Esteban Masuda, PhD

Rigel Pharmaceuticals, Inc., South San Francisco, CA

839

Megakaryocyte-Derived TGF-β1 Negatively Regulates Megakaryopoiesis in Mice

Gostynska Sandra, MS^*, Amanda Robertson^* and Jasimuddin Ahamed, PhD

Cardiovascular Biology Research Program, Oklahoma Medical Research Foundation (OMRF), Oklahoma City, OK

840

Risk Factors of Neonatal Immune Thrombocytopenia in Pregnant Women Previously Diagnosed with ITP: Results from a French Nationwide Prospective Study

Stephanie Guillet¹^*, Valentine Loustau²^*, Anissa Zarour³^*, Emmanuelle Boutin, MD⁴^*, Thibault Comont⁵^*, Odile Souchaud-Debouverie⁶^*, Nathalie Costedoat-Chalumeau, MD, PhD⁷^*, Brigitte pan-Petesch, MD⁸^*, Delphine Gobert, M.D⁹^*, Stephane Cheze, MD¹⁰^*, Jean-Francois Viallard¹¹^*, Anne-Sophie Morin¹²^*, Gaetan Sauvetre¹³^*, Manuel Cliquennois, MD, MSc¹⁴^*, Bruno Royer, MD¹⁵^*, Corinne Haioun, MD PhD¹⁶, Agathe Masseau¹⁷^*, Louis Terriou¹⁸^*, Claire Fieschi, MD, PhD¹⁹^*, Matthieu Mahevas²⁰^*, Marc Michel²¹^* and Bertrand Godeau²¹^*

¹Department of Internal Medecine, Henri Mondor University Hospital, Creteil, FRA
²Department of Internal Medecine, Henri Mondor University Hospital, Creteil, France
³Henri Mondor University Hospital, Creteil, France
⁴Department of Public Health and Biostatistics, Henri Mondor Hospital, Créteil, France
⁵Service de médecine interne, IUCT Oncopole, Centre Hospitalier Universitaire de Toulouse, Toulouse, France
⁶Department of Internal Medecine, Miletrie University Hospital, Poitiers, France
⁷Department of Internal Medecine, Cochin University Hospital, Paris, FRA
⁸Haemophilia Treatment Centre, CHRU Brest, Brest, France
⁹Internal Medicine, Saint Antoine Hospital, Paris, France
¹⁰Service d'Hématologie Clinique, CHU Côte de Nacre, Caen, France
¹¹Haut-Leveque Hospital, Pessac, FRA
¹²Department of Internal Medicine, Bondy Hospital, Bondy, FRA
¹³Department of Internal Medecine, Charles-Nicolle University Hospital, Rouen, France
¹⁴Hôpital Saint Vincent De Paul, Lille Cedex, FRA
¹⁵Department of Immuno hematology, Hôpital Saint-Louis, Assistance Publique – Hôpitaux de Paris, Paris, France
¹⁶Clinical Hematology, Henri Mondor University Hospital, UPEC, Creteil, France
¹⁷Department of Internal Medecine, Hotel Dieu Hospital, Nantes, France
¹⁸Hôpital Claude Huriez, Lille, France
¹⁹Service d’Immunologie clinique, Hôpital Saint-Louis, AP-HP, Paris, France
²⁰Service de Médecine Interne, Centre national de référence des cytopénies auto-immunes de l’adulte, Hôpital Henri Mondor, 51 avenue du Maréchal de Lattre de Tassigny, Assistance Publique Hôpitaux de Paris, Université Paris Est Créteil, 94000 Créteil, Creteil, France
²¹Service de Médecine Interne, Centre national de référence des cytopénies auto-immunes de l’adulte, Hôpital Henri Mondor, 51 avenue du Maréchal de Lattre de Tassigny, Assistance Publique Hôpitaux de Paris, Université Paris Est Créteil, 94000 Créteil, Créteil, France

841

A Phase II Study to Assess the Sustained Response Off Treatment in Patients with ITP Receiving Eltrombopag, Who Had a Previous Insufficient Response to Corticosteroids (TAPER): A Recruitment Update

Nichola Cooper, MD¹, Waleed Ghanima², Jens Haenig³^*, James Lee⁴^*, Masiur Rahman⁵^* and Francesco Zaja⁶^*

¹Hammersmith Hospital, Imperial College London, London, United Kingdom
²Department of Medicine, Østfold Hospital Trust, Kalnes, Norway
³Novartis Pharma AG, Basel, Switzerland
⁴Novartis Pharmaceuticals Corporation, East Hanover, NJ
⁵IQVIA, Kirkland, QC, Canada
⁶Department of Medical, Surgical and Health Sciences, University of Trieste, Trieste, Italy

842

Genome-Wide Association Study Identifies Variation in ABO As Risk Factor for Platelet Reactivity in Heparin-Induced Thrombocytopenia

Jason B Giles¹^*, Jérôme Rollin, PhD²^*, Christian M Shaffer³^*, Heidi E Steiner¹^*, Yukihide Momozawa, DVM, Ph.D⁴^*, Ian Stanaway, PhD⁵^*, Taisei Mushiroda⁶^*, Claire Pouplard, PharmD, PhD²^*, Nancy Heddle, MSc⁷, Michiaki Kubo, MD, Ph.D⁸^*, Elizabeth Phillips, MD³^*, Theodore E. Warkentin, MD⁹, Yves Gruel, MD, PhD¹⁰, Andreas Greinacher, MD¹¹, Dan Roden, MD³^* and Jason Karnes, PhD, PharmD¹

¹University of Arizona, Tucson, AZ
²Department of Hematology-Hemostasis, University Hospital of Tours, Tours, France
³Vanderbilt University Medical Center, Nashville, TN
⁴Laboratory for Genotyping Development, RIKEN Center for Integrative Medical Sciences, Yokohama, Japan
⁵University of Washington, Seattle, WA
⁶RIKEN Center for Integrative Medical Sciences (IMS), Yokohama, Japan
⁷Department of Medicine, McMaster University, Hamilton, ON, Canada
⁸Center for Integrative Medical Sciences, RIKEN, Yokohama, Japan
⁹McMaster University, Hamilton, ON, Canada
¹⁰Department of Hematology-Hemostasis, University Hospital of Tours, Chambray-Lès-Tours, France
¹¹Institute for Immunology & Transfusion Ernst-Moritz-Arndt-University, Greifswald, Germany

843

Outcomes of Patients with Worsening Acquired Thrombotic Thrombocytopenic Purpura Despite Daily Therapeutic Plasma Exchange in the Phase 3 Hercules Trial

Marie Scully, MD¹, Javier de la Rubia^2,3^*, Flora Peyvandi^4,5, Spero Cataland⁶, Paul Coppo⁷^*, Johanna A Kremer Hovinga Strebel⁸^*, Paul Knoebl⁹^*, Katerina Pavenski, MD¹⁰^*, Filip Callewaert¹¹^*, Jessica Minkue Mi Edou¹²^* and Rui de Passos Sousa¹³^*

¹Department of Haematology, University College London Hospitals, Cardiometabolic Program, National Institute for Health Research UCLH/UCL Biomedical Research Centre, London, United Kingdom
²Hematology Department, Internal Medicine, School of Medicine and Dentistry, Catholic University of Valencia, Valencia, Spain
³Hospital Doctor Peset, Valencia, Spain
⁴Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
⁵Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
⁶Division of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, OH
⁷Department of Hematology, Reference Center for Thrombotic Microangiopathies (CNR-MAT), Saint-Antoine University Hospital, AP-HP, Paris, France
⁸Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland
⁹Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Wien, Austria
¹⁰Departments of Medicine and Laboratory Medicine, St. Michael’s Hospital, University of Toronto, Toronto, ON, Canada
¹¹Sanofi, Diegem, Belgium
¹²Sanofi, Zwijnaarde, Belgium
¹³Sanofi, Lisbon, Portugal

844

Characterization of Thromboembolic Events Occurring during the Avatrombopag Immune Thrombocytopenia (ITP) Clinical Development Program

Caroline I. Piatek, MD¹^*, Brian Jamieson, MD, BA² and Michael Vredenburg, PhD²^*

¹Jane Anne Nohl Division of Hematology, Keck School of Medicine of University of Southern California, Los Angeles, CA
²Dova Pharmaceuticals, Durham, NC

845

Patients’ Reported Perceptions on Satisfaction with Immune Thrombocytopenia Treatments: Results from the ITP World Impact Survey (I-WISh)

Waleed Ghanima¹, James B. Bussel², Drew Provan³^*, Alexandra Kruse⁴^*, Caroline Kruse⁴^*, Yoshiaki Tomiyama⁵, Ming Hou⁶, Cristina Santoro⁷^*, Serge Laborde⁸^*, Mervyn Morgan⁹^*, Barbara Lovrencic¹⁰^*, Tom Bailey, MSci¹¹^*, Jens Haenig¹²^* and Nichola Cooper, MD¹³

¹Department of Medicine, Østfold Hospital Trust, Kalnes, Norway
²Division of Hematology/Oncology, Weill Cornell Medical College, New York, NY
³Barts and The London School of Medicine and Dentistry, London, United Kingdom
⁴Platelet Disorder Support Association, Cleveland, OH
⁵Osaka University Hospital, Osaka, Japan
⁶Department of Hematology, Qilu Hospital of Shandong University, Jinan, China
⁷Hematology, University Hospital Policlinico Umberto I, Rome, Italy
⁸O’Cyto, Saint Loubès, France
⁹ITP Support Association, Bolnhurst, United Kingdom
¹⁰Italian Association for Immune Thrombocytopenic Purpura, Caprino Veronese, Italy
¹¹Adelphi Real World, Bollington, United Kingdom
¹²Novartis Pharma AG, Basel, Switzerland
¹³Hammersmith Hospital, Imperial College London, London, United Kingdom

846

The ADAMTS13 Family: In-Patient Trends and Outcomes of Thrombotic Thrombocytopenic Purpura

Siddharth Bhesania¹^*, Deepali Sharath²^*, Stuti Pandya³^*, Dennis Kum⁴^*, Astha Upadhyaya, MD⁵^*, Neha Bapat⁶^*, Pritika Manaktala, MD⁷^*, Michaela Murphy, MD¹^*, Janki Bhesania, MD⁸^*, Achint Patel, MD, MPH⁹^*, Nivetha Vishnuvardhan, MD¹, Parag Mehta, MD¹^* and Eshan Patel, MD¹

¹NewYork-Presbyterian Brooklyn Methodist Hospital, Brooklyn, NY
²Vydehi Institute of Medical Sciences and Research Center, Bangalore, India
³Ahmedabad Municipal Corporation Medical Education Trust Medical, Ahmedabad, India
⁴Charles University and Motol University Hospital, Praha, Czech Republic
⁵Staten Island University Hospital, New York, NY
⁶Mount Sinai Hospital, New York, NY
⁷Canton Medical Education Foundation, Canton, OH
⁸Independent Researcher, Brooklyn
⁹Independent Researcher, Sayre, PA

847

Treatment Patterns and Bleeding Events Among Patients with Immune Thrombocytopenia Based on Duration of Corticosteroid Treatment

Mark D. Hatfield, PhD¹^*, Janna Manjelievskaia, PhD, MPH²^*, Kristin A. Evans, PhD²^*, Philip K. Chan, PhD, MS²^*, Neel Shah, BPharm, PhD¹^* and Hossam A Saad, MD¹

¹Amgen Inc., Thousand Oaks, CA
²IBM Watson Health, Cambridge, MA

848

Pharmacokinetic/Phamacodynamic (PK/PD) Modeling Providing Guidance for Selecting Avatrombopag (AVA) Dose When Switching from Eltrombopag in Chronic Immune Thrombocytopenia (ITP)

Nashat Y. Gabrail, MD¹^*, Kavita Aggarwal, PharmD²^*, Michael Vredenburg, PhD²^*, Ruemu Birhiray, MD³ and Brian Jamieson, MD, BA²

¹Gabrail Cancer Center, Canton, OH
²Dova Pharmaceuticals, Durham, NC
³Hematology Oncology of Indiana, PC/American Oncology Network, PA, Indianapolis, IN

849

Long-Term Overuse of Corticosteroids in Patients with Immune Thrombocytopenia: A Real-World Analysis of a US Claims Database

James B. Bussel¹, Joseph Tkacz²^*, Janna Manjelievskaia, PhD, MPH²^*, Jens Haenig³^*, Joan Maier³^* and Adam Cuker, MD, MS⁴

¹Division of Hematology/Oncology, Weill Cornell Medical College, New York, NY
²IBM Watson Health, Cambridge, MA
³Novartis Pharma AG, Basel, Switzerland
⁴Department of Medicine and Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA

850

Use of a Von Willebrand Factor/Coagulation Factor VIII Complex for Treatment of Refractory Inherited Platelet Disorders

Amber Federizo

Hemostasis and Thrombosis Center of Nevada, Las Vegas, NV

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^*signifies non-member of ASH

311.P1.60 311. Disorders of Platelet Number or Function: Poster I