Scientific Workshops

Moderators:

Marcin W. Wlodarski, MD, PhD
St. Jude Children's Research Hospital
Memphis, IN

Lucy A. Godley, MD, PhD
The University of Chicago
Chicago, IL

Moderators:

Paul P. Liu, MD, PhD
National Institutes of Health
Bethesda, MD

Jun J. Yang, PhD
St. Jude Children's Research Hospital
Memphis, TN

Speakers:

Ravi Majeti, MD, PhD
Stanford University
Stanford, CA
Modeling RUNX1-FPD/Acute Myeloid Leukemia Mutations in Primary Human Hematopoietic Stem Cells

Marcela Cavalcante De Andrade Silva, MD
ICESP
Maceio, Brazil
GATA2 Deficiency Syndrome and Functional Testing

Emery H. Bresnick, PhD
University of Wisconsin
Middleton, WI
GATA2 Deficiency Syndrome and Functional Testing

Parvathy Venugopal, PhD
Centre for Cancer Biology, SA Pathology and University of South Australia
Adelaide, Australia
Modeling Germline GATA2 Mutations to Identify Biological Pathways Associated with Hematological Malignancy Development in Families

Victor Bengt Pastor Loyola, PhD
University Medical Center — University of Freiburg
Freiburg, Germany
Interpretation and Functional Assessment of SAMD9 and SAMD9L Variants

Richard James, PhD
Seattle Children's Research Institute
Seattle, WA
Functional Determination of All Possible Disease-Associated Variants in a Region of CARD11 Using Saturation Genome Editing

Moderators:

Thierry M Leblanc, MD
Robert Debré Hospital
Paris, France

Panagiotis Baliakas, MD
Uppsala University
Uppsala, Uppsala, Sweden

Speakers:

Abhishek A. Mangaonkar, MBBS
Mayo Clinic
Rochester, MN
Novel Variant in MECOM Gene Associated with Bone Marrow Failure and ASXL1 Mutated Clonal Evolution

Marie Sebert, MD, PhD
Hopital Saint-Louis
Paris, France
MECOM-Mutated Severe Bone Marrow Failure

Taizo A. Nakano, MD
University of Colorado School of Medicine
Denver, CO
Expanded Phenotyping and Clonal Abnormalities Beyond Bone Marrow Failure in MECOM-Associated Syndromes

Kasiani C. Myers, MD
Cincinnati Children's Hospital
Cincinnati, OH
Myelodysplastic Syndrome and Acute Myeloid Leukemia in the Leukemia Predisposition Disorder Shwachman Diamond Syndrome

Joanne Yacobovich, MD, MPH
Tel Aviv University
Tel Aviv, Israel
Pancytopenia in Early Infancy- Diamond-Blackfan Anemia with Myelodysplastic Syndrome (MDS) or Congenital MDS?

This session will focus on speakers who will share their experiences in international registries and collaborations designed to accelerate progress in rare hematologic conditions.

Moderators:

Akiko Shimamura, MD, PhD
Boston Children's Hospital
Boston, MA

Anna L. Brown, PhD,BSc
Centre For Cancer Biology, SA Pathology/IMVS
Adelaide, SA, Australia

Speakers:

Charlotte M. Niemeyer, MD
Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg,
Freiburg, Germany
EWOG-MDS/JMML/SAA: From Lumpers to Splitters and How to Move the Field

Akiko Shimamura, MD, PhD
Boston Children's Hospital
Boston, MA
NAPAAC: Accelerating Research in Pediatric Marrow Failure and Myelodysplastic Syndrome

Alison A. Bertuch, MD
Baylor College of Medicine
Houston, TX
Clinical Care Consortium for Telomere Associated Ailments

Sharon A. Savage, MD
National Cancer Institute
Bethesda, MD
Advancing RAS/Rasopathy Therapies (ART): An NCI-Sponsored Intramural and Extramural Collaboration for the Study of Rasopathies

Anna L. Brown, PhD,BSc
Centre For Cancer Biology, SA Pathology/IMVS
Adelaide, SA, Australia
Aggregation and Analysis of Genomics Data in Familial Platelet Disorders with Predisposition to Myeloid Malignancy, Shows the Spectrum of Mutations Is Different

Simone K Feurstein, MD
University of Chicago
Chicago, IL
Frequency of Causative Germline Genetic Variants in Young Adults with Myelodysplastic Syndrome or Aplastic Anemia Irrespective of Family History

Moderators:

Chimene Kesserwan, MD
St. Jude Children's Hospital
Memphis, TN

Michael C. Chicka, PhD
Prevention Genetics
Marshfield, WI

Speakers:

Lucy Fox, MD
Peter MacCallum Cancer Centre
Melbourne, Australia
Significant Phenotypic Variability Across a Large Pedigree Harboring a Pathogenic Synonymous GATA2 Mutation

Xi Luo, PhD
Baylor College of Medicine
Houston, TX
Update on ASH-Sponsored Clingen Variant Curation Panel for Myeloid Malignancies

Shruthi Mohan, PhD
University of North Carolina, Chapel Hill
Chapel Hill, NC
Update on ASH-Sponsored Clingen Variant Curation Panel for Myeloid Malignancies

Marlie H. Fisher, BA
University of Colorado
Aurora, CO
Investigating the Role of ETV6 in Peripheral Inflammation and Megakaryopoiesis

Marena Rebekka Niewisch, MD
National Institutes of Health
Bethesda, MD
Prevalence of PARN Germline Mutations in Disease and the General Population

Moderators:

Kayla Hamilton, MS, CGC
Dana-Farber Cancer Institute
Boston, MA

Sara Lewis, MS, LCGC
St. Jude Children's Research Hospital
Memphis, TN

Speakers:

Piers Blombery, MBBS
University of Melbourne
East Melbourne, Australia
Clinical Utility and Implementation of Accredited Genomic Testing in Bone Marrow Failure Syndromes

Michael W. Drazer, MD
The University of Chicago
Chicago, IL
Molecular Profiling in Patients with Germline Predisposition Syndromes: When to Act, When to Watch

Lea C. Cunningham, MD
National Institutes of Health
Bethesda, MD
Molecular Profiling in Patients with Germline Predisposition Syndromes: When to Act, When to Watch

Moderators:

Outi Kilpivaara, PhD
University of Helsinki
Helsinki, Finland

Elvira Velloso, MD, PhD
University of São Paulo
São Paulo, Brazil

Speakers:

Yizhen Li, PhD
St. Jude Children’s Research Hospital
Memphis, TN
Germline RUNX1 Variation and Predisposition to T-Cell Acute Lymphoblastic Leukemia in Children

Amy M Trottier, MSc,MD,FRCPC
The University of Chicago
Chicago, IL
Heterozygous CSF3R Mutations: A Possible New Germline Predisposition Syndrome

Evadnie Rampersaud, PhD
St. Jude Children's Research Hospital
Memphis, TN
Whole Genome Sequencing of Multiplex Classic Hodgkin Lymphoma Pedigrees

Ulla Wartiovaara-Kautto, MD,PhD
Helsinki University Hospital, Helsinki University
Helsinki, Finland
Germline Gene Aberrations Are Common in High-Risk Adult and Pediatric Acute Lymphoblastic Leukemia Patients

Deepak Singhal, MBBS,MD,FRACP,FRCPA
Royal Adelaide Hospital
Adelaide, SA, Australia
Deleterious Germline Variants, Especially in the DNA Repair Pathway, Are Common in Therapy-Related Myeloid Neoplasms and Patients with Multiple Cancers

Co-Chairs:

Marcin W. Wlodarski, MD, PhD
St. Jude Children's Research Hospital
Memphis, IN

Lucy A. Godley, MD, PhD
The University of Chicago
Chicago, IL

Co-chair ASH Friday Scientific Workshop Hematology and Aging

Co-Chair:

Ashley E. Rosko, MD
The Ohio State University
Columbus, OH

Speaker:

Felipe Sierra, PhD
National Institute of Aging
Bethesda, MD
Geroscience Integration in Hematologic Disorders

Moderator:

Heidi D. Klepin, MD,MS
Wake Forest University School of Medicine
Winston Salem, NC

Speakers:

John C Byrd, MD
The Ohio State University
Columbus, OH
Biology of Acute Myeloid Leukemia in Older Adults

Jing Zhang, PhD
University of Wisconsin-Madison
Madison, WI
Oncogenic Ras Mutations in CHIP Vs Chronic Myelomonocytic Leukemia

Christopher Y. Park, MD, PhD
New York University School of Medicine
New York, NY
Hematopoietic Stem Cell Heterogeneity in Aging

Moderator:

Tanya M Wildes, MD, MSCI
Washington University
Saint Louis, MO

Speakers:

Evangelos Terpos, MD,PhD
National and Kapodistrian University of Athens, School of Medicine
Drosia, Attiki, Greece
Molecular Mechanisms in Proteasome Inhibitor Induced Cardiotoxicty: Focus on Carfilzomib

Daniel W. Sherbenou, MD, PhD
University of Colorado Anschutz Medical Campus
Aurora, CO
Drug Sensitivity Profiling in Patients with Multiple Myeloma: Who Are Older or Transplant-Ineligible?

Adriana Pascual, MD
Hospital Universitario Infanta
Elana, Spain
Patient Portal and Navigation Program in Monitoring PROMs and HRQoL for Older Adults with Hematologic Malignancies

Moderator:

Reena Jayani, MD
Vanderbilt University Medical Center
Tampa, FL

Speakers:

Jennifer R Brown, MD, PhD
Dana-Farber Cancer Institute
Boston, MA
Side Effects of Ibrutinib in Relation to Age

Daniel Addison, MD
The Ohio State University
Columbus, OH
Cardiovascular Disease Among Older Adults in the Era of Bruton Tyrosine Kinase Inhibitors

Alberto Lopez Garcia, MD
Clinico San Carlos University Hospital
Madrid, Spain
Cardiac Toxicity of Anthracyclines in Older Adults with Lymphoma: Predictive Factors and Monitoring Recommendations

Marco L. Davila, MD, PhD
Moffitt Cancer Center
Tampa, FL
Defects in Aged Chimeric Antigen Receptor T Cell Function Can be Mitigated By Transcription Factor Modulation of Gene Accessibility

In the field of hemostasis and thrombosis, aging is providing new and unique challenges for the clinical management of our patients. With the introduction of prophylactic regimens in hemophilia and the availability of novel therapies, the life expectancy and disease burden for patients with bleeding disorders continues to improve. The impact and influence of age-related co-morbidities (such as cardiovascular disease) in this cohort requires careful consideration and improved evidence based approaches. With aging in the general population the risk of thrombosis increases, and both choice of and suitability for anticoagulation is influenced by co-morbidities. In reviewing the current evidence base in this field, discussing knowledge gaps and therapeutic strategies, this session will provide an insight into the influence of aging on hemostatic and thrombotic conditions. Prof. Frank Leebeek will examine the impact of aging for patients with Von Willebrand Disease, including data derived from the Von Willebrand Disease in the Netherlands (WIN) study. Prof. Craig M. Kessler will highlight the new and evolving challenges in the management of older people living with hemophilia. Prof. Suzanne C. Cannegieter will discuss the heightened risk of venous and arterial thromboembolism in the aging population and optimal management strategies in this cohort.

Moderator:

Michelle Lavin, MB, PhD, FRCPath
RCSI
Dublin 2, Ireland

Speakers:

Frank W.G. Leebeek, MD, PhD
Erasmus University Medical Center
Rotterdam, Netherlands
The Impact of Aging in Patients with Von Willebrand Disease

Craig M. Kessler, MD
Georgetown University Medical Center
Washington, DC
Embracing New Challenges: Haemophilia and Aging

Suzanne C. Cannegieter, MD, PhD
Leiden University, Netherlands
Leiden, Netherlands
Venous and Arterial Thromboembolism in the Aging Population

Moderators:

Rebecca L. Olin, MD
University of California, San Francisco
San Francisco, CA

Ashley E. Rosko, MD
The Ohio State University
Columbus, OH

Speaker:

Bindu Kanapuru, MD
U.S. Food and Drug Administration
Silver Spring, MD
Efforts By the U.S. Food and Drug Administration to Expand Eligibility Criteria in Hematology and Oncology Clinical Trials

Co-Chair:

Rebecca L. Olin, MD
University of California, San Francisco
San Francisco, CA

Chair:

Philip L. McCarthy, MD
Roswell Park Comprehensive Cancer Center
Buffalo, NY

Co-Chair:

Moderator:

Manisha Bhutani, MD
Levine L. Cancer Institute
Charlotte, NC

Speakers:

Mehmet K. Samur, PhD
Dana Farber Cancer Institute
Boston, MA
Molecular Landscape of Multiple Myeloma

G David Roodman, MD, PhD
Indiana University School of Medicine
Indianapolis, IN
Bone Marrow Microenvironment in Multiple Myeloma

Manisha Bhutani, MD
Levine L. Cancer Institute
Charlotte, NC
Immune Dysregulation in Multiple Myeloma

Zihai Li, MD, PhD
The Ohio State University
Columbus, OH
Immune Dysregulation in Solid Tumors – Common Links with Multiple Myeloma

Madhav V. Dhodapkar, MBBS
Emory University
Atlanta, GA
Immune Microenvironment in Multiple Myeloma

Samir Parekh, MD
Icahn School of Medicine at Mount Sinai
New York, NY
Multi-Dimensional Single Cell Immune Profiling in Myeloma

Moderator:

Manisha Bhutani, MD
Levine L. Cancer Institute
Charlotte, NC

Recent advances in the treatment of multiple myeloma have made the need of more sophisticated assessment of treatment response desirable. Achievement of minimal residual disease negativity has been identified as a strong prognostic factor in myeloma and several techniques are used to assess small amounts of malignant cells or monoclonal proteins. Since myeloma is in many patients not involving the bone marrow and skeleton in a homogeneous but in a rather patchy fashion, sensitive imaging techniques complete the arsenal of tools to assess response.

Moderator:

Jens Hillengass, MD
Roswell Park Comprehensive Cancer Center
Buffalo, NY

Speakers:

Noemi Puig, MD, PhD
Hospital Universitario de Salamanca Hematología. Instituto de investigación biomédica de Salamanca (IBSAL)
Salamanca, Spain
Flow Cytometric Measurement Issues for MRD and IP in Multiple Myeloma

Neha Korde, MD
Memorial Sloan Kettering Cancer Center
South Orange, NJ
MALDI-TOF Mass Spectometry in Multiple Myeloma

Urvi A Shah, MD
Memorial Sloan Kettering Cancer Center
New York, NY
89Zr-Labeled CD38 MoAb Immuno-PET: Targeted Myeloma Imaging

Francesca Gay, MD
Azienda Ospedaliero City of Health and Science of Turin
Turin, Italy
Next Generation Sequencing Comparison to Next Generation Flow Cytometry

Andrew Spencer, DM
Alfred Health-Monash University
Melbourne, Australia
Cytof Populations during Myeloma Treatments

Paola Neri, MD, PhD
University of Calgary
Calgary, AB, Canada
Single Cell Immune Profiling in Multiple Myeloma

Ruth M. De Tute, BSc,MSc
St James Hospital
Leeds, United Kingdom
MRD Testing of Myeloma XI Patients

Moderator:

Jens Hillengass, MD
Roswell Park Comprehensive Cancer Center
Buffalo, NY

Summary of session

Moderator:

Nina Shah, MD
University of California San Francisco
San Francisco, CA

Speakers:

David E. Avigan, MD
Beth Israel Deaconess Medical Center
Boston, MA
Designing the Optimal Vaccine and T Cell Strategy for Myeloma

Adam D. Cohen, MD
University of Pennsylvania
Philadelphia, PA
Immunophenotyping of Apheresis and CAR T Cell Products and Predictors of Response to CAR T Cell Therapy in Myeloma

Sham Mailankody, MBBS
Memorial Sloan Kettering Cancer Center
New York, NY
CAR T Therapy for Multiple Myeloma: What Next?

Damian J. Green, MD
Fred Hutchinson Cancer Research Center
Seattle, WA
The Role of Antibody Binding Capacity in Bcma CAR-T Cell Therapy for Multiple Myeloma

Moderator:

Nina Shah, MD
University of California San Francisco
San Francisco, CA

Moderator:

Sarah A. Holstein, MD,PhD
University of Nebraska Medical Center
Omaha, NE

Speakers:

Nicole J. Gormley, MD
U.S. Food and Drug Administration
Silver Spring, MD
Challenges of Multiple Myeloma Drug Approval and the Issues of Drug Safety in the Multiple Myeloma Population

Vera Jean Suman, PhD
Mayo Clinic -CCS
Rochester, MN
Challenges of Progression-Free Survival and Overall Survival Endpoints in Multipe Myeloma

Qian Shi, PhD
Division of Biomedical Statistics and Informatics, Mayo Clinic
Rochester, MN
iiTEAMM Project: Evaluating Minimal Residual Disease Surrogate Endpoint in Multiple Melanoma – An International Collaboration

Luciano J. Costa, MD, PhD
University of Alabama at Birmingham
Vestavia, AL
Harmonization of MRD Assessment Timepoints and Outcomes Reporting

Moderator:

Sarah A. Holstein, MD,PhD
University of Nebraska Medical Center
Omaha, NE

Chair:

Philip L. McCarthy, MD
Roswell Park Comprehensive Cancer Center
Buffalo, NY

Co-Chair:

Co-Chairs:

Alan E. Mast, MD,PhD
Blood Center of Wisconsin
Milwaukee, WI

Grzegorz Nowakowski, MD
Mayo Clinic
Rochester, MN

Speaker:

John Concato, MD
Food and Drug Administration
Silver Spring, MD
Utilization of Real-World Data in Hematologic Drug Development

Moderators:

Juliana Perez, MD
University of Wisconsin
Madison, WI

Richard F. Little, MD,MPH
National Cancer Institute
Washington, DC

Speakers:

Donald Berry, MD, PhD
The University of Texas MD Anderson Cancer Center
Houston, TX
Statistical Science Underlying Trial Design

Carsten Utoft, MD, PhD
Copenhagen University Hospital
Roskilde, DNK
Clinical Trial to Change Immune Dysfunction in Chronic Lymphocytic Leukemia Based on Modelling Nationwide Registry Data

Michael Crump, MD
Princess Margaret Cancer Centre, University Health Network
Toronto, ON, Canada
Opening Doors for Chimeric Antigen T-Cells: Scholar-1 Project

Moderator:

Laura Maria Fogliatto, MD, PhD
Universidade Federal do Rio Grande do Sul (UFRS)
Porto Alegre, Brazil

Speakers:

Fangxin Hong, PhD
Dana-Farber Cancer Institute
Boston, MA
Efficient Statistical Design

Umberto Vitolo, MD
University Hospital Città della Salute e della Scienza Torino
Torino, Italy
Learning from Failures: Impact of Patient Selection on Outcomes in Front-Line Trials of Diffuse Large B-cell Lymphoma

Gallia G Levy, MD, PhD
Genentech, Inc.
South San Francisco, CA
Obtaining Approval of a Drug for Rare Blood Disorders

Moderators:

Donna DiMichele, MD
National Heart, Lung, and Blood Institute
Bethesda, MD

Georg Lenz, Professor Dr., MD
University of Muenster
Munster, Germany

Speakers:

Marie E. Steiner, MD,MS
University of Minnesota
Minneapolis, MN
Hemostasis Endpoints in Pediatric and Adult Clinical Trials: Summary of NHLBI Workshop

Mary M. Horowitz, MD, MS
Medical College of Wisconsin
Milwaukee, WI
Conducting Successful Trials in Blood and Marrow Transplantation

Arash A. Alizadeh, MD, PhD
Stanford University Medical Center
Stanford, CA
Liquid Biopsies As Integrated Biomarkers in Pediatric and Adult Hematologic Malignancies

Co-Chairs:

Alan E. Mast, MD,PhD
Blood Center of Wisconsin
Milwaukee, WI

Grzegorz Nowakowski, MD
Mayo Clinic
Rochester, MN

Co-Chairs:

James M. Foran, MD
Mayo Clinic Florida
Jacksonville, FL

Wendy Cozen, DO,MPH
University of Southern California
Los Angeles, CA

Moderators:

Wendy Cozen, DO,MPH
Univ. of Southern California Norris Cancer Ctr.
Los Angeles, CA

James R. Cerhan, MD, PhD
Mayo Clinic
Rochester, MN

Speakers:

Joseph L. Wiemels, PhD
UCSF
San Francisco, CA
Ctytomegalovirus and Risk of Acute Lymphoblastic Leukemia: Can We Translate to Screening and Prevention?

Xiaomei Ma, MD, PhD
Yale University
New Haven, CT
Early Life Risk Factors and Early-Onset Non-Hodgkin Lymphoma

Sam M. Mbulaiteye, MBBChir, MPhil
National Cancer Institute
Rockville, MD
Epidemiology of Burkitt Lymphoma - EMBLEM Cohort

Jonathan N. Hofmann
National Cancer Institute
Bethesda, MD
Permethrin and Myeloma Risk: Investigating the Biologic Plausibility and Potential Mechanisms of Action

Jean L. Koff, MD
Winship Cancer Institute of Emory University
Atlanta, GA
Is Lymphoma Different in Patients with Autoimmune Disease?

Moderators:

Wendy Cozen, DO,MPH
University of Southern California
Los Angeles, CA

James R. Cerhan, MD, PhD
Mayo Clinic
Rochester, MN

Speakers:

Akil Merchant, MD
Cedars-Sinai Medical Center
Los Angeles, CA
Can the Role of the Tumor Microenvironment be Extended to Population-Based Studies?

Caroline Besson, MD,PhD
Inserm U1018
Villejuif, France
New Insights Regarding the Epidemiology of HCV and HBV Related Lymphomas

Howard A. Liebman, MD
University of Southern California
Los Angeles, CA
How Can We Expand the Study of Rare Hematologic Diseases to Populations in Diseases Such As Antiphospholipid Syndrome?

Moderators:

James M. Foran, MD
Mayo Clinic Florida
Jacksonville, FL

Lindsay M. Morton, PhD
National Cancer Institute, NIH, DHHS
Bethesda, MD

Speakers:

Steven D. Gore, MD
Yale University School of Medicine
New Haven, CT
NHLBI Myelodysplastic Syndrome Natural History Study: What Are We Learning?

Jenny Poynter, PhD
University of Minnesota
Minneapolis, MN
Insights from a Population-Based Case-Control Study of Myelodysplastic Syndrome: Results from the AIMMS Study

Rong Wang, PhD
Yale University School of Public Health
New Haven, CT
Patterns of Care and Comparative Effectiveness in Myelodysplastic Syndrome: Epidemiology of Clinical Intervention

Theresa Keegan, PhD, MS
University of California, Davis
Sacramento, CA
Disparities in Late Medical Conditions Following Treatment Among Adolescent and Young Adult Survivors of Acute Myeloid Leukemia

Dr. Rong Wang will discuss treatment pattern and economic burden of myelodysplastic syndromes.

Moderators:

James M. Foran, MD
Mayo Clinic
Jacksonville, FL

Lindsay M. Morton, PhD
National Cancer Institute, NIH, DHHS
Bethesda, MD

Speakers:

Bronwen E. Shaw, PhD, MRCP, FRCPath
Medical College of Wisconsin
Milwaukee, WI
CIBMTR As a Resource for Epidemiology Research for Myeloid Malignancy

Ezgi Karaesmen, MSc
The Ohio State University
Columbus, OH
Exploring Genetic Associations with Bone Marrow Transplant Outcomes

Moderators:

James M. Foran, MD
Mayo Clinic
Jacksonville, FL

Lindsay M. Morton, PhD
National Cancer Institute, NIH, DHHS
Bethesda, MD

Speakers:

Brenda M. Birmann, BA, DSc, MSc
Brigham and Women's Hospital and Harvard Medical School
Boston, MA
Clonal Hematopoiesis and Risk of Acute Myeloid Leukemia: A Prospective Analysis in Two Large Cohorts

Pinkal Desai, MD,MPH
Weill Cornell Medical College
New York, NY
Clonal Hematopoesis, Myeloid Precursor States, and Progression to Myeloid Disorders in Healthy and High-Risk Populations

Wolfgang Kern, MD
Munich Leukemia Laboratory
Munich, Germany
Population Observations on Clonal Hematopoiesis – European Perspective

Co-Chairs:

James M. Foran, MD
Mayo Clinic Florida
Jacksonville, FL

Wendy Cozen, DO,MPH
Univ. of Southern California Norris Cancer Ctr.
Los Angeles, CA

Co-Chairs:

Jeffrey I. Zwicker, MD
Beth Israel Deaconess Medical Center Harvard Medical School
Boston, MA

Lisa B. Kreuziger, MD, MS
Blood Research Institute, Versiti
Milwaukee, WI

Myeloproliferative neoplasms (MPNs) are comprised of three phenotypically distinct clonal disorders: polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). These diseases are characterized by excessive production of blood cellular components including red blood cells, platelets, and/or leukocytes. Thrombosis is the leading cause of mortality among patients with MPNs, accounting for 40% of deaths. Current thrombosis prevention strategies rely on non-targeted antiplatelet and cytoreductive therapies, such as aspirin and phlebotomy. However, these therapies fail to completely mitigate thrombotic risk. Many MPN patients express somatic mutations either in Janus kinase-signal transductor and activator of transcription (JAK-STAT) Almost 95% of patients with PV have a JAK2 V617F mutation, and up to 50% of ET and MF patients have a JAK2 V617F mutation. The risk of thrombosis in MPN patients with JAK2 V617F far surpasses that of patients with secondary thrombocythemia. Thus, it is likely that a pro-thrombotic mechanism independent of increased blood cellular components contributes to thrombosis in MPN. Today’s panel highlights advances in the basic and translational sciences surrounding arterial and venous thrombosis in MPNs.

Moderators:

Gabriela S. Hobbs, MD
Massachusetts General Hospital
Boston, MA

Joan D. Beckman, MD,PhD
University of Minnesota
Minneapolis, MN

Speakers:

Nan Wang, PhD
Columbia University Medical Center
New York, NY
Mechanisms of Atherosclerosis and JAK2

Brandi Reeves, MD
University of North Carolina-Chapel Hill
Chapel Hill, NC
Neutrophil Tissue Factor and Thrombosis in Myeloproliferative Neoplasms

Ofir Wolach, MD
Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center
Petah Tikva, Israel
Inhibition of NET Formation with Ruxolitinib

Vivien Chen, PhD, MBBS
University of Sydney
Sydney, Australia
Procoagulant Platelets and JAK2 V617F Mutation

Dr. Wang will discuss the mechanism underlying atherosclerosis promoted by JAK2V617F driven clonal hematopoiesis. The therapeutic potential of targeted IL-1b inhibition will be discussed as well. Dr. Wolach will discuss neutrophil extracellular traps (NETs) as a potential mechanism for venous thrombosis in myeloproliferative neoplasms. The use of JAK inhibitors to reduce NET formation will also be discussed. Dr Chen will discuss a novel assay for procoagulant platelets that segregates with JAK2V617F mutuation status and thrombosis, as a potential risk stratification marker in essential thrombocythaemia.

Moderators:

Lalitha V. Nayak, MD
Case Western Reserve University
Cleveland, OH

Christophe Dubois, PhD
Aix Marseille Université
Marseille, France

Speakers:

Matthew J. Flick, PhD
University of North Carolina
Chapel Hill, NC
Role of PAR-1 Signaling in Pancreatic Tumor Progression

Henri H. Versteeg, PhD
Leiden University Medical Center
Leiden, N-Holland, NLD
Role of TF/FVII in Tumor Progression

Evi X. Stavrou, MD
Louis Stokes Cleveland VA Medical Center
Cleveland, OH
Host and Tumor Factor XII Drive Ovarian Cancer Maintenance and Progression

Joseph S. Palumbo, MD
Children's Hospital Med. Ctr.
Cincinnati, OH
Thrombin Tips the Balance in Favor of Adaptive Tumor Immunity

Anna Falanga, MD
University of Milan Bicocca/Hospital Papa Giovanni XXIII
Bergamo, Italy
Hemostatic Biomarkers and Prediction of Cancer Prognosis

Dr. Anna Falanga will discuss the role of markers of hypercoagulation to predict cancer diagnosis and disease outcome in patients from the Hypercan Study. Dr. Henri Versteeg will discuss the role of coagulation factors in pre-metastatic events and novel options to inhibit these processes Dr. Joseph Palumbo will discuss the role of thrombin in promoting an adaptive tumor immune response.

This session presents new mechanisms by which the hemostatic system support tumor progression and opportunities for therapeutic intervention.

Moderators:

Wolfram Ruf, MD
Johannes Gutenberg University Medical Center Mainz
Mainz, CA, Germany

Agnes Y. Lee, MD, MSc
University of British Columbia
Vancovuer, BC, Canada

Speakers:

Claudine Graf, MD, PhD
Johannes Gutenberg University Medical Center Mainz
Mainz, Germany
FXa Inhibitors Target Tumor-Associated Macrophage FXa-PAR2 Signaling

Anthony Maraveyas, MD, PhD
University of Hull
Hull, United Kingdom
Influence of DOAC and LMWH on Pancreatic Tumor Cell Growth

Julia Volz
Universitätsklinikum Würzburg
Würzburg, DEU
Inhibition of Platelet GPVI Increases Chemotherapeutic Efficacy

Co-Chairs:

Jeffrey I. Zwicker, MD
Beth Israel Deaconess Medical Center Harvard Medical School
Boston, MA

Lisa B. Kreuziger, MD, MS
Blood Research Institute, Versiti
Milwaukee, WI

Co-Chairs:

Ross L. Levine, MD
Memorial Sloan Kettering Cancer Center
New York, NY

Patricia Ernst, PhD
University of Colorado Denver
Aurora, CO

Moderator:

Eric Pietras, PhD
University of Colorado
Aurora, CO

Speakers:

Leonard I. Zon, MD
Harvard University
Brookline, MA
Macrophages in Early Hematopoietic Development

Sandra S. Zinkel, MD, PhD
Vanderbilt University School of Medicine
Nashville, TN
Necroptosis and Inflammatory Microenvironment Impact on Red Cell Development in CHIP: Phenomics, Human Genetics, and Mice

Qing Li, MD, PhD
University of Michigan
Ann Arbor, MI
Endoplasmic Reticulum Associated Degradation Preserves Hematopoietic Stem Cell Quiescence and Self-Renewal By Restricting mTOR Activity

Cesar Nombela Arrieta, PhD
University and University Hospital Zürich
Zürich, MA, Switzerland
Chronic LCMV Infection and the Bone Marrow Niche

Moderator:

Claudia Lengerke, MD
University Hospital Basel
Basel, Switzerland

Speakers:

Christopher M. Sturgeon, PhD
Washington University School of Medicine
Saint Louis, MO
Identification of a Retinoic Acid-Dependent Definitive Hematopoietic Progenitor from Human Pluripotent Cells

Stephanie Halene, MD
Yale Univ. School of Medicine
New Haven, CT
METTL3 Mediated m6A RNA Modification Prevents Aberrant Innate Immune Signaling in Fetal Hematopoietic Stem Cells

Paul S. Frenette, MD
Albert Einstein College of Medicine
New York, NY
Macrophage Vetting in the Bone Marrow

Michael D. Milsom, PhD
HI-STEM Ggmbh
Heidelberg, Germany
Identification of Molecular Marks of Lineage Commitment via Comprehensive DNA Methylome Analysis

Moderator:

Ulrich G. Steidl, MD,PhD
Albert Einstein College of Medicine
Bronx, NY

Speakers:

Louise E. Purton, PhD
St. Vincent's Institute of Medical Research
Fitzroy, Australia
Key Roles of Hoxa1 in Myelodysplastic Syndromes

Eirini P. Papapetrou, MD, PhD
Icahn School of Medicine at Mount Sinai
New York, NY
Sequentially CRISPR Gene-Edited Human induced Pluripotent Stem Cells Chart the Clonal Evolution of Myeloid Leukemia

Sridhar Rao, MD,PhD
Versiti
Milwaukee, WI
Genetic Interaction between Cohesin and Driver Mutations in Acute Myeloid Leukemia

Yun Huang, PhD
Institute of Biosciences and Technology, Texas A&M University
Houston, TX
A Combination Strategy Targeting Enhancer Plasticity Exerts Synergistic Lethality Against BETi-Resistant Leukemia Cells

Moderator:

Nancy Speck, PhD
University of Pennsylvania
Philadelphia, PA

Speakers:

Britta Will, PhD
Albert Einstein College of Medicine
New York, NY
Iron Homeostasis-Regulatory Pathways Reinforce Hematopoietic Stem Cell Identity

Megan E. McNerney, MD, PhD
University of Chicago
Chicago, IL
CUX1 Deficiency Potentiates Ras Signaling to Drive High-Risk Myeloid Malignancy

Jing Fang, MD, PhD
University of South Carolina College of Pharmacy
Columbia, SC
Targeting the Multifunctional GPR68 Signaling Plexus in Acute Myeloid Leukemia

Owen J. Tamplin, PhD
University of Illinois College of Medicine
Chicago, IL
The Neurotransmitter Receptor Gabbr1 Regulates Proliferation and Function of Hematopoietic Stem and Progenitor Cells

Julia E. Maxson
Oregon Health & Science University
Portland, OR
Myeloid-Lineage Enhancers Drive Oncogene Synergy and Represent a Novel Therapeutic Target in CSF3R-CEBPA Mutant Acute Myeloid Leukemia

Moderator:

Benjamin Ebert, MD
Dana-Farber Cancer Institute, Harvard Medical School
Boston, MA

Speakers:

Jennifer Trowbridge, PhD
The Jackson Laboratory
Bar Harbor, ME
Age and Cell Context-Dependence of DNMT3a Mutations Driving Clonal Hematopoiesis

Omar Abdel-Wahab, MD
Memorial Sloan-Kettering Cancer Center
New York, NY
Spliceosomal Disruption of the Non-Canonical SWI/SNF Chromatin Remodeling Complex in SF3B1 Mutant Leukemias

R. Coleman Lindsley, MD, PhD
Dana-Farber Cancer Institute
Boston, MA
BCOR/PRC1.1 Inactivation in Myeloid Leukemogenesis

Ross L. Levine, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Mapping Clonal Evolution in Myeloid Malignancies

Speakers:

Asif Rizwan, PhD
National Heart, Lung, and Blood Institute
Bethesda, MD
National Heart, Lung and Blood Institute - Update

Cindy N. Roy
National Institute of Diabetes, Digestive, and Kidney Disease
Bethesda, MD
National Institute of Diabetes and Digestive and Kidney Diseases - Update

Co-Chairs:

Ross L. Levine, MD
Memorial Sloan Kettering Cancer Center
New York, NY

Patricia Ernst, PhD
University of Colorado Denver
Aurora, CO

Introduction to workshop, overview of current state

Co-Chairs:

Alexis A. Thompson, MD
Northwestern University
Chicago, IL

Punam Malik, MD
Cincinnati Children's Hosp.
Cincinnati, OH

This session will focus on the transduction of human hematopoietic stem cells using lentiviral vectors and methods to overcome barriers to efficiency.

Moderators:

Donald B. Kohn, MD
University of California - Los Angeles
Los Angeles, CA

Punam Malik, MD
Cincinnati Children's Hosp.
Cincinnati, OH

Speakers:

Harry L. Malech, MD
National Institute of Health
Bethesda, MD
Introduction to CRISPR/Cas9 Gene Editing of Human CD34+ Hematopoietic Stem Cells: Some Issues Affecting Culture Conditions for Optimization of Genetic Correction

Matthew Porteus, MD, PhD
Stanford University School of Medicine
Stanford, CA
Advances in Cell Manufacturing and Challenges of In Vivo Pre-Clinical Models

Moderators:

Matthew Porteus, MD, PhD
Stanford University School of Medicine
Stanford, CA

Mitchell J. Weiss, MD, PhD
St. Jude Children's Research Hospital
Memphis, TN

Speakers:

Daniel E. Bauer, MD, PhD
Boston Children’s Hospital, Harvard Medical School
Boston, MA
Opportunities and Challenges for Clinical Translation of HbF Induction Gene Editing

Mitchell J. Weiss, MD, PhD
St. Jude Children's Research Hospital
Memphis, TN
Targets and Technologies for HbF Induction

Moderators:

Daniel E. Bauer, MD, PhD
Boston Children's Hospital / Dana-Farber Cancer Institute
Cambridge, MA

Harry L. Malech, MD
National Institute of Health
Bethesda, MD

Speakers:

Elizabeth Benitez, MD
University of California, Los Angeles
Los Angeles, CA
Temporal Control of Cas9 Nuclease Activity to Improve Editing Outcomes in Hematopoietic Stem Cells

Punam Malik, MD
Cincinnati Children's Hosp.
Cincinnati, OH
Novel Approaches that Skew DNA Repair Towards Precise correction

Moderator:

John F. Tisdale, MD
National Institutes of Health
Bethesda, MD

Speakers:

John Pierciey
bluebird bio, Inc.
Cambridge, MA
Considerations for Developing a Manufacturing Process for Gene Modified Hematopoietic Stem and Progenitor Cells

Donald B. Kohn, MD
University of California - Los Angeles
Los Angeles, CA
Challenges of Transducing Human Hematopoietic Stem and Progenitor Cells Using Globin Lentiviral Vectors

Moderator:

John F. Tisdale, MD
National Institutes of Health
Bethesda, MD

Speakers:

Patricia A Shi, MD
New York Blood Center
New York, NY
Current Questions With Plerixafor-Based Mobilization and Collection

John F. Tisdale, MD
National Institutes of Health
Bethesda, MD
Optimization of Cell Harvesting for Genetic Strategies Targeting Hematopoietic Stem Cells in Sickle Cell Disease

Moderator:

Alexis A. Thompson, MD
Northwestern University
Chicago, IL

Speaker:

Wendy Pang, MD, PhD
Stanford University
Stanford, CA
Reducing the Toxicity of Hematopoietic Cell Transplantation Conditioning

Moderators:

Alexis A. Thompson, MD
Northwestern University
Chicago, IL

Punam Malik, MD
Cincinnati Children's Hosp.
Cincinnati, OH

Speakers:

Alexis A. Thompson, MD
Northwestern University
Chicago, IL
How to Define the Curative Threshold: Is a Hemoglobin Electrophoresis Enough?

Punam Malik, MD
Cincinnati Children's Hosp.
Cincinnati, OH
How to Define the Curative Threshold: Is a Hemoglobin Electrophoresis Enough?

Moderators:

Mark C. Walters, MD
Children's Hospital and Res. Ctr. - Oakland
Oakland, CA

Charles S. Abrams, MD
University of Pennsylvania
Philadelphia, PA

Speakers:

Mark C. Walters, MD
Children's Hospital and Res. Ctr. - Oakland
Oakland, CA
Summary of the ASH FDA Clinical Endpoints Workshop - Curative Therapies Panel

Peter W. Marks, MD, PhD
FDA
Washington, DC
Some Regulatory Considerations for Gene Therapy Development

Edward J. Benz Jr., MD
Dana-Farber Cancer Institute
Boston, MA
Cure Sickle Cell Initiative

Keith Hoots, MD
National Heart, Lung, and Blood Institute
Bethesda, MD
National Heart, Lung, and Blood Institute: Sickle Cell Portfolio

Charles S. Abrams, MD
University of Pennsylvania
Philadelphia, PA
American Society of Hematology: Sickle Cell Clinical Trials Network

Co-Chairs:

Alexis A. Thompson, MD
Northwestern University
Chicago, IL

Punam Malik, MD
Cincinnati Children's Hosp.
Cincinnati, OH

Co-Chairs:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN

Ronald Levy, MD
Stanford Univ. School of Medicine
Stanford, CA

Moderators:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN

Ronald Levy, MD
Stanford Univ. School of Medicine
Stanford, CA

Speakers:

Anne J. Novak, PhD
Mayo Clinic
Rochester, MN
Do Genetic Subtypes in Lymphoma Impact the Tumor Microenvironment?

Jonathan W. Friedberg, MD
University of Rochester
Rochester, NY
Can We Use Genetic Subgroups of Lymphoma in the Design of Clinical Trials?

Moderators:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN

Ronald Levy, MD
Stanford Univ. School of Medicine
Stanford, CA

Speakers:

Matteo Maria Bellone, MD
IRCCS Ospedale San Raffaele
Milan, MI, Italy
Do Intestinal Microbes Affect Multiple Myeloma Progression?

Jonathan U. Peled, MD
MSKCC
New York, NY
The Microbiome As an Outcome Predictor of Bone Marrow Transplant

Moderators:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN

Ronald Levy, MD
Stanford Univ. School of Medicine
Stanford, CA

Speakers:

Shannon L. Maude, MD, PhD
Children's Hospital of Philadelphia
Philadelphia, PA
Combining Immune Checkpoint Blockade with Chimeric Antigen Receptor T-Cells

Craig S. Sauter, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Stem Cell Transplant and Chimeric Antigen Receptor T-Cells - Before, After, or Instead Of.

Co-Chairs:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN

Ronald Levy, MD
Stanford Univ. School of Medicine
Stanford, CA