-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: Immune AA and VEXAS – Biology, Diagnosis and Treatment Clinically Relevant Abstract

Symposia: Bone Marrow Failure: Acquired Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Research, Acquired Marrow Failure Syndromes, Biological therapies, Antibody Therapy, adult, Translational Research, Bone Marrow Failure Syndromes, Non-Biological therapies, elderly, Clinical Research, Aplastic Anemia, Genetic Disorders, Chemotherapy, Diseases, Immune Disorders, immunodeficiency, real-world evidence, Therapies, registries, Infusion, young adult , Monoclonal Antibody Therapy, Study Population, Human, Transplantation
Monday, December 11, 2023: 10:30 AM-12:00 PM
Room 6DE (San Diego Convention Center)
Moderators:
Taha Bat, MD, and Swapna Thota, MD, University of Tennessee Health Science Center
Disclosures:
Bat: Alexion pharma: Membership on an entity's Board of Directors or advisory committees.
In this session we will focus on two diseases - immune AA and VEXAS. We will focus on the pathophysiology underpinning these disease and possible predictors for outcomes. Therapy will be discussed, with a particular emphasis on hematopoietic stem cell transplant.
10:30 AM

Bhavisha A. Patel, MD1, Marcela Ferrada, MD2*, Kaitlin Quinn, MD2*, Kristy Freeman, PA-C3*, Ivana Darden, RN1*, Wendy Goodspeed, RN2*, Lisa Duncan, RN3*, Roxanne Merkel, RN3*, Alice Fike, PA-C2*, Peter C Grayson, MD2*, Neal S. Young, MD1 and Emma M. Groarke, MD4

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
2NIH/NIAMS, Bethesda, MD
3NIH/NCI, Bethesda, MD
4Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD

10:45 AM

Pedro H. Prata, MD, PhD1, Dirk-Jan Eikema Sr.2*, Brian Piepenbroek3*, Austin Kulasekararaj, MD, MBBS, FRCPath, MRCP4, Beatrice Drexler, MD5*, Jennifer Tjon6*, Krista Vaht, MD7*, Mahmoud Aljurf, MD8*, Patrice Chevallier, MD9, Tobias Gedde-Dahl, MD10*, Ain Kaare, MD11*, Uwe Platzbecker, MD12, Urpu Salmenniemi13*, Thomas Schroeder14*, Jenny Louise Byrne, MD, PhD15*, Denis Guyotat16*, Nicolaus Kröger, MD17*, Fiorenza Barraco18*, Johan Maertens, MD, PhD19*, Eleni Tholouli, MD20*, Didier Blaise, MD, PhD21, Simona Sica22*, Jürgen Finke, MD23, Antonio M Risitano, MD, PhD24 and Regis Peffault De Latour25*

1Hematology and Transplantation, Hopital Saint-Louis, Paris, France
2EBMT Statistical Unit, Leiden, Netherlands
3EBMT Leiden Study Unit, Leiden, Netherlands
4King’s College London, London, United Kingdom
5Department of Hematology, University Hospital Basel, Basel, Switzerland
6Leiden University Medical Center, Leiden, NLD
7Sahlgrenska University Hospital, Gothenburg, SWE
8King Faisal Specialist Hospital & Research Centre, Oncology (Section of Adult Haematolgy/BMT) – Riyadh, Saudi Arabia, Riyadh, Saudi Arabia
9Service d'hématologie, CHU de Nantes, Nantes, France
10Oslo University Hospital, Rikshospitalet, Clinic for Cancer Medicine, Hematology Department, Section for Stem Cell Transplantation, Oslo, Norway
11Department of Hematology and Bone Marrow Transplant, Tartu University Hospital, Tartu, Estonia
12University Leipzig Medical Center, Leipzig, Germany
13Dept of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland
14Department of Hematology and Stem Cell Transplantation, University Hospital Essen, Essen, Germany
15Nottingham University, Nottingham, ENG, GBR
16CHU Saint-Etienne, Saint-Etienne, France
17University Hospital Eppendorf, Bone Marrow Transplantation Centre, Hamburg, Germany
18CH LYON SUD, PIERRE BENITE CEDEX, FRA
19Department of Hematology, University Hospitals Leuven, Leuven, Belgium
20Manchester Royal Infirmary, Clinical Haematology Department, Manchester, United Kingdom
21Program of Transplant and cellular immunotherapy, Department of Hematology, Institut Paoli Calmettes, Marseille, France
22Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Università Cattolica del Sacro Cuore, Rome, Italy
23Department of Hematology, Oncology and Stem Cell Transplantation, Faculty of Medicine, University of Freiburg, Freiburg, Germany
24AORN Moscati, Avellino, Italy
25Hematology and Transplantation Unit, Hôpital Saint Louis, AP-HP, Paris, France

11:00 AM

Masanori Yoshida, MD, PhD1*, Paula Arnold, PhD2*, Carmelo Gurnari, MD3,4, Charnise Goodings, PhD1, Shinsuke Hirabayashi, M.D., Ph.D.5*, Miriam Erlacher6*, Jaroslaw P. Maciejewski, M.D., Ph.D., FACP4, Charlotte Marie Niemeyer, MD, MA7 and Marcin W. Wlodarski, MD, PhD8

1Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN
2Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN
3Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
4Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
5Department of Pediatrics, Faculty of Medicine, Graduate School of Medicine, Hokkaido University, Sapporo, Japan
6Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, University Medical Center Freiburg, Germany, Freiburg, Germany
7Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, University of Freiburg, Freiburg, DEU
8St. Jude Children's Research Hospital, Memphis, TN

11:15 AM

Yoshitaka Zaimoku, MD, PhD1,2, Hirohito Yamazaki, MD, PhD3*, Minoru Kanaya, MD, PhD4*, Nobuhiro Hiramoto5*, Ken Ishiyama, MD, PhD6, Naoyuki Uchida, MD, PhD7, Noriko Doki, MD, PhD8, Ryusuke Yamamoto9*, Tetsuya Nishida, MD, PhD10*, Koichi Onodera11*, Shinichiro Machida12*, Yoshinobu Kanda13*, Tetsuya Eto, MD, PhD14*, Fumihiko Ishimaru, MD, PhD15, Makoto Onizuka, MD, PhD12, Tatsuo Ichinohe, MD, PhD16, Yoshiko Atsuta, MD, PhD17,18* and Yasushi Onishi, MD, PhD11*

1Department of Hematology, Kanazawa University Hospital, Kanazawa, Japan
2Department of Infection Control and Prevention, Kanazawa University Hospital, Kanazawa, Japan
3Division of Transfusion Medicine, Kanazawa University Hospital, Kanazawa, Ishikawa, Japan
4Blood Disorders Center, Aiiku Hospital, Sapporo, Japan
5Department of Hematology, Kobe City Medical Center General Hospital, Kobe, Hyogo, Japan
6Department of Hematology, National Center for Global Health and Medicine, Tokyo, Japan
7Department of Hematology, Federation of National Public Service Personnel Mutual Aid Associations Toranomon Hospital, Tokyo, Japan
8Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan
9Department of Hematology, Kobe City Medical Center General Hospital, Kobe, Japan
10Department of Hematology, Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital, Nagoya, Japan
11Department of Hematology, Tohoku University Hospital, Sendai, Japan
12Department of Hematology/Oncology, Tokai University School of Medicine, Isehara, Japan
13Division of Hematology, Jichi Medical University Saitama Medical Center, Saitama, Japan
14Department of Hematology, Hamanomachi Hospital, Fukuoka, JPN
15Japanese Red Cross Kanto-Koshinetsu Block Blood Center, Tokyo, Japan
16Department of Hematology and Oncology, Research Institute For Radiation Biology and Medicine, Hiroshima, Japan
17Department of Registry Science for Transplant and Cellular Therapy, Aichi Medical University School of Medicine, Nagakute, Japan
18Japanese Data Center For Hematopoietic Cell Transplantation, Nagakute, Japan

11:30 AM

Carlos Bravo-Perez, MD1,2*, Carmelo Gurnari, MD1,3, Luca Guarnera, MD1*, Naomi Kawashima, MD, PhD1*, Serhan Unlu, MD1*, Aashray Mandala, BS1*, Nakisha D Williams, MBBS1*, Arooj Ahmed, MD1*, Yasuo Kubota, MD, PhD4*, Arda Durmaz, PhD1*, Valeria Visconte, PhD1 and Jaroslaw P. Maciejewski, M.D., Ph.D., FACP1

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
4Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Beachwood, OH

11:45 AM

Carmelo Gurnari, MD1,2, Linda Koster3*, Laurien Baaij3*, Mael Heiblig4*, Ibrahim Yakoub-Agha, MD, PhD5*, Jakob R. Passweg, MD, MS6, Jennifer Clay Sr., MD, PhD7*, Jurgen Kuball, MD8, Alessandro Rambaldi, M.D.9, Patrick John Hayden, MD10, Manuela Badoglio11*, Francesco Onida, MD12*, Christof Scheid, MD13*, Franco Franceschini14*, Arsene Mekinian15*, Sinisa Savic, MD, PhD16*, Maria Teresa Voso, MD1, Joanna Drozd-Sokolowska, MD, PhD17*, John Snowden, MD18*, Kavita Raj, MD, PhD19, Tobias Alexander20*, Marie Robin, MD21*, Raffaella Greco22* and Donal P McLornan, MD, PhD19*

1Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
2Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
3EBMT Leiden Study Unit, Leiden, Netherlands
4Hematology Department, Centre Hospitalier Lyon Sud, Pierre-Bénite, France
5CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000, LILLE, FRA
6Department of Hematology University Hospital of Basel, Basel, Switzerland
7Yorkshire Blood & Marrow Transplant Program, Leeds, United Kingdom
8Utrecht University, University Medical Centre, Utrecht, Netherlands
9Department of Hematology, ASST Papa Giovanni XXIII, Bergamo, ITA
10Department of Haematology, Trinity College Dublin, St. James’s Hospital, Dublin, Ireland
11EBMT Paris Study Unit, Paris, France
12ASST Fatabanefratelli-Sacco, University of Milan, Milano, Italy
13Department of Internal Medicine I, University Hospital Cologne, Cologne, Germany, Cologne, Germany
14Rheumatology Unit and Chair, University and ASST Spedali Civili, Brescia, Italy, Brescia, ITA
15Saint Antoine Hospital AP-HP, Paris, France
16University of Leeds, Leeds, United Kingdom
17Central Clinical Hospital, The Medical University of Warsaw, Warsaw, Poland
18Sheffield Blood & Marrow Transplant and Cellular Theraphy Programme, Sheffield, United Kingdom
19University College London Hospitals NHS Trust, London, United Kingdom
20Charité University Medicine, Berlin, DEU
21Hopital Saint-Louis, Paris, France
22Hematology and Bone Marrow Transplantation Unit, I.R.C.C.S. Ospedale San Raffaele, Milano, Italy

*signifies non-member of ASH