-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

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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

Symposia: Bone Marrow Failure: Acquired Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
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
Saturday, December 7, 2024: 9:30 AM-11:00 AM
Room 11 (San Diego Convention Center)
Moderators:
Antonio M Risitano, MD, PhD, ARRAY(0xe591da8) 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

Carlos Bravo-Perez, MD1,2*, Carmelo Gurnari, MD, PhD1,3, Arda Durmaz, PhD1*, Stephan Hutter, PhD4*, Zachary Brady1*, Hussein Awada1, Aashray Mandala, BS1*, Luca Guarnera, MD1,3*, 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

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

Denise Kaya1*, Riley Cook2*, Simona Iacobelli3*, Austin G. Kulasekararaj, MD, MBBS, FRCPath, MRCP4,5,6, Giorgio Napolitani, PhD7*, Sila Gerlevik, MSc8*, Nogayhan Seymen9,10*, 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, PhD25,26*, Regis Peffault De Latour27*, Antonio M Risitano, MD, PhD28,29,30 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
4King’s College Hospital-NHS Foundation Trust, NIHR/Wellcome King’s Clinical Research Facility, and King’s College London, London, 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

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, PhD2,4, 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

Kartik Lakhotiya, MS1,2*, Carmelo Gurnari, MD, PhD1,3, Carlos Bravo-Perez, MD1,4*, Aashray Mandala, BS1*, Matteo D'Addona1,5*, 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

François Rodrigues, MD1*, Giulia Hardouin2*, Sara El Hoss, PhD1*, Aya Ghoul1*, Emilie-Fleur Gautier3*, Michael Dussiot4*, Sandy Peltier5*, Pascal Amireault6*, Annarita Miccio, PhD7,8,9,10,11,12,13, Vincent Jachiet14*, Thiago Trovati Maciel, PhD15,16,17*, Sophie Georgin Lavialle18*, Julien Rossignol19*, Arsène Mekhinian, MD, PhD18*, Olivier Kosmider, PharmD, PhD20*, Manuel Rodriguez21* and Olivier Hermine, MD, PhD17,22,23

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

*signifies non-member of ASH