66th ASH Annual Meeting: 508. Bone Marrow Failure: Acquired: Biological Findings Associated with the Pathophysiology and Outcomes of Aplastic Anemia and PNH

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

Research, Acquired Marrow Failure Syndromes, Clinical trials, MDS, Combination therapy, Fundamental Science, Bleeding and Clotting, Artificial intelligence (AI), Translational Research, Bone Marrow Failure Syndromes, CHIP, Assays, Aplastic Anemia, Genomics, Clinical Research, Genetic Disorders, Bioinformatics, Paroxysmal Nocturnal Hemoglobinuria, Hematopoiesis, Chronic Myeloid Malignancies, Thromboembolism, Diseases, Immune mechanism, Real-world evidence, Treatment Considerations, Immunology, Computational biology, Biological Processes, Myeloid Malignancies, Technology and Procedures, Machine learning, Pathology, Serologic Tests

Room 11 (San Diego Convention Center)

Moderators:

Antonio M Risitano, MD, PhD, ARRAY(0xdccc854) and Bhavisha A. Patel, MD, National Institutes of Health

Disclosures:

No relevant conflicts of interest to declare.

This session will cover the most recent biological observation derived by the use of high-throughput technologies in patients suffering from acquired bone marrow failure syndromes. In particular, investigator will discuss data concerning TCR clonality, and its possibly relationship with causative antigen(s), the emergence of clonal hematopoiesis eventually tracked by somatic mutations, as well as genetic background which may shape the clinical presentation of a given disease

9:30 AM

Genetic Determinants of Hypercoagulability in Complement and Hemostatic Systems Shape the Thrombotic Risk of Paroxysmal Nocturnal Hemoglobinuria

Carlos Bravo-Perez, MD^1,2^*, Carmelo Gurnari, MD, PhD^1,3, Arda Durmaz, PhD¹^*, Stephan Hutter, PhD⁴^*, Zachary Brady¹^*, Hussein Awada¹, Aashray Mandala, BS¹^*, Luca Guarnera, MD^1,3^*, Matteo D'Addona¹^*, Kartik Lakhotiya, MS¹^*, Praveena Thiagarajan, MD, PhD¹^*, Alan Eli Lichtin, MD¹, Torsten Haferlach, MD⁴, Valeria Visconte, PhD¹ and Jaroslaw Maciejewski¹

¹Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
²Department of Hematology and Medical Oncology, Hospital Universitario Morales Meseguer, University of Murcia, IMIB-Pascual Parrilla, CIBERER - Instituto de Salud Carlos III, Murcia, Spain
³Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
⁴MLL Munich Leukemia Laboratory, Munich, Germany

9:45 AM

Longitudinal Contribution of Distinct Hematopoietic Clones during Marrow Recovery in Immune Aplastic Anemia

Fernanda Gutierrez-Rodrigues, PhD, Emma M. Groarke, MD, Lemlem Alemu, BS^*, Olga Rios^*, Diego Quinones Raffo, BS^*, Jennifer Lotter, ms, Neal S. Young, MD and Bhavisha A. Patel, MD

Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

10:00 AM

Dynamics of Oligoclonal Hematopoiesis in Severe Aplastic Anemia Patients Undergoing Immunosuppressive Treatment: Longitudinal Somatic Mutation Analysis from the EBMT Race Trial

Denise Kaya¹^*, Riley Cook²^*, Simona Iacobelli³^*, Austin G. Kulasekararaj, MD, MBBS, FRCPath, MRCP^4,5,6, Giorgio Napolitani, PhD⁷^*, Sila Gerlevik, MSc⁸^*, Nogayhan Seymen^9,10^*, Flore Sicre de Fontbrune, MD¹¹^*, Morag Griffin, MBChB, MRCP¹²^*, Camilla Frieri, MD, PhD¹³^*, Constantijn J.M. Halkes, MD¹⁴^*, Christian Recher, MD, PhD¹⁵^*, Fiorenza Barraco¹⁶^*, Edouard Forcade, MD, PhD¹⁷^*, Jean-Baptiste Méar, MD¹⁸^*, Marica Laurino, MD¹⁹^*, Beatrice Drexler, MD²⁰^*, Etienne Daguindau, MD²¹^*, Marleen Van Os²²^*, Sofie Terwel, MSc²³^*, Carlo Dufour, MD²⁴, Mohammad M. Karimi, PhD^25,26^*, Regis Peffault De Latour²⁷^*, Antonio M Risitano, MD, PhD^28,29,30 and Ghulam Mufti⁷^*

¹Comprehensive Cancer Centre, School of Cancer & Pharmaceutical Sciences, Faculty of Life Sciences & Medicine, King’s College London, London, United Kingdom, London, United Kingdom
²King's College London, London, GBR
³Centro Di Biostatistica E Bioinformatica Università Tor Vergata, Rome, ITA
⁴London, ENG, United Kingdom
⁵Department of Hemato-oncology, Kings College London, London, United Kingdom
⁶National Institute for Health and Care Research and Wellcome King’s Research Facility, London, United Kingdom
⁷Comprehensive Cancer Centre, School of Cancer and Pharmaceutical Sciences, King's College London, London, United Kingdom
⁸King's college London, London, United Kingdom
⁹Comprehensive Cancer Centre, School of Cancer and Pharmaceutical Sciences, Faculty of Life Sciences and Medicine, King’s College London, London, United Kingdom, London, United Kingdom
¹⁰Comprehensive Cancer Centre, King's College London, London, United Kingdom
¹¹Centre de Référence Aplasie Médullaire, Service d’Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France
¹²Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom
¹³AORN S. Giuseppe Moscati, Avellino, AL, ITA
¹⁴Hematology, Leiden University Medical Center, Leiden, Netherlands
¹⁵Service d'hématologie, Centre Hospitalo-universitaire de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Université de Toulouse, Toulouse, France
¹⁶Service d’Hematologie Clinique, Hôpital universitaire, Lyon, France
¹⁷CHU Bordeaux, Pessac, France
¹⁸Clinical hematology, University Hospital of Rennes, Rennes, France, Rennes, France
¹⁹UO Ematologia e terapie cellulari, IRCCS Ospedale Policlinico San martino, Genova, Italy
²⁰Department of Hematology, University Hospital Basel, Basel, Switzerland
²¹Department of Clinical Hematology, Hopital Jean Minjoz, Service d'Onco-hématologie, Besançon, France
²²EBMT Data Office, Leiden, NLD
²³Leiden University, Leiden, NV, NLD
²⁴G. Gaslini Chidlren's Hospital, Genova, Italy
²⁵Comprehensive Cancer Centre, School of Cancer and Pharmaceutical Sciences, King’s College London, London, United Kingdom
²⁶King’s College London, Comprehensive Cancer Centre, School of Cancer and Pharmaceutical Sciences, London, United Kingdom
²⁷Hôpital Saint-Louis,, Paris, France
²⁸University of Naples, Avellino, Italy
²⁹Severe Aplastic Anemia Working Party, European Group for Blood and Marrow Transplantation (EBMT), Leiden, Netherlands
³⁰The Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation, Barcelona, Spain

10:15 AM

T Cell Receptor Clonal Dynamics and Hematopoietic Recovery after Immunotherapy in Severe Aplastic Anemia

Zhijie Wu, MD, PhD¹^*, Shouguo Gao, PhD¹^*, Xingmin Feng, PhD¹^*, Haoran Li, BS¹, Nicolas Sompairac, PhD²^*, Shirin Jamshidi, PhD²^*, Desmond Choy, PhD²^*, Rita Antunes Dos Reis²^*, Qingyan Gao³^*, Sachiko Kajigaya, PhD¹^*, Lemlem Alemu, BS¹^*, Diego Quinones Raffo, BS¹^*, Emma M. Groarke, MD¹, Shahram Kordasti, MD, PhD^2,4, Bhavisha A. Patel, MD¹ and Neal S. Young, MD¹

¹Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Comprehensive Cancer Centre, School of Cancer and Pharmaceutical Sciences, Faculty of Life Sciences and Medicine, King's College London, London, United Kingdom
³Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
⁴Department of Clinical Haematology, Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom

10:30 AM

First Results of High Throughput Pipeline for Serotypic and TCR Clonotypic Specificities in Aplastic Anemia and Large Granular Lymphocyte Leukemia Uncovering Antigenic Peptides Relevant to Disease Etiology

Kartik Lakhotiya, MS^1,2^*, Carmelo Gurnari, MD, PhD^1,3, Carlos Bravo-Perez, MD^1,4^*, Aashray Mandala, BS¹^*, Matteo D'Addona^1,5^*, Christopher Haddad¹^*, Mark Orland, MD¹, Zachary Brady¹^*, Luca Guarnera, MD⁶, Praveena Thiagarajan, MD, PhD¹^*, Ishani Nautiyal, BS¹^*, Simona Pagliuca, MD, PhD⁷, Hari Krishnamurthy⁸^*, Karthik Krishna⁸^*, Vasanth Jayaraman⁸^*, Valeria Visconte, PhD⁹, Jaroslaw Maciejewski⁹ and Arda Durmaz, PhD¹^*

¹Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
²Department of Pathology, School of Medicine, Case Western Reserve University, Cleveland, OH
³Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
⁴Centro Regional de Hemodonación, IMIB-Arrixaca, CIBERER, University of Murcia, Murcia, Spain
⁵Hematology and Transplant Center, University Hospital “San Giovanni di Dio e Ruggi d’Aragona”, Ercolano, Italy
⁶Tor Vergata University, Rome, Italy
⁷Department of Hematology, Nancy University Hospital, Vandœuvre-lès-Nancy, France, France
⁸Vibrant Sciences, Santa Clara, CA
⁹Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH

10:45 AM

Vexas Syndrome Is a New Cause of p53-Mediated Erythroblastopenia

François Rodrigues, MD¹^*, Giulia Hardouin²^*, Sara El Hoss, PhD¹^*, Aya Ghoul¹^*, Emilie-Fleur Gautier³^*, Michael Dussiot⁴^*, Sandy Peltier⁵^*, Pascal Amireault⁶^*, Annarita Miccio, PhD^{7,8,9,10,11,12,13}, Vincent Jachiet¹⁴^*, Thiago Trovati Maciel, PhD^15,16,17^*, Sophie Georgin Lavialle¹⁸^*, Julien Rossignol¹⁹^*, Arsène Mekhinian, MD, PhD¹⁸^*, Olivier Kosmider, PharmD, PhD²⁰^*, Manuel Rodriguez²¹^* and Olivier Hermine, MD, PhD^17,22,23

¹Institut Imagine, Paris, France
²Institut Imagine, Paris, FRA
³INSERM U1016, PARIS, FRA
⁴Imagine Institute, PARIS, FRA
⁵INSERM, Paris, France
⁶Instittut Imagine, Paris, FRA
⁷IMAGINE Institute, Université Paris Descartes, Sorbonne Paris Cité, Paris, France
⁸IMAGINE Institute, Universit, Paris, France
⁹Imagine Institute, INSERM UMR1163, Paris, Paris, France
¹⁰Imagine Institute, Paris, France
¹¹Université de Paris, Paris, France
¹²Genethon, INSERM UMR951, Evry, France
¹³Laboratory of chromatin and gene regulation during development, Inserm, Imagine Institute, Paris, France
¹⁴Saint Antoine Hospital AP-HP, Paris, France
¹⁵Institut IMAGINE - INSERM U1163, Paris, FRA
¹⁶Université Paris Cité, Paris, France
¹⁷Institut Imagine - INSERM U1163, Paris, France
¹⁸APHP, Paris, France
¹⁹Hématologie, NECKER ENFANTS MALADES Hospital APHP, Paris, FRA
²⁰Laboratory of Hematology, Université Paris Cité and Assistance Publique-Hôpitaux de Paris. Centre, Hôpital Cochin, Paris, France
²¹Laboratoire De Chimie De Coordination LCC-UPR 8241-CNRS, Toulouse, FRA
²²CEREMAST Necker Hospital, Paris, France
²³Hematology, Necker Hospital, Greater Paris University Hospitals (AP-HP), Paris, France

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508.O2.6 508. Bone Marrow Failure: Acquired: Biological Findings Associated with the Pathophysiology and Outcomes of Aplastic Anemia and PNH