64th ASH Annual Meeting and Exposition: 508. Bone Marrow Failure: Acquired: Biology

Symposia: Bone Marrow Failure: Acquired Oral and Poster Abstracts
Oral

Fundamental Science, Research, Acquired Marrow Failure Syndromes, Biological therapies, Antibody Therapy, autoimmune disorders, adult, Translational Research, Bone Marrow Failure Syndromes, Aplastic Anemia, GVHD, drug development, Diseases, Immune Disorders, Therapies, immunology, Biological Processes, Study Population, Human

260-262 (Ernest N. Morial Convention Center)

Moderators:

David J. Young, MD, PhD, Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institute of Health and Alicia Rovo, MD, Inselspital, University Hospital Bern, University of Bern

Disclosures:

Rovo: OrPhaSwiss GmbH: Membership on an entity's Board of Directors or advisory committees; Swedish Orphan Biovitrum AG: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Research Funding; CSL Behring: Research Funding; Alexion: Consultancy, Research Funding; BMS: Consultancy, Honoraria; AstraZeneca: Membership on an entity's Board of Directors or advisory committees.

This session contains oral presentation on the biology of acquired bone marrow failure, including diagnosis, genomics, and murine models for therapy.

2:00 PM

193

JAK 1/2 Inhibition Preserves Hematopoietic Progenitor and Stem Cells, Prevents Aplasia, Inhibits Pro-Inflammatory Cytokines, and Prolongs Survival in Murine Immune Bone Marrow Failure

Emma M. Groarke, MD¹, Xingmin Feng, PhD¹, Nidhi Aggarwal, BS², Ash Lee Manley³^*, Zhijie Wu, MD¹^*, Shouguo Gao, PhD¹^*, Bhavisha A. Patel, MD¹, Jichun Chen, PhD¹ and Neal S. Young, MD¹

¹Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Medical College of Georgia, Statesboro, GA
³Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Silver Spring, MD

2:15 PM

194

Blockade of Common Gamma Chain Cytokine Signaling with REGN7257, an Interleukin 2 Receptor Gamma (IL2RG) Monoclonal Antibody, Protected Mice from Inflammatory and Autoimmune Diseases

Audrey Le Floch-Ramondou, PhD¹^*, Kirsten Nagashima²^*, Pamela Farrales³^*, Amanda Tay³^*, Olivier Herbin³^*, Dharani Ajithdoss³^*, Thomas Norton²^*, Lorah Perlee, PhD⁴^*, Andrew J. Murphy, PhD¹^*, Matthew Sleeman²^* and Jamie Orengo²^*

¹Regeneron, Tarrytown, NY
²Regeneron, Tarrytown
³regeneron, tarrytown
⁴Regeneron Pharmaceuticals, Inc., Tarrytown, NY

2:30 PM

195

Single Cell Analysis Highlights Th17-Polarized CD4⁺ Naive T Cells with JAK3/STAT3 Pathway Activation in Pediatric Severe Aplastic Anemia

Jingliao Zhang, MD¹, Tianfeng Liu²^*, Yanxia Chang³^*, Yongjuan Duan⁴^*, Chao Liu, MD⁵^*, Xiaoyan Chen⁶^*, Cheng Tao, MD, PhD⁷^*, Xiaofan Zhu, PhD⁸ and Yingchi Zhang, PhD⁷^*

¹Chinese Academy of Medical Science & Peking Union Medical College, Tianjin, CHN
²State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical Colle, Tianjin, China
³Novogene Co, Ltd, Beijing, China
⁴State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
⁵Division of Pediatric Blood Diseases Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China
⁶Center for Pediatric Blood Disease, State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Tianjin, China
⁷State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
⁸Department of Pediatric, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical Colleg, Tianjin, China

2:45 PM

196

High-Dimensional Mass Cytometric Analysis of the Bone Marrow Compartment Identifies a Disease-Specific Immune Cell Network in Aplastic Anemia

Emma S. Pool¹^*, Yvonne Kooy-Winkelaar, BSc²^*, Vincent van Unen, PhD^2,3^*, J.H.Frederik Falkenburg, MD, PhD¹, Frits Koning, PhD²^*, Mirjam H.M. Heemskerk¹ and Jennifer M.L. Tjon, MD, PhD¹^*

¹Department of Hematology, Leiden University Medical Center, Leiden, Netherlands
²Department of Immunology, Leiden University Medical Center, Leiden, Netherlands
³Institute for Immunity, Transplantation and Infection, Stanford University, Stanford

3:00 PM

197

The Role of Heterozygous Variants in SAMD9/9L and Recessive Bone Marrow Failure-Related Genes in Adults with Severe Aplastic Anemia

Dalton Hironaka, BA¹^*, Xiaoyang Ma, MS²^*, Bhavisha A. Patel, MD¹, Emma M. Groarke, MD¹, Jennifer Lotter, PA-C¹^*, Olga Rios, RN¹^*, Juan L Coelho-Silva, PhD¹^*, Colin O. Wu, PhD²^*, Jorg Cammenga, MD³^*, Fernanda Gutierrez-Rodrigues, PhD⁴ and Neal S. Young, MD¹

¹Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
³Lund University Hospital, Lund, SWE
⁴Hematology Branch, National Heart, Lung, and Blood Institute, National Institute of Health, Bethesda, MD

3:15 PM

198

The Transcription Factor ETS-1 Correlates with the Increased Age Associated B Cells in Patients with Acquired Aplastic Anemia

Elena E. Solomou, MD¹, Antonis Kattamis, MD², Argiris Symeonidis, MD, PhD³, Haroula Sirinian, PhD⁴^*, Christina Salamaliki, MD⁴^*, Panagiotis T Diamantopoulos, MD, PhD⁵^*, Athanasios Galanopoulos, MD, PhD⁶^*, Maria D. Palasopoulou, MD⁷^*, Christina-Nefeli Kontandreopoulou⁸^*, Nikolaos Giannakoulas, MD⁹^*, Nora-Athina Viniou, MD, PhD¹⁰, Stamatis-Nick Liossis, MD¹¹^* and George Vassilopoulos, MD, PhD¹²

¹Department of Internal Medicine, University of Patras Medical School, Rion, Greece
²First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
³Hematology Division, Dept. of Internal Medicine, University of Patras Medical School, Patras, Greece
⁴University of Patras Medical School, Patras, Greece
⁵First Department of Internal Medicine, National and Kapodistrian University of Athens, Athens, Greece
⁶Department of Hematology, Gennimats General Hospital, Athens, Greece
⁷University of Hospital of Thessaly, Larissa, GRC
⁸Laikon General Hospital, First Department of Internal Medicine, National and Kapodistrian University of Athens, Athens, Greece
⁹Department of Hematology, University of Thessaly, University Hospital of Larissa, Larissa, Greece
¹⁰University of Athens Medical School, Athens, Greece
¹¹University of Patras medical School, Patras, Greece
¹²Department of Hematology, University of Thessaly Medical School, Larissa, Greece

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^*signifies non-member of ASH

508.O1.6 508. Bone Marrow Failure: Acquired: Biology