65th ASH Annual Meeting & Exposition: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Innovative Treatments for Immune Thrombocytopenia

Symposia: Disorders of Platelet Number or Function: Clinical and Epidemiological Oral and Poster Abstracts
Oral

autoimmune disorders, Biological therapies, platelet disorders, Chimeric Antigen Receptor (CAR)-T Cell Therapies, drug development, Diseases, thrombocytopenias, Immune Disorders, Therapies, Monoclonal Antibody Therapy

Hall A (San Diego Convention Center)

Moderators:

Amanda B. Grimes, MD, Texas Children's Hospital and Annabel Kate Frank, MD, University of California, San Francisco

Disclosures:

No relevant conflicts of interest to declare.

10:30 AM

685

Initial Report of Part B Phase 1/2 Efficacy and Safety Results for Bruton Tyrosine Kinase Inhibitor Rilzabrutinib in Patients with Relapsed Immune Thrombocytopenia

Nichola Cooper, MD¹^*, A.J. Gerard Jansen, MD², Robert Bird, MBBS³^*, Jiří Mayer, MD⁴, Michelle Sholzberg, MDCM, MSc⁵, Michael D. Tarantino, MD⁶, Mamta Garg⁷^*, Paula F Ypma, MD, PhD⁸^*, Vickie McDonald, MD, PhD, FRPATH, MRCP⁹^*, Charles Percy, MD¹⁰^*, Milan Košťál¹¹^*, Isaac Goncalves, MD¹²^*, Lachezar H. Bogdanov¹³^*, Terry B Gernsheimer, MD¹⁴, Remco Diab¹⁵^*, Mengjie Yao¹⁶^*, Ahmed Abd Almalik Daak, MD, PhD, MSc, DPM/MFPM¹⁵ and David J Kuter, MD, DPhil¹⁷

¹Hammersmith Hospital, London, United Kingdom
²Erasmus MC, University Medical Center, Rotterdam, Netherlands
³Princess Alexandra Hospital, Woolloongabba, Australia
⁴Masaryk University Hospital, Brno, CZE
⁵Li Ka Shing Knowledge Institute University of Toronto, St. Michael’s Hospital, Toronto, ON, Canada
⁶The Bleeding and Clotting Disorders Institute, University of Illinois College of Medicine-Peoria, Peoria, IL
⁷Leicester Royal Infirmary, Leicester, United Kingdom
⁸Department of Hematology, HagaZiekenhuis, Den Haag, Netherlands
⁹Barts Health NHS Trust, The Royal London Hospital, London, United Kingdom
¹⁰NHS Foundation Trust, University Hospitals Birmingham, Birmingham, United Kingdom
¹¹Fourth Department of Internal Medicine and Hematology, Faculty of Medicine, University Hospital of Hradec Králové, Hradec Králové, Czech Republic
¹²Royal Melbourne Hospital and Peter MacCallum Cancer Centre, Parkville, Australia
¹³Clinic of Hematology, University Hospital, Pleven, Bulgaria
¹⁴University of Washington and Fred Hutchinson Cancer Center, Seattle, WA
¹⁵Sanofi, Cambridge, MA
¹⁶Sanofi, Bridgewater, NJ
¹⁷Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, MA

10:45 AM

686

Integrin αvβ8-Mediated Activation of TGF-β1 Is Involved in Regulating the Sustained Response after TPO-Ras Withdrawal in ITP Patients

Min Xu¹^*, Jie Zhou²^*, Jinhui Shu¹^*, Yu Hu, MD^1,3,4^* and Heng Mei¹^*

¹Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
²Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China, Wuhan, CHN
³Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
⁴Hubei Clinical and Research Centre of Thrombosis and Haemostasis, Wuhan, China

11:00 AM

688

The Therapeutic Efficacy of CD19 Chimeric Antigen Receptor T Cells in Immune Thrombocytopenia Model

Yun Wang¹^*, Fengjiao Han¹^*, Qiuyu Guo, MBBS²^*, Qi Feng³^*, Jun Peng³^*, Ming Hou³^* and Miao Xu, MD, PhD⁴^*

¹Department of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, China
²Department of Hematology, Qilu Hospital of Shandong University, Jinan, China
³Qilu Hospital of Shandong University, Jinan, China
⁴Department of Hematology, Qilu Hospital of Shandong University, Jinan, AL, China

11:15 AM

689

Time to Achieve Platelet Count Response after Intravenous Efgartigimod in Adults with Primary Immune Thrombocytopenia: A Phase 3, Multicenter, Double-Blinded, Placebo-Controlled, Randomized Clinical Trial (ADVANCE IV)

Catherine M. Broome, MD¹, Vickie McDonald, MD, PhD, FRPATH, MRCP²^*, Yoshitaka Miyakawa, MD³^*, Monica Carpenedo, MD^4,5^*, David J Kuter, MD, DPhil⁶, Hanny Al-Samkari, MD⁷, James B. Bussel, MD⁸, Anna Hultberg, PhD⁹^*, Filip Matthijssens, PhD¹⁰^*, Jaume Ayguasanosa, MD¹⁰^*, Kristof De Beuf, PhD¹⁰^*, Francesco Rodeghiero, MD¹¹, Marc Michel¹²^* and Adrian Newland, FRCP¹³

¹Georgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, DC
²Barts Health NHS Trust, The Royal London Hospital, London, United Kingdom
³Department of Hematology, Saitama Medical University Hospital, Saitama, Japan
⁴Hematology and Transplant Unit, IRCCS Ospedale San Gerardo di Monza, Monza, Italy
⁵Ospedale L. Sacco, ASST Fatebenefratelli-Sacco, Milan, Italy
⁶Hematology Division, Massachusetts General Hospital, Boston, MA
⁷Massachusetts General Hospital, Cambridge, MA
⁸Weill Cornell, New York, NY
⁹argenx, Zwijnaarde, Belgium
¹⁰argenx, Ghent, Belgium
¹¹Haematology Project Foundation, Affiliated to the Department of Haematology, S. Bortolo Hospital, Vicenza, Italy
¹²Department of Internal Medicine, National Reference Center for Immune Cytopenias, Henri-Mondor University Hospital, Assistance Publique–Hôpitaux de Paris, Université Paris-Est Créteil, Creteil, France
¹³Centre for Haematology, Barts and the London School of Medicine & Dentistry, Queen Mary University of London, London, United Kingdom

11:30 AM

690

The Effects of Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, on Bleeding Symptoms and Health-Related Quality of Life in Patients with Immune Thrombocytopenia

Michael D. Tarantino, MD¹, A.J. Gerard Jansen, MD², Nichola Cooper, MD^3,4^*, Imene Gouia, PharmD, MSc Health Economics⁵^*, Mengjie Yao⁶^*, Ahmed Abd Almalik Daak, MD, PhD, MSc, DPM/MFPM⁷ and David J Kuter, MD, DPhil⁸

¹The Bleeding and Clotting Disorders Institute, University of Illinois College of Medicine-Peoria, Peoria, IL
²Erasmus MC, University Medical Center, Rotterdam, Netherlands
³Centre for Haematology, Department of Immunology and Inflammation, Imperial College London, London, United Kingdom
⁴Hammersmith Hospital, London, United Kingdom
⁵Sanofi, Gentilly, France
⁶Sanofi, Bridgewater, NJ
⁷Sanofi, Cambridge, MA
⁸Massachusetts General Hospital, Harvard Medical School, Boston, MA

11:45 AM

687

[CANCELED] Efficacy and Safety of Human Umbilical Cord-Derived Mesenchymal Stem Cells in the Treatment of Immune Thrombocytopenia: A Prospective, Single Centre, Single Arm, Phase I Trial

Yunfei Chen^1,2^*, Xu Yanmei^1,2^*, Yuchen Gao^1,2^*, Ying Chi^1,2^*, Ting Sun^1,2^*, Xueqing Dou³^*, Zhibo Han^4,5^*, Feng Xue, MD^1,2^*, Huiyuan Li^1,2^*, Wei Liu^1,2^*, Xiaofan Liu, MD^1,6^*, Huan Dong^1,2^*, Rongfeng Fu^1,2^*, Mankai Ju^1,2^*, Xinyue Dai^1,2^*, Wentian Wang^1,2^*, Yueshen Ma^1,2^*, Zhen Song^1,2^*, Jundong Gu, MD, PhD⁵^*, Wei Gong⁵^*, Renchi Yang, MD^1,6 and Lei Zhang, MD^1,7

¹Tianjin Institutes of Health Science, Tianjin, China
²Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
³National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China
⁴State Key Laboratory of Experimental Hematology, National Clinical Research Centre for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences&Peking Union Medical College, Tianjin, China
⁵National Engineering Research Centre of Cell Products, Tianjin Key Laboratory of Engineering Technologies for Cell Pharmaceutical, AmCellGene Engineering Co., Ltd, Tianjin, China
⁶Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
⁷State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, China

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^*signifies non-member of ASH

311.O1.6 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Innovative Treatments for Immune Thrombocytopenia