-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.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

508.O1.6 508. Bone Marrow Failure: Advancing Our Biologic Understanding in Inherited and Acquired Bone Marrow Failure Disorders

Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Anemias, Adult, AML, aplastic anemia, Diseases, iPSCs, Diamond Blackfan Anemia, Bone Marrow Failure, Genetic Disorders, Fanconi Anemia, MDS, red blood cells, Biological Processes, DNA damage, MPN, PNH, Cell Lineage, erythropoiesis, Study Population, genomics, Clinically relevant, Myeloid Malignancies, hematopoiesis, immune mechanism
Saturday, December 5, 2020: 2:00 PM-3:30 PM
Moderators:
Daria V. Babushok, MD, PhD, and Austin G Kulasekararaj, MD, MBBS, MRCP, FRCPath, King’s College Hospital NHS Foundation Trust, King’s College, London, UK, and NIHR Wellcome King’s Clinical Research Facility
Disclosures:
Kulasekararaj: Alnylam: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Membership on an entity's Board of Directors or advisory committees; Regeneron: Honoraria, Membership on an entity's Board of Directors or advisory committees; BioCryst: Honoraria, Membership on an entity's Board of Directors or advisory committees; Achilleon: Honoraria, Membership on an entity's Board of Directors or advisory committees; Apellis: Membership on an entity's Board of Directors or advisory committees; Ra Pharma: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Alexion Pharmaceuticals Inc.: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene/BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees.
2:00 PM

Alyssa L. Kennedy, MD, PhD1, Kasiani C. Myers, MD2, James Bowman, PhD3*, Christopher J. Gibson, MD4, Gwen M. Muscato5*, Robert Klein1*, Kaitlyn Ballotti5*, Nicholas Camarda1*, Elissa M. Furutani, MD6, Chad E. Harris, MS5*, Shanshan Liu, PhD7*, Ashley Galvin5*, Maggie Malsch, MSN, RN5*, David C. Dale, MD8, John M Gansner, MD, PhD9, Taizo A. Nakano, MD10, Alison A. Bertuch, MD11, Adrianna Vlachos, MD12, Jeff H. Lipton, MD, PhD13, Paul Castillo, MD14*, James Connelly15*, John Edwards, MD16*, Nobuko Hijiya, MD17, Richard Ho, MD18*, Inga Hofmann, MD19, James N Huang, MD20, Sioban B. Keel, MD21, Adam J. Lamble, MD22, Bonnie W Lau, MD, PhD23, Kelly J. Walkovich, MD24, Maxim Norkin, MD25, Wendy Stock, MD26, Steffen Boettcher27*, Christian Brendel, PhD28*, Elliot Stieglitz, MD29, Mark D. Fleming, MD, DPhil30, Stella M. Davies, PhD, MBBS, MRCP31, Edie A. Weller32*, Chris Bahl, PhD3*, Scott L. Carter, PhD33*, Akiko Shimamura, MD, PhD34* and R. Coleman Lindsley, MD, PhD35

1Dana-Farber Cancer Institute, Boston, MA
2Department of Pediatrics, Division of Bone Marrow Transplantation and Immune Deficieny, Cincinnati Children's Hospital, Cincinnati, OH
3Institute for Protein Innovation, Boston, MA
4Department of Medical Oncology, Dana-Farber Cancer Institute, Chestnut Hill, MA
5Boston Children's Hospital, Boston, MA
6Boston Children's Cancer and Blood Disorders Center, Boston Children’s Hospital, Boston, MA
7Division of Hematology and Oncology and Biostatistics and Research Design Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Boston, MA
8Department of Medicine, University of Washington, Seattle, WA
9Hematology Division, Brigham and Women's Hospital, Newton, MA
10Children's Hospital Colorado, University of Colorado School of Medicine, Denver, CO
11Baylor College of Medicine, Houston, TX
12Feinstein Institute of Medical Research, Cohen Children's Medical Center, Manhasset, NY
13Blood and Marrow Transplant Program, Department of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada
14Shand's Children's Hospital, Department of Pediatrics, Division of Pediatric Hematology Oncology, University of Florida, Gainesville, FL
15Department of Pediatrics, Division of Pediatric Hematology Oncology, Vanderbilt University Medical Center, Nashville, TN
16Indiana Blood and Marrow Transplantation, Indianapolis, IN
17Pediatric Hematology Oncology Transplant, Columbia University Irving Medical Center, New York, NY
18Department of Pediatrics, Division of Pediatric Hematology/Oncology, Vanderbilt University Medical Center, Nashville, TN
19Department of Pediatrics, Division of Hematology/Oncology and Bone Marrow Transplantation, University of Wisconsin, Madison, WI
20Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco, Benioff Children's Hospital, San Francisco, CA
21Department of Medicine, Division of Hematology, Seattle, WA
22Division of Hematology-Oncology, Seattle Children's Hospital, Seattle, WA
23Johns Hopkins University, Grafton, NH
24University of Michigan, Ann Arbor, MI
25University of Florida, Gainesville, FL
26Department of Medicine, University of Chicago, Chicago, IL
27Medical Oncology and Hematology, University and University Hospital Zurich, Zurich, Switzerland
28Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston
29Department of Pediatrics, Division of Hematology-Oncology, UCSF, San Francisco, CA
30Department of Pathology, Boston Children's Hospital, Harvard Medical School, Boston, MA
31Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
32Institutional Centers for Clinical and Translational Research, Biostatistics, Boston Children’s Hospital, Boston, MA
33Dana Farber Cancer Institute, Boston, MA
34Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
35Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA

2:15 PM

Richa Sharma, MD1, Sushree Sangita Sahoo, PhD2*, Masayoshi Honda3*, Charnise Goodings-Harris, PhD1, Victor Pastor, PhD1*, Hauke Busch, PhD4,5*, Melanie Börries, PhD6*, Fabian Beier, MD7,8,9*, Miriam Erlacher, MD, PhD10,11*, Melchior Lauten12*, Marc Wold, PhD13*, Maria Spies, PhD14*, Charlotte M. Niemeyer, MD11,15,16,17 and Marcin Wlodarski, MD, PhD18,19

1St Jude Children's Research Hospital, Memphis, TN
2Hematology, St. Jude Children's Research Hospital, Memphis, TN
3Department of Biochemistry, University of Iowa, Iowa City, IA
4Lübeck Institute of Experimental Dermatology, Lübeck, Germany
5Institute of Molecular Medicine and Cell Research, University of Freiburg, Freiburg, Germany
6Institute of Molecular Medicine and Cell Research, Albert-Ludwigs-University, Freiburg, German Cancer Consortium (DKTK), partner site Freiburg, German Cancer Research Center (DKFZ), Heidelberg, Germany, Freiburg, Germany
7Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital Aachen, Aachen, Germany
8Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital Aachen, Aachen, Germany
9Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital Aachen, Aachen, NRW, Germany
10University Medical Center Freiburg, Freiburg, Germany
11German Cancer Consortium (DKTK) and German Cancer Research Center (DKFZ), partner site Freiburg, Freiburg, Germany
12Pediatrics, University Hospital Schleswig-Holstein, Campus Lübeck, Lübeck, Germany
13Department of Biochemistry, Carver College of Medicine, University of Iowa, Iowa City, IA
14University of Iowa, Iowa City, IA
15University Children's Hospital, Freiburg, Germany
16Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany
17Department of Pediatrics and Adolescent Medicine, Freiburg University Hospital, Freiburg, Germany
18Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN
19Department of Pediatrics and Adolescent Medicine, University Medical Center Freiburg, Freiburg, Germany

2:30 PM

Richard N Armstrong, PhD, BSc1*, Mariana Benicio, PhD1*, Houtan Moshiri, Ph.D1*, Hongbing Li, PhD1*, Bozana Zlateska, MD, MSc1*, Olga I. Gan, PhD2*, Eric R Lechman, Ph.D.3*, John E. Dick, PhD, FRS2 and Yigal Dror, MD, FRCPC1,4

1Department of Genetics and Genome Biology, The Hospital for Sick Children, Toronto, ON, Canada
2Princess Margaret Cancer Centre, University Health Network (UHN), Toronto, ON, Canada
3Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada
4Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, Toronto, ON, Canada

2:45 PM

Misam Zawit, MBBS1*, Jibran Durrani, MD1*, Wenyi Shen, MD2*, Vera Adema, PhD3*, Cassandra M Kerr, MS1*, Hassan Awada, MD1, Sunisa Kongkiatkamon, MD1*, Carmelo Gurnari, MD4*, Simona Pagliuca, MD1, Laila Terkawi1*, Bhumika J. Patel, MD3,5, Valeria Visconte, PhD1, Hetty E. Carraway, MD, MBA1,6, Thomas LaFramboise, Ph.D.7*, Seth J. Corey, MD8, Torsten Haferlach, MD9 and Jaroslaw P. Maciejewski, MD, PhD3

1Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
2Department of Translational Hematology and Oncology Research,Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
3Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
4Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland
5Leukemia Program, Department of Hematology and Medical Oncology, Cleveland Clinic, Cleveland, OH
6Department of Hematology and Medical Oncology, Leukemia Program, Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
7Department of Genetics and Genome Sciences Case Western Reserve University School of Medicine, Cleveland, OH
8Departments of Pediatrics, Cancer Biology, THOR, Cleveland Clinic, Cleveland, OH
9MLL Munchner Leukamie Labor Gmbh, Munchen, Germany

3:00 PM

Kohei Hosokawa, MD, PhD 1, Hiroki Mizumaki, MD1*, Takeshi Yoroidaka, MD1*, Hiroyuki Maruyama, MD, PhD1*, Tatsuya Imi, MD, PhD1*, Noriaki Tsuji, MD1*, Ryota Urushihara, MD1*, Mikoto Tanabe, MD1*, Yoshitaka Zaimoku, MD, PhD1*, Mai Anh Thi Nguyen, MD1*, Dung Cao Tran, MD1*, Ken Ishiyama, MD, PhD1, Hirohito Yamazaki, MD, PhD2*, Takamasa Katagiri, PhD3*, Kazuyoshi Hosomichi, PhD4*, Atsushi Tajima, PhD4*, Fumihiro Azuma5*, Seishi Ogawa, MD, PhD6 and Shinji Nakao, MD, PhD1

1Department of Hematology, Kanazawa University, Kanazawa, Japan
2Division of Transfusion Medicine, Kanazawa University Hospital, Kanazawa, Japan
3Clinical Laboratory Sciences, Kanazawa University Graduate School, Kanazawa, Japan
4Department of Bioinformatics and Genomics, Graduate School of Advanced Preventive Medical Sciences, Kanazawa University, Kanazawa, Japan
5HLA Laboratory, Japanese Red Cross Kanto-Koshinetsu Block Blood Center, Kotoku, Japan
6Department of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan

3:15 PM

Simona Pagliuca, MD1,2, Carmelo Gurnari, MD2,3*, Diego Chowell, PhD4*, Chirag Krishna5*, Bhumika J. Patel, MD6, Hassan Awada, MD2, Cassandra M Kerr, MS2*, Sunisa Kongkiatkamon, MD2,7*, Ashwin Kishtagari, MBBS2, Thomas Laframboise, PhD8*, Yogenthiran Saunthararajah, MD2, Babal K. Jha, PhD2*, Valeria Visconte, PhD2, Timothy Chan, MD, PhD9* and Jaroslaw Maciejewski, PhD2*

1Université de Paris, Paris, France
2Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
3Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
4Immunogenomics and Precision Oncology Platform, Memorial Sloan Kettering Cancer Center, New York
5Computational and Systems Biology Program, Memorial Sloan Kettering Cancer Center, New York
6Leukemia Program, Department of Hematology and Medical Oncology, Cleveland Clinic, Cleveland, OH
7Department of hematology, King Chulalongkorn Memorial Hospital, Bangkok, Thailand
8Genetics and Genome Sciences Department, Case Western Reserve University, Cleveland, OH
9Immunotherapy and Precision Immuno-Oncology, Cleveland Clinic, Cleveland

*signifies non-member of ASH