2015 BioMedTracker Pre-ASH Report

2015 Pre-ASH Report
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2015
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BioMedTracker
Pre-ASH Report
American Society of Hematology Annual Meeting
Orlando, FL | December 5-8, 2015

2015 Pre-ASH Report
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Summary
The 57th Annual Meeting of the American Society of Hematology (ASH) will be held in Orlando, FL from
December 5-8, 2015. We have assembled a pre-meeting listing of highlights based on presentation titles
and abstracts that were released. We have also included a full, easy-to-read meeting planner detailing
the presentations and posters scheduled for each day of the ASH meeting.
Conference Highlights
 Profiled themes in the 2015 ASH Agenda include: Genomic Profiling and Chemical Biology,
Genome Editing and Gene Therapy, Epigenetic Mechanisms, Immunologic Treatments, Stem Cell
Biology and Regenerative Medicine and Preventing Venous Thromboembolic Disease
 Venetoclax for CLL (ABBV). Results will be presented for the pivotal Phase II M13-982 trial of
venetoclax monotherapy in patients with relapsed/refractory chronic lymphocytic leukemia
(CLL) with a 17p deletion (#LBA-6). Venetoclax, a BCL-2 inhibitor, was granted Breakthrough
Therapy Designation by the FDA in May 2015.
 KTE-C19 for NHL (KITE). Top-line results will be presented from the Phase I portion of the ZUMA-
1 trial with KTE-C19 in patients with refractory, aggressive non-Hodgkin’s lymphoma (NHL)
including DLBCL, PMBCL and TFL (#3991). The CAR-T therapy KTE-C19 was first granted Orphan
Drug designation for treatment of DLBCL in Mar 2014 by the FDA.
 Zydelig for CLL (GILD). Top-line results will be presented from the Phase III 115 trial of Zydelig
(idelalisib) plus bendamustine and rituximab (BR) in patients with refractory/relapsed CLL. An
interim analysis demonstrated that idelalisib, a PI3K delta inhibitor, in combination with BR was
superior to BR alone for PFS and OS, key primary and secondary endpoints (#LBA-5).
 Ninlaro for Multiple Myeloma (Takeda). Data from the Phase III TOURMALINE-MM1 trial of the
once-weekly oral proteasome inhibitor Ninlaro (ixazomib) in combination with
Revlimid (lenalidomide) and dexamethasone in relapsed/refractory (R/R) multiple myeloma
(MM) will be presented (#727). Takeda announced that this trial met its primary endpoint of
improving PFS in February 2015 and data from the trial have recently supported the drug’s
approval for treatment of R/R MM. This is the first presentation of Phase III data for Ninlaro in
MM, and should give some idea as to whether Ninlaro will be able to compete with the
approved proteasome inhibitors Velcade (bortezomib) and Kyprolis (carfilzomib).
 Keytruda for CLL (MRK). Merck & Co. will showcase the first clinical trial data for a programmed
death-1 receptor (PD-1) inhibitor in CLL (#834). In a Phase II trial (MC1485), Keytruda
(permbrolizumab) was well-tolerated and showed promising efficacy in heavily pre-treated R/R
CLL patients, including patients with Richter’s Transformation.
 Imbruvica for CLL (ABBV/JNJ). The results of the Phase III RESONATE-2 trial of Imbruvica
(ibrutinib) monotherapy versus chlorambucil monotherpay in previously untreated elderly (≥65)
CLL patients will be presented (#495). Imbruvica is already approved for treatment of R/R and

2015 Pre-ASH Report
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high-risk (17p-deletion) CLL, and an sNDA was filed in September 2015 to expand its use to first-
line therapy based on RESONATE-2 data. AbbVie announced in June that the RESONATE-2 trial
achieved its primary endpoint and multiple secondary endpoints, and now we are seeing for the
first time strong response rates, PFS, and OS data that will justify the drug’s approval and
provide a valuable first-line option for elderly CLL patients.
 CTL019 for ALL (NVS/ University of Pennsylvania). On Monday afternoon, Novartis/University
of Pennsylvania will report on an interesting and potentially important development to their
CAR-T program in (#683). The first data from a Phase I study of humanized CD-19-directed CAR T
cells (dubbed CTL119) in pediatric ALL patients who previously received the standard murine-
based CTL019 will be presented, and theories relating to immune-mediated rejection of murine-
based CTL019 and how to overcome this problem to improve remission rats will be discussed.
 First-in-class clinical results include: AG-120, a mutant IDH1 inhibitor (#1306), cirmtuzumab, an
anti-ROR1 monoclonal antibody (#1736), Selinexor, an XPO1 inhibitor (#258), CB-839, a
glutaminase inhibitor (#2566), AG-221, a mutant IDH2 inhibitor (#2509), vosaroxin, a quinolone-
derived DNA intercalator and topoisomerase II inhibitor (#461), sotatercept, an activin type IIA
receptor fusion protein (#4241) and ricolinostat, a HDAC6 selective inhibitor (#3040).

Session Name: Friday Scientific Workshop on Hematology and Aging
Session Location: Tangerine 2 (WF2) (Orange County Convention Center)
Session Time: 1:00pm-6:00pm
Start Time End Time Presentation Title Company Drug Presenter Presentation ID
1:00 PM 6:00 PM Inherited Bmf Syndromes Presenting in Older Adults Jane Churpek
1:00 PM 6:00 PM Telomere Dysfunction and Senescence in Marrow Failure Syndrome Rodrigo Calado
1:00 PM 6:00 PM Erythroid Progenitor Cell Differentiation in Normal Elderly and in Unexplained Anemia Wendy Pang
1:00 PM 6:00 PM Cytopenias and Clonal Hematopoiesis of Indeterminate Potential Rafael Bejar
1:00 PM 6:00 PM Nutritional Impairment in Hematologic Malignancies, Impact on Outcomes Reinhard Stauder
1:00 PM 6:00 PM Markers of Frailty and Survival in MDS Rena Buckstein
1:00 PM 6:00 PM Use of Geriatric Assessment to Predict Toxicities and Survival in Multiple Myeloma Sara Bringhen
1:00 PM 6:00 PM Biologic Correlates of Frailty in Stem Cell Transplantation Andrew Artz
1:00 PM 6:00 PM BTK Inhibitors in Older Adults with CLL- Resistance Patterns and Correlation with Toxicity Jennifer Woyach
1:00 PM 6:00 PM Demethylation to Reverse Age-Related Stem Cell Senescence Nadim Mahmud
1:00 PM 6:00 PM Monoclonal Gammopathy in Older Adults Irene Ghobrial
1:00 PM 6:00 PM Cellular Senescence and Aging Ashley Rosko
Session Name: Friday Scientific Workshop on Myeloid Development
1:00 PM 6:00 PM
A Novel MLL-AF4 In Vivo Model Phenocopies t(4;11) Pro-B ALL and Reveals a Lymphoid Lineage
Bias of the Fusion Protein in Human Cells Michael Thirman
1:00 PM 6:00 PM Rpl22 Deficiency Predisposes Hematopoietic Stem and Progenitor Cells to Leukemogenesis David Wiest
1:00 PM 6:00 PM LNK(SH2B3) Signaling in Normal and Malignant B-Progenitors Wei Tong
1:00 PM 6:00 PM
Interleukin-1 Drives Precocious Myeloid Differentiation of Hematopoietic Stem Cells at the
Expense of Self-Renewal Eric Pietras
1:00 PM 6:00 PM Functional Proteomics of Acute Myeloid Leukemia Alex Kentsis
1:00 PM 6:00 PM LMO2 Regulates DNA Replication in Hematopoietic Cells Trang Hoang
1:00 PM 6:00 PM
AURKA Restricts Megakaryocytic Differentiation By Phosphorylation of NFE2: Implications for MPN
Therapies John Crispino
1:00 PM 6:00 PM
High Mobility Group A1 Chromatin Remodeling Protein Drives Self-Renewal, Niche Formation, and
Regenerative Function in Adult Stem Cells through Wnt/β-Catenin Signaling and SOX9 Linda Resar
1:00 PM 6:00 PM The LRF/ZBTB7A Transcription Factor Regulates BCL11A-Independent Globin Switching Takahiro Maeda
1:00 PM 6:00 PM Beyond FLT3: Recurrent Mutations in CCND3 Confer Resistance to FLT3 Inhibition in AML Catherine Smith
1:00 PM 6:00 PM Integrative Genome-Wide Analysis of RNA Binding and Splicing By Mutant SRSF2 Stephanie Halene
1:00 PM 6:00 PM HIF-1α Signaling Is a Central Pathobiologic Mediator of Myelodysplastic Syndromes Gang Huang
1:00 PM 6:00 PM Cytokine-Mediated Differentiation Reflects Genome-Wide Loss of a Ustat Transcriptional Program Anthony Green
1:00 PM 6:00 PM Regulation of the MLL Complex and H3K4me3 By Son and Its Isoforms Eun-Young Erin Ahn
1:00 PM 6:00 PM Monocyte Lineages in the Bone Marrow Frederic Geissmann
1:00 PM 6:00 PM Modeling Leukemia Progression with Patient-Derived iPSCs Eirini Papapetrou
1:00 PM 6:00 PM Regulation of Neutrophil Lifespan and NET Release By Mlkl Ben Croker
1:00 PM 6:00 PM Serotonin and HSC Production Trista North
1:00 PM 6:00 PM Notch Regulation of Enhancer Function Determines Drug Resistance in Leukemia Warren Pear
1:00 PM 6:00 PM
Inflammation-Induced Emergency Megakaryopoiesis Driven By Hematopoietic Stem Cell-like
Megakaryocyte Progenitors Marieke Essers
1:00 PM 6:00 PM Hematopoietic Stem Cell Loss after Chronic Infection Katherine King
1:00 PM 6:00 PM
Thrombopoietin but Not G-CSF Receptor Stimulation Induces Self-Renewing Hematopoietic Stem
Cell Divisions In Vivo Markus Manz
1:00 PM 6:00 PM Pulse-Mediated Circumferential Stretch Stimulates Hematopoietic Stem Cell Formation Dhvanit Shah
1:00 PM 6:00 PM Loss of Crebbp Confers Pre-Lymphoma/Lymphoma Stem Cell Properties on Lymphoid Progenitor Brian Huntly
1:00 PM 6:00 PM
Polycomb Repressive Complex 1 Enhances HSC Self-Renewal through Repressing the Canonical
Wnt Signaling Pathway Yan Liu
Session Name: American Board of Internal Medicine Maintenance of Certification Learning Session
Session Location: Regency Ballroom O-P (Hyatt Regency Orlando)
2:00 PM 5:00 PM Coagulation/Platelet/Hemostasis Alice Ma
2:00 PM 5:00 PM Leukemia/Stem Cell Transplant Christian Cable
2:00 PM 5:00 PM Hematologic Malignancy (Myeloma, Lymphoma, CLL) James W. Lynch
2015 BioMedTracker ASH Planner
Friday December 4- Oral Presentations

Session Name: 311. Disorders of Platelet Number or Function: Immune Thrombocytopenia and Platelet Survival
Session Location: W315 (Orange County Convention Center)
Session Time: 7:30 AM-9:00 AM
7:30 AM 9:00 AM
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Safety and
Efficacy of Romiplostim in Children with Immune Thrombocytopenia (ITP) Amgen Nplate Michael Tarantino 7
7:30 AM 9:00 AM
Splenic and Circulating Human T Follicular Helper Cell Regulation By B Cell Depleting Therapy
during Immune Thrombocytopenia Sylvain Audia 8
7:30 AM 9:00 AM
Serious and Non-Serious Infections in Non-Splenectomized Adults Treated for Persistent or Chronic
Primary Immune Thrombocytopenia: Effect of Treatments and Protection of Vaccines. Results of
the French Nationwide Faith Study Guillaume Moulis 9
7:30 AM 9:00 AM
A Humanized Anti FcÎ³riia Scfv Inhibits Platelet Activation, Aggregation and Thrombus Formation in
Heparin-Induced Thrombocytopenia (HIT) Beng Chong 10
7:30 AM 9:00 AM Regulation of Platelet Lifespan in the Presence and Absence of TPO Signaling Emma Josefsson 11
7:30 AM 9:00 AM
Platelet GPIba Is Important for Thrombopoietin Production and Thrombopoietin-Induced Platelet
Generation Miao Xu 12
Session Name: 631. Chronic Myeloid Leukemia: Biology and Pathophysiology, excluding Therapy: CML – Mechanisms of Disease Progression and Leukemic Stem Cell Persistence
7:30 AM 9:00 AM
Single Cell Whole Transcriptome Analysis Reveals Distinct Molecular Signatures of Therapy-
Resistant Chronic Myeloid Leukemia Stem Cells Alice Giustacchini 13
7:30 AM 9:00 AM
MS4A3 Improves Imatinib Response and Survival in BCR-ABL1 Primary TKI Resistance and in Blastic
Transformation of Chronic Myeloid Leukemia Anna Eiring 14
7:30 AM 9:00 AM
A Novel TNF-Alpha Antibody Based Therapeutic Approach to Target Leukemic Stem Cells in Bcr-Abl
Disease Mirle Schemionek 15
7:30 AM 9:00 AM
Beclin-1 Phosphorylation By BCR-ABL Is Crucial for CML Leukemogenesis By Suppression of
Autophagy Chuanjiang Yu 16
7:30 AM 9:00 AM
Identification of TKI-Sensitive Point Mutations That Activate c-ABL Kinase Activity and
Transformation Potential and Confer in Vitro Resistance to the Allosteric ABL Inhibitor GNF-5 Bianca Lee 17
7:30 AM 9:00 AM
Chronic Myeloid Leukemia Patients with Deep Molecular Responses to Tyrosine Kinase Inhibitors
Have Increased Effector Natural Killer and Cytotoxic T Cell Immune Responses to Leukaemia-
Associated Antigens and Concomitant Reduced Immune Suppressors Amy Hughes 18
Session Name: 651. Myeloma: Biology and Pathophysiology, excluding Therapy: Revealing Subclonal Heterogeneity in Multiple Myeloma
7:30 AM 9:00 AM
Prognostic Value of Antigen Expression in Multiple Myeloma (MM): A Large GEM/Pethema Study
Based in Four Consecutive Clinical Trials Bruno Paiva 19
7:30 AM 9:00 AM
High Risk Multiple Myeloma Demonstrates Marked Spatial Genomic Heterogeneity Between Focal
Lesions and Random Bone Marrow; Implications for Targeted Therapy and Treatment Resistance Niels Weinhold 20
7:30 AM 9:00 AM
Deep Igh Sequencing Identifies an Ongoing Somatic Hypermutation Process with Complex and
Evolving Clonal Architecture in Myeloma Nikhil Munshi 21
7:30 AM 9:00 AM
Humanized Mouse Model of Myeloma Reveals Clinically Occult Genomic Changes in Primary
Tumor Cells Rakesh Verma 22
7:30 AM 9:00 AM Mutational Profile and Prognostic Relevance of Circulating Tumor Cells in Multiple Myeloma Bruno Paiva 23
7:30 AM 9:00 AM
An Amplicon-Targeted Ultra-Deep Sequencing Approach Reveals the Presence at the Onset of
Multiple Myeloma and the Selection over Time of TP53 Sub-Clonal Variants, Which Adversely
Influence Patients' Overall Survival Carolina Terragna 24
Session Name: 653. Myeloma: Therapy, excluding Transplantation: Advances in Newly Diagnosed and Relapsed Myeloma
7:30 AM 9:00 AM
Bortezomib, Lenalidomide and Dexamethasone Vs. Lenalidomide and Dexamethasone in Patients
(Pts) with Previously Untreated Multiple Myeloma without an Intent for Immediate Autologous
Stem Cell Transplant (ASCT): Results of the Randomized Phase III Trial SWOG S0777 Celgene Revlimid Brian Durie 25
7:30 AM 9:00 AM
Randomized Phase 2 Study of the All-Oral Combination of Investigational Proteasome Inhibitor (PI)
Ixazomib Plus Cyclophosphamide and Low-Dose Dexamethasone (ICd) in Patients (Pts) with Newly
Diagnosed Multiple Myeloma (NDMM) Who Are Transplant-Ineligible (NCT02046070) Takeda Ninlaro Meletios Dimopoulos 26
7:30 AM 9:00 AM
Bortezomib Induction and Maintenance in Patients with Newly Diagnosed Multiple Myeloma: Long-
Term Follow-up of the HOVON-65/GMMG-HD4 Trial Takeda Velcade Pieter Sonneveld 27
7:30 AM 9:00 AM
Eloquent-2 Update: A Phase 3, Randomized, Open-Label Study of Elotuzumab in Combination with
Lenalidomide/Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma - 3-Year
Safety and Efficacy Follow-up Bristol-Myers Squibb Elotuzumab Meletios Dimopoulos 28
7:30 AM 9:00 AM
Clinical Efficacy of Daratumumab Monotherapy in Patients with Heavily Pretreated Relapsed or
Refractory Multiple Myeloma Johnson & Johnson Darzalex Saad Usmani 29
7:30 AM 9:00 AM
Efficacy and Safety of Carfilzomib and Dexamethasone Vs Bortezomib and Dexamethasone in
Patients with Relapsed Multiple Myeloma Based on Cytogenetic Risk Status: Subgroup Analysis
from the Phase 3 Study Endeavor (NCT01568866) Amgen Kyprolis Wee-Joo Chng 30
Session Name: 701. Experimental Transplantation: Basic Biology, Engraftment, and Disease Activity
Session Location: W414AB (Orange County Convention Center)
7:30 AM 9:00 AM Oncostatin M Enhances Hematopoietic Stem Cell Homing and Engraftment Hannah Rasmussen 31
7:30 AM 9:00 AM Immunotoxin Enables Non-Genotoxic Conditioning for Hematopoietic Stem Cell Transplantation Rahul Palchaudhuri 32
7:30 AM 9:00 AM
EPCR/PAR1 Signaling Navigates Long-Term Repopulating Hematopoietic Stem Cell Bone Marrow
Homing to Thrombomodulin-Enriched Blood Vessels Shiri Gur Cohen 33
7:30 AM 9:00 AM
Hematopoeitic Stem Cells and Their Progenitors Critically Require Autophagy to Promote Early
Engraftment Following Allogeneic Stem Cell Transplantation Katie Lineburg 34
7:30 AM 9:00 AM
The Use of Eicosanoids to Enhance Donor Cell Engraftment after in Utero Hematopoietic Cell
Transplantation (IUHCT) Matthew Boelig 35
7:30 AM 9:00 AM Ciclopirox Ethanolamine Is a Novel Modifier of Human Hematopoietic Stem Cell Ex Vivo Expansion
Mehrnaz Safaee
Talkhoncheh 36
Saturday December 5- Oral Presentations

Session Name: 901. Health Services and Outcomes Research – Non-Malignant Conditions: Health Outcomes in Inherited and Acquired Bleeding Disorders
7:30 AM 9:00 AM
Effect of Romiplostim on Health-Related Quality of Life in Children with Immune
Thrombocytopenia and Associated Burden in Their Parents: Results from a Phase 3, Randomized,
Double-Blind, Placebo-Controlled Study Amgen Nplate Susan Mathias 37
7:30 AM 9:00 AM
Rate of Bleeding-Rated Episodes (BREs) in Elderly Patients with Primary Immune
Thrombocytopenia (ITP): A Population-Based Retrospective Cohort Study Using Medicare 20%
Sample Data Shuling Li 38
7:30 AM 9:00 AM
Impact of Pain and Functional Impairment in US Adult People with Hemophilia (PWH): Patient-
Reported Outcomes and Musculoskeletal Evaluation in the Pain, Functional Impairment, and
Quality of Life (P-FiQ) Study Christine Kempton 39
7:30 AM 9:00 AM Numeracy in Patients with Hemophilia Duc Tran 40
7:30 AM 9:00 AM Outcomes of Referrals for Mild and Moderate Thrombocytopenia Charles Schlappi 41
7:30 AM 9:00 AM Content, Utilization and Impact of a Hematology e-Consultation Service Adam Fogel 42
Session Name: Advances in the Understanding and Diagnosis of Myelodysplasia and Bone Marrow Failure Syndromes
Session Location: Valencia D (W415D) (Orange County Convention Center)
7:30 AM 9:00 AM Genetics in Inherited Bone Marrow Failure Disorders Sioban Keel SCI-1
7:30 AM 9:00 AM Megakaryocytic Fate Specification and Maturation Diane Krause SCI-2
7:30 AM 9:00 AM Clonal Diversity and Mutated Genes in Myelodysplastic Syndromes Benjamin Ebert SCI-3
Session Name: Challenging Scenarios in Thrombosis
7:30 AM 9:00 AM The Antiphospholipid Syndrome: Still an Enigma Keith McCrae
7:30 AM 9:00 AM Perinatal Thrombosis: Implications for Mother and Neonate Sarah O'Brien
7:30 AM 9:00 AM Mechanical Circulatory Support: Balancing Bleeding and Clotting in High Risk Patients Lisa Baumann Kreuziger
Session Name: Clonal Architecture and Hierarchy: Implications for Biology and Translation
7:30 AM 9:00 AM Clonal Events in Normal Aging Hematopoiesis Lambert Busque SCI-10
7:30 AM 9:00 AM Clonal Evolution of Pre-Leukemic Hematopoietic Stem Cells in Human Acute Myeloid Leukemia Ravi Majeti SCI-11
7:30 AM 9:00 AM Clonal Mutations and Clonal Hierarchy in Pediatric Myeloid Neoplasms Charlotte Niemeyer SCI-12
Session Name: Determinants of Clot Structure: Beyond Fibrin
7:30 AM 9:00 AM Platelet Activation Gradients During Thrombus Formation Timothy Stalker SCI-13
7:30 AM 9:00 AM Factor XIII and Red Blood Cells in Venous Thrombosis Alisa Wolberg SCI-14
7:30 AM 9:00 AM Contribution of Red Blood Cells and Clot Structure to Thrombosis Robert Ariens SCI-15
Session Name: Genetics vs. Response: What Should Direct Future Therapy of Childhood Acute Lymphoblastic Leukemia?
Session Location: Tangerine 3 (WF3-4) (Orange County Convention Center)
7:30 AM 9:00 AM
Disease Response Guiding Therapy in Acute Lymphocytic Leukemia: Pivotal Role of Minimal
Residual Disease Michael Pulsipher SCI-34
7:30 AM 9:00 AM Genetics Directing Therapy in Acute Lymphocytic Leukemia: Identifying Risk, Defining Targets Andrea Biondi SCI-35
7:30 AM 9:00 AM Epigenetics Directing Therapy in Acute Lymphocytic Leukemia: Emerging Roles and New Therapies Kathrin Bernt SCI-36
Session Name: Harnessing T Cells in Therapy of Hematologic Malignancy
7:30 AM 9:00 AM Choice of Better T-cells for Adoptive Immunotherapy Dirk Busch SCI-22
7:30 AM 9:00 AM From T-cell Engineering to CAR therapy: Progress and Prospects Michel Sadelain SCI-23
7:30 AM 9:00 AM Latest in Clinical Application of CAR Cell Therapy for B-cell Malignancy and Transplantation Crystal Mackall SCI-24
Session Name: Management of Challenging Bleeding Complications
Session Location: Chapin Theater (W320) (Orange County Convention Center)
7:30 AM 9:00 AM Bleeding Complications in Immune Thrombocytopenia Donald Arnold
7:30 AM 9:00 AM Acquired Bleeding Disorders in the Elderly Rebecca Kruse-Jarres
7:30 AM 9:00 AM Coagulopathy of Liver Disease Jody Kujovich
Session Name: Myeloproliferative Neoplasms: A Clinical Update
7:30 AM 9:00 AM Management of Symptoms in Polycythemia Vera and Essential Thrombocythemia Deepti Radia
7:30 AM 9:00 AM Looking Forward: Novel Therapeutic Approaches in Chronic and Advanced Phases of Myelofibrosis John Mascarenhas
7:30 AM 9:00 AM Myeloproliferative/Myelodysplastic Overlap Syndromes Eric Padron
Session Name: Take a Breath: Heme, Iron, and Cellular Metabolism
7:30 AM 9:00 AM The Role of Hepcidin in Health And Disease Martina Muckenthaler SCI-43
7:30 AM 9:00 AM Iron Requirement in Hypoxic Conditions Max Gassmann SCI-44
7:30 AM 9:00 AM Special Delivery: The Role of Iron Chaperones in the Distribution of Iron in Developing Red Cells Caroline Philpott SCI-45

Session Name: Targeting the Epigenome
Session Location: Hall E1 (Orange County Convention Center)
7:30 AM 9:00 AM Systematic Approaches to Identify Actionable Epigenetic Vulnerabilities in Cancer Christopher Vakoc
7:30 AM 9:00 AM Hierarchical Epigenetic Modifications and Clonal Evolution Ari Melnick
7:30 AM 9:00 AM Mechanisms of Sensitivity and Resistance to Epigenetic Therapies Mark Dawson
Session Name: Update in Large Cell Lymphoma
Session Location: Hall D (Orange County Convention Center)
7:30 AM 9:00 AM Utilizing the Pathology Report to Inform Treatment Options Jonathan Friedberg
7:30 AM 9:00 AM Understanding the Lymphoma Pathology Report Eric Hsi
7:30 AM 9:00 AM Treatment of the Patient with Transformed Lymphoma Silvia Montoto
Session Name: 503. Hematopoiesis: Epigenetic, Transcriptional and Translational Control: Epigenetic and Transcriptional Networks in Hematopoiesis and Leukemia
9:30 AM 11:00 AM Cistrome Control of Hematopoietic Stem/Progenitor Cell Function Kyle Hewitt 43
9:30 AM 11:00 AM Modular Domains within a Super Enhancer Determine Drug Resistance in Leukemia Yumi Ohtani 44
9:30 AM 11:00 AM
Integrated Analysis of the Human Hematopoietic Non-Coding RNA Landscape Reveals Lnc-RNA
Stem Cell Signature in AML Adrian Schwarzer 45
9:30 AM 11:00 AM
GFI1 Snag Domain Methylation Directs LSD1 Recruitment to Control Transcription and Cell Fate
Determination in Hematopoiesis Matthew Velinder 46
9:30 AM 11:00 AM
Quantitative Proteomic and Transcriptomic Analysis Reveals Post-Transcriptional Regulation of
Mitochondrial Biogenesis during Erythropoiesis Jian Xu 47
9:30 AM 11:00 AM A Hyperactive Transcriptional State Marks Genome Reactivation during Mitotic Exit Gerd Blobel 48
Session Name: 631. Chronic Myeloid Leukemia: Biology and Pathophysiology, excluding Therapy: Targeting Leukemic Stem Cells in Chronic Myeloid Leukemia
9:30 AM 11:00 AM CD93 Is a Novel Biomarker of Leukemia Stem Cells in Chronic Myeloid Leukemia Ross Kinstrie 49
9:30 AM 11:00 AM Targeting of Quiescent and Proliferating CML Stem Cells By DNA Repair Inhibitors Katherine Sullivan 50
9:30 AM 11:00 AM
Knockdown (KD) of Mir-126 Expression Enhances Tyrosine Kinase Inhibitor (TKI)-Mediated
Targeting of Chronic Myelogenous Leukemia (CML) Stem Cells Bin Zhang 51
9:30 AM 11:00 AM
A New AHI-1-DNM2-BCR-ABL Complex Regulates Leukemic Properties of Primitive CML Cells
through Mediation of Cellular Endocytosis and ROS-Induced Autophagy Xiaohu Liu 52
9:30 AM 11:00 AM
Role of the MSC-Derived Exosomal and Endogenous JAK2-SET/PP2A-Beta Catenin-Modulator Mir-
300 in Leukemic Stem/Progenitor Proliferation and Survival in CML Giovannino Silvestri 53
9:30 AM 11:00 AM Inhibition of CML Stem Cell Renewal By the Porcupine Inhibitor WNT974 Puneet Agarwal 54
Session Name: 634. Myeloproliferative Syndromes: Clinical: Early and Late Stage Trials in MPN
9:30 AM 11:00 AM
Telomerase Inhibitor Imetelstat Therapy in Refractory Anemia with Ring Sideroblasts with or
without Thrombocytosis Geron Imetelstat Ayalew Tefferi 55
9:30 AM 11:00 AM PRM-151 in Myelofibrosis: Durable Efficacy and Safety at 72 Weeks Promedior PRM-151 Srdan Verstovsek 56
9:30 AM 11:00 AM Dynamics of Mutations in Patients with ET Treated with Imetelstat Geron Imetelstat
Elisabeth Oppliger
Leibundgut 57
9:30 AM 11:00 AM
Analysis of Outcomes By Patient Subgroups in Patients with Myelofibrosis Treated with Pacritinib
Vs Best Available Therapy (BAT) in the Phase III Persist-1 Trial Pacritinib CTI BioPharma Alessandro Vannucchi 58
9:30 AM 11:00 AM
Long-Term Efficacy and Safety in COMFORT-II, a Phase 3 Study Comparing Ruxolitinib with Best
Available Therapy for the Treatment of Myelofibrosis: 5-Year Final Study Results Incyte Jakafi Claire Harrison 59
9:30 AM 11:00 AM
Efficacy and Safety of Pegylated Interferon Alpha-2a in Patients with Essential Thrombocythemia
and Polycythemia Vera: Results after a Median 7-Year Follow-up of a Phase 2 Study Lucia Masarova 60
Session Name: 732. Clinical Allogeneic Transplantation: Results I
9:30 AM 11:00 AM
Combined Donor and Recipient Non-HLA Genotypes Show Evidence of Genome Wide Association
with Transplant Related Mortality (TRM) after HLA-Matched Unrelated Donor Blood and Marrow
Transplantation (URD-BMT) (DISCOVeRY-BMT study) Lara Sucheston-Campbell 61
9:30 AM 11:00 AM
Stem-Cell Transplantation in Adults with Philadelphia-Negative High-Risk Acute Lymphoblastic
Leukemia in First Complete Remission: A Prospective Multicenter Trial Comparing Haploidentical
Donors with Identical Sibling Donors Yu Wang 62
9:30 AM 11:00 AM
Allogeneic Hematopoietic Stem Cell Transplantation for Secondary Acute Myeloid Leukemia- a
Report from the Acute Leukemia Working Party of the EBMT Bipin Savani 63
9:30 AM 11:00 AM
A Prognostic System Predictive of Outcomes in Persons Undergoing Allogeneic Hematopoietic Cell
Transplantation for Myelodysplastic Syndrome Brian Shaffer 64
9:30 AM 11:00 AM
Alemtuzumab-Cyclosporine Versus Tacrolimus-Methotrexate-Sirolimus for Graft-Versus-Host
Disease Prophylaxis in Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation from
Unrelated Donors: Final Results of a Randomized Trial Lauren Curtis 65
9:30 AM 11:00 AM
Feasibililty of Azacitidine As Bridge to Allogeneic Stem Cell Transplantation in Patients with Higher-
Risk MDS or Low-Blast Count AML: Results of the BMT-AZA Multicenter Prospective Study Maria Teresa Voso 66
Session Name: Aging and Thrombosis
9:30 AM 11:00 AM Animal Models of Aging Research Marc Hoylaerts SCI-4
9:30 AM 11:00 AM Vascular and Endothelial Biology of Aging Frank Faraci SCI-5
9:30 AM 11:00 AM Epidemiology of Thrombosis in Aging Weihong Tang SCI-6
Session Name: Checkpoint, Please?
9:30 AM 11:00 AM Cytotoxic T-lymphocyte-associated Protein 4 and Cancer Therapy James Allison SCI-7

9:30 AM 11:00 AM Role of PD-1/PD-L1 in Acute and Chronic Graft Versus Host Disease Bruce Blazar SCI-8
9:30 AM 11:00 AM Checkpoint Blockade with Neoantigen Cancer Vaccines for Personalized Immunotherapy Catherine Wu SCI-9
Session Name: Genomics, The Next Frontier: Novel Approaches to Study Cancer and Development
9:30 AM 11:00 AM Engineering the Cancer Genome Tyler Jacks SCI-19
9:30 AM 11:00 AM Single Cell RNA seq for Analysis of Cell Fate Decisions H. Leighton Grimes SCI-20
9:30 AM 11:00 AM New Approaches for Mapping Epigenome Dynamics Steven Henikoff SCI-21
Session Name: Great Expectations: Hematology in Pregnancy
9:30 AM 11:00 AM Hemolytic Disease of the Fetus and Newborn: Managing the Mother, Fetus, and Neonate Meghan Delaney
9:30 AM 11:00 AM Postpartum Hemorrhage: A Continuing Challenge Evelyn Lockhart
9:30 AM 11:00 AM Fertility Issues in Patients with Hematologic Malignancies Alison Loren
Session Name: Immunological Ctrl-Alt-Del: Stem Cell Transplantation for Hematological Malignancies
9:30 AM 11:00 AM Immune Reconstitution Following Stem Cell Transplantation Marcel van den Brink
9:30 AM 11:00 AM Use of Alternative Donors for Allogeneic Stem Cell Transplantation Claudio Anasetti
9:30 AM 11:00 AM Graft-Versus-Host Disease vs. Graft-Versus-Leukemia Effect Robert Negrin
Session Name: Managing Typical and Atypical Chronic Myeloid Leukemia
Session Location: Valencia BC (W415BC) (Orange County Convention Center)
9:30 AM 11:00 AM
Molecular Monitoring in Chronic Myeloid Leukemia and the Prospects for Treatment-Free
Remissions Michael Deininger
9:30 AM 11:00 AM Is There a Best Tyrosine Kinase Inhibitor for Chronic-Phase Chronic Myeloid Leukemia? Richard Larson
9:30 AM 11:00 AM What’s Different about Atypical Chronic Myeloid Leukemia and Chronic Neutrophilic Leukemia? Kim-Hien Dao
Session Name: Mechanism of Phosphatidylserine Exposure
9:30 AM 11:00 AM Flippases and Scramblases at Plasma Membranes that Regulate Phosphatidylserine Exposure Shigekazu Nagata SCI-31
9:30 AM 11:00 AM Platelet Phosphatidylserine Exposure, Survival and Blood Coagulation in Mice Lacking TMEM16F Gary Gilbert SCI-32
9:30 AM 11:00 AM Regulation of Platelet Procoagulant Activity Johan Heemskerk SCI-33
Session Name: Myelodysplastic Syndromes: Evolving Diagnosis and Therapy
9:30 AM 11:00 AM
The Shadowlands of Myelodysplastic Syndromes: Idiopathic Cytopenias of Undetermined
Significance and Clonal Hematopoiesis of Indeterminate Potential Luca Malcovati
9:30 AM 11:00 AM Reclassifying Myelodysplastic Syndromes: What's Where in the New WHO and Why Daniel Arber
9:30 AM 11:00 AM
Nine Years Without a New FDA Approved Therapy for Myelodysplastic Syndromes: How Can We
Break Through the Impasse? Amy DeZern
Session Name: New Insights in Hemolytic Anemia
9:30 AM 11:00 AM Complement in Hemolytic Anemia Robert Brodsky
9:30 AM 11:00 AM Management of Hemolytic Anemia Following Allogeneic Stem Cell Transplantation Andreas Holbro
9:30 AM 11:00 AM Diagnosis and Management of Rare Congenital Non-Immune Hemolytic Disease Patrick Gallagher
Session Name: Novel Approaches to Acute Lymphoblastic Leukemia
9:30 AM 11:00 AM Management Of Adult BCR/ABL1+ Acute Lymphocytic Leukemia Robin Foà
9:30 AM 11:00 AM Novel Monoclonal Antibodies in Acute Lymphocytic Leukemia Daniel DeAngelo
9:30 AM 11:00 AM How Can We Improve on the Already Impressive Results in Pediatric Acute Lymphocytic Leukemia? Angela Thomas
Session Name: Optimizing Therapy in Sickle Cell Disease
9:30 AM 11:00 AM Optimizing Hydroxyurea Therapy in Sickle Cell Disease Russell Ware
9:30 AM 11:00 AM The Five Key Things You Need to Know to Manage Adult Patients with Sickle Cell Disease Sophie Lanzkron
9:30 AM 11:00 AM
Can Selectin and Invariant Natural Killer T-Cell Therapies Meet the Needs of People with Sickle Cell
Disease? Joshua Field
Session Name: Pediatric Hematological Malignancies: Beyond Acute Lymphocytic Leukemia
Session Location: Valencia A (W415A) (Orange County Convention Center)
9:30 AM 11:00 AM Hodgkin Lymphoma in Children and Adolescents Kara Kelly
9:30 AM 11:00 AM Acute Myeloid Leukemia in Children and Adolescents E. Kolb
9:30 AM 11:00 AM Non-Hodgkin Lymphoma in Children and Adolescents Rachel Kobos

Session Name: Practical Management of T-Cell and Natural Killer Cell Lymphomas
9:30 AM 11:00 AM Peripheral T-Cell Lymphoma Not Otherwise Specified and Anaplastic Large Cell Lymphoma Anne Beaven
9:30 AM 11:00 AM Choosing a Systemic Therapy for Advanced Mycosis Fungoides and Sezary Syndrome Madeleine Duvic
9:30 AM 11:00 AM Zebras and Hen's Teeth: Recognition and Management of Rare T and NK Lymphomas Steven Horwitz
Session Name: Recent Insights into Efficacy of Red Blood Cells in Transfusions and Physiology
9:30 AM 11:00 AM
New Insights in the Potential Effect of Transfusing Red Blood Cells that Have Been Stored for
Different Periods Simone Glynn SCI-37
9:30 AM 11:00 AM Impact of Genetic Predispositions, Diet, and Environment on Storage of Red Blood Cells Steven Spitalnik SCI-38
9:30 AM 11:00 AM Role of Red Cells in Thrombosis and Hemostasis John Weisel SCI-39
Session Name: Special Education Session on Clinical Applications of Newly Approved Drugs
9:30 AM 11:00 AM Blinatumomab Amgen Blincyto Anjali Advani
9:30 AM 11:00 AM Idarucizumab Boehringer Ingelheim Praxbind Kenneth Bauer
9:30 AM 11:00 AM Panobinostat Novartis Farydak Sagar Lonial
Session Name: Telomeres and Bone Marrow Failure
9:30 AM 11:00 AM Mechanisms Governing Disruption of Telomere Maintenance in Dyskeratosis Congenita Steven Artandi SCI-49
9:30 AM 11:00 AM Therapeutic Strategies for the Treatment of Telomere-Associated Diseases Maria Blasco SCI-50
9:30 AM 11:00 AM Extrahematopoietic Manifestations of Telomere Syndromes Mary Armanios SCI-51
Session Name: The Path Toward Curing Multiple Myeloma
9:30 AM 11:00 AM Characterization of Minimal Residual Disease Alberto Orfao SCI-52
9:30 AM 11:00 AM Clonal Heterogeneity and Genetic Instability of Multiple Myeloma and Impact on Treatment Jonathan Keats SCI-53
9:30 AM 11:00 AM Mathematical Models of Cancer Evolution and Cure Kenneth Shain
Session Name: Career Development Lunch- Room 1
Session Location: Regency Ballroom P (Hyatt Regency Orlando)
Session Time: 11:15 AM-12:15 PM
11:15 AM 12:15 PM Laboratory & Translational Hematology Careers Ross Levine
11:15 AM 12:15 PM Industry Careers Benjamin Kim
11:15 AM 12:15 PM Industry Careers William Hobbs
11:15 AM 12:15 PM Government Careers Ann Farrell
11:15 AM 12:15 PM Government Careers Wyndham Wilson
11:15 AM 12:15 PM Pediatric Hematology/Oncology Careers Mitchell Weiss
11:15 AM 12:15 PM Pediatric Hematology/Oncology Careers William Carroll
11:15 AM 12:15 PM PhD Careers Ellinor Peerschke
11:15 AM 12:15 PM PhD Careers Margaret Goodell
11:15 AM 12:15 PM Clinical Careers in Hematology Alexander Spira
11:15 AM 12:15 PM Medical Educators in Hematology Jason Gotlib
11:15 AM 12:15 PM Medical Educators in Hematology Jennifer Kesselheim
11:15 AM 12:15 PM Transfusion Medicine Careers Gary Schiller
11:15 AM 12:15 PM Laboratory & Translational Hematology Careers Pavan Reddy
11:15 AM 12:15 PM Transfusion Medicine Careers Marc Zumberg
11:15 AM 12:15 PM Clinical Benign Hematology Careers Margaret Ragni
11:15 AM 12:15 PM Clinical Careers in Hematology Robb Friedman
11:15 AM 12:15 PM Clinical Malignant Hem/BMT Careers Richard Stone
11:15 AM 12:15 PM Clinical Benign Hematology Careers Kenneth Bauer
11:15 AM 12:15 PM Clinical Malignant Hem/BMT Careers David Avigan
Session Name: Career Development Lunch- Room 2
Session Location: Regency Ballroom Q (Hyatt Regency Orlando)
11:15 AM 12:15 PM Clinical Malignant Hem/BMT Careers Steven Devine
11:15 AM 12:15 PM Clinical Malignant Hem/BMT Careers John DiPersio
11:15 AM 12:15 PM Clinical Benign Hematology Careers Kenneth Ataga
11:15 AM 12:15 PM Clinical Benign Hematology Careers Gowthami Arepally
11:15 AM 12:15 PM Laboratory & Translational Hematology Careers Robert Flaumenhaft
11:15 AM 12:15 PM Industry Careers Solveig Ericson
11:15 AM 12:15 PM Industry Careers Jeffery Kutok
11:15 AM 12:15 PM Government Careers Nicole Gormley
11:15 AM 12:15 PM Government Careers Louis Staudt
11:15 AM 12:15 PM Pediatric Hematology/Oncology Careers Smita Bhatia
11:15 AM 12:15 PM Pediatric Hematology/Oncology Careers Staci Arnold
11:15 AM 12:15 PM PhD Careers Emmanuelle Passegue
11:15 AM 12:15 PM PhD Careers John Crispino
11:15 AM 12:15 PM Medical Educators in Hematology Leslie Kersun
11:15 AM 12:15 PM Medical Educators in Hematology Alexandra Wolanskyj

11:15 AM 12:15 PM Clinical Careers in Hematology Michael Lankiewicz
11:15 AM 12:15 PM Transfusion Medicine Careers Lawrence Goodnough
11:15 AM 12:15 PM PhD Careers Barbara Furie
11:15 AM 12:15 PM Laboratory & Translational Hematology Careers Catherine Bollard
Session Name: Continuing Conversations with the Speakers on Checkpoint Please?
Session Location: W232A (Orange County Convention Center)
11:15 AM 12:15 PM Speaker Bruce Blazar
11:15 AM 12:15 PM Speaker James Allison
11:15 AM 12:15 PM Speaker Catherine Wu
Session Name: Continuing Conversations with the Speakers on Genomics, the Next Frontier: Novel Approaches to Study Cancer and Development
Session Location: W231C (Orange County Convention Center)
11:15 AM 12:15 PM Speaker H. Leighton Grimes
11:15 AM 12:15 PM Speaker Tyler Jacks
11:15 AM 12:15 PM Speaker Steven Henikoff
Session Name: 114. Hemoglobinopathies, Excluding Thalassemia – Clinical: Novel Insights into Pathophysiology in Sickle Cell Disease
Session Time: 12:00 PM-1:30 PM
12:00 PM 1:30 PM
Thermal Pain and Pain Anticipation Induce a Decrease in Microvascular Perfusion in Sickle Cell and
Normal Subjects Maha Khaleel 67
12:00 PM 1:30 PM Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era Janet Kwiatkowski 68
12:00 PM 1:30 PM
Elevations in MR Measurements of Whole Brain and Regional Cerebral Blood Flow and Oxygen
Extraction Fraction Suggest Cerebral Metabolic Stress in Children with Sickle Cell Disease
Unaffected By Overt Stroke Melanie Fields 69
12:00 PM 1:30 PM
Association of Hemoglobin S and C Traits with Kidney Disease in African Americans in the Reasons
for Geographic and Racial Differences in Stroke (REGARDS) Study Rakhi Naik 70
12:00 PM 1:30 PM Survival in Sickle Cell Disease: Data from a Well-Resourced, National Health System Setting Kate Gardner 71
12:00 PM 1:30 PM
Association Between Hyperhemolysis and Vascular Complications in Sickle Cell Disease Sub-
Saharan African Patients Brigitte Ranque 72
Session Name: 311. Disorders of Platelet Number or Function: Genetics of Inherited and Acquired Thrombocytopenias
12:00 PM 1:30 PM
Genes Influencing the Development and Severity of Chronic ITP Identified through Whole Exome
Sequencing Jenny Despotovic 73
12:00 PM 1:30 PM
Blvrb Mutation Induces Thrombocytosis Via Redox Dysregulation in the Heme Degradation
Pathway Song Wu 74
12:00 PM 1:30 PM
Functional Characterization of a Novel GFI1B Mutation Causing Congenital
Macrothrombocytopenia Katsumasa Kitamura 75
12:00 PM 1:30 PM
A Dominant Negative Mutation (p.P214L) in ETV6 is Associated with Megakaryocyte and Erythroid
Transcript Misregulation Leila Noetzli 76
12:00 PM 1:30 PM New CNV Regions Identified in ITP Provide Evidence for Genetic Predisposition M. Cem Ar 77
12:00 PM 1:30 PM
Homozygous Mutation of the ETS DNA-Binding Domain of FLI1 Causes a Bleeding Disorder with
Giant Alpha Granules Similar to Paris-Trousseau Thrombocytopenia William Stevenson 78
Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies I
12:00 PM 1:30 PM
Effectiveness of High-Dose MTX Therapy for Adult Ph-Negative ALL By Randomized Trial: JALSG
ALL202-O Toru Sakura 79
12:00 PM 1:30 PM
A Multicenter Phase II Study Using a Dose Intensified Pegylated-Asparaginase Pediatric Regimen in
Adults with Untreated Acute Lymphoblastic Leukemia: A DFCI ALL Consortium Trial Daniel DeAngelo 80
12:00 PM 1:30 PM
Multicenter Total Therapy Gimema LAL 1509 Protocol for De Novo Adult Ph+ Acute Lymphoblastic
Leukemia (ALL) Patients. Updated Results and Refined Genetic-Based Prognostic Stratification Sabina Chiaretti 81
12:00 PM 1:30 PM
6-Mercaptopurine (6MP) Intake during Maintenance for Childhood Acute Lymphoblastic Leukemia
(ALL) - a Comparison of Self-Report and Electronic Monitoring: A Report from the Children's
Oncology Group (COG) Study AALL03N1 Wendy Landier 82
12:00 PM 1:30 PM
Frontline Inotuzumab Ozogamicin in Combination with Low-Intensity Chemotherapy (mini-hyper-
CVD) for Older Patients with Acute Lymphoblastic Leukemia (ALL) Elias Jabbour 83
12:00 PM 1:30 PM Characterization of Leukemias with ETV6-ABL1 Fusion Marketa Zaliova 84
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics and Molecular Markers in Diagnosis and Prognosis: Omics-based Approaches for the Identification of Novel Genetic Modalities in AML
12:00 PM 1:30 PM Personally Tailored Risk Prediction of AML Based on Comprehensive Genomic and Clinical Data Moritz Gerstung 85

12:00 PM 1:30 PM
The Complexity of Interpreting Genomic Data in Patients with Primary and Secondary Acute
Myeloid Leukemia (AML) Aziz Nazha 86
12:00 PM 1:30 PM
Discovery and Functional Validation of Novel Pediatric Specific FLT3 Activating Mutations in Acute
Myeloid Leukemia: Results from the COG/NCI Target Initiative Katherine Tarlock 87
12:00 PM 1:30 PM
Genomic and Proteomic Analysis of Primary Chemoresistance and Induction Failure in Acute
Myeloid Leukemia Fiona Brown 88
12:00 PM 1:30 PM
Dysregulation of DNA Methylation Involves in Progression of Myeloid Leukemia in Down
Syndrome Satoshi Saida 89
12:00 PM 1:30 PM
Immunophenotypic-Defined Stage of Leukemia Differentiation Arrest Identifies Oncogenic and
Metabolic Signatures in AML Francois Vergez 90
Session Name: 637. Myelodysplastic Syndromes – Clinical Studies: New Therapeutic Approaches
12:00 PM 1:30 PM
Eltrombopag for the Treatment of Thrombocytopenia of Low and Intermediate-1 IPSS Risk
Myelodysplastic Syndromes: Interim Results on Efficacy, Safety and Quality of Life of an
International, Multicenter Prospective, Randomized, Trial Novartis Promacta Esther Oliva 91
12:00 PM 1:30 PM
Luspatercept Treatment Leads to Long Term Increases in Hemoglobin and Reductions in
Transfusion Burden in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes
(MDS): Preliminary Results from the Phase 2 PACE-MDS Extension Study Acceleron Pharma Luspatercept Aristoteles Giagounidis 92
12:00 PM 1:30 PM
A Phase 1b Study of Birinapant in Combination with 5-Azacitadine in Patients with Myelodysplastic
Syndrome Who Are Naïve, Refractory or Have Relapsed to 5-Azacitadine TetraLogic Birinapant Gautam Borthakur 93
12:00 PM 1:30 PM
Low-Dose Hypomethylating Agents (HMAs) Are Effective in Patients (Pts) with Low- or
Intermediate-1-Risk Myelodysplastic Syndrome (MDS): A Report on Behalf of the MDS Clinical
Research Consortium Nicholas Short 94
12:00 PM 1:30 PM
Outcome of Patients Treated for Myelodysplastic Syndromes after Failure of Lenalidomide
Therapy Thomas Prebet 95
12:00 PM 1:30 PM
Predicting MDS Response to Drug Therapies Based on a New Method of Interpreting the MDS
Mutanome Cindy Medina 96
Session Name: 703. Adoptive Immunotherapy: Clinical Studies
12:00 PM 1:30 PM
Adoptive Transfer of WT1-Specific TCR Gene-Transduced Lymphocytes in Patients with
Myelodysplastic Syndrome and Acute Myeloid Leukemia Isao Tawara 97
12:00 PM 1:30 PM
Wilms´Tumor 1 Protein Is Highly Expressed on Malignant Plasma Cells and Provides a Novel Target
for Immunotherapeutic Approaches Guenther Koehne 98
12:00 PM 1:30 PM
Allogeneic T-Cells Expressing an Anti-CD19 Chimeric Antigen Receptor Cause Remissions of B-Cell
Malignancies after Allogeneic Hematopoietic Stem Cell Transplantation without Causing Graft-
Versus-Host Disease James Kochenderfer 99
12:00 PM 1:30 PM
CD4 CAR T Cells Mediate CD8-like Cytotoxic Anti-Leukemic Effects Resulting in Leukemic Clearance
and Are Less Susceptible to Attenuation By Endogenous TCR Activation Than CD8 CAR T Cells Yinmeng Yang 100
12:00 PM 1:30 PM
Human Cytokine-Induced Memory-like NK Cells Exhibit in Vivo Anti-Leukemia Activity in
Xenografted NSG Mice and in Patients with Acute Myeloid Leukemia (AML) Rizwan Romee 101
12:00 PM 1:30 PM
Phase I Trial of IL-21 Ex Vivo Expanded NK Cells Administration to Prevent Disease Relapse after
Haploidentical Stem-Cell Transplantation for Myeloid Leukemias Stefan Ciurea 102
Session Name: Ham-Wasserman Lecture
12:30 PM 1:30 PM Game of Clones: The Genomic Evolution of Severe Congenital Neutropenia Ivo Touw
Session Name: 311. Disorders of Platelet Number or Function: Thrombotic Microangiopathies
2:00 PM 3:30 PM Modified Ham Test Distinguishes aHUS from TTP and Predicts Response to Eculizumab Eleni Gavriilaki 103
2:00 PM 3:30 PM
Anti-Idiotypic-Designed-Ankyrin-Repeat-Molecules (DARPins) Universally Neutralize Plasma-
Derived Autoantibodies in Acquired TTP Patients Monica Schaller 104
2:00 PM 3:30 PM
Human Neutrophil Peptides (HNPs) Enhance Thrombus Formation Under Flow By Inhibiting
Proteolytic Activity of Plasma ADAMTS13 Metalloprotease Vikram Pillai 105
2:00 PM 3:30 PM Identification of T Cell Epitope of ADAMTS13 in Thrombotic Thrombocytopenic Purpura Patients Laurent Gilardin 106
2:00 PM 3:30 PM
ADAMTS13 Reactive CD4+ T Cells Targeting CUB-2 Domain Derived Peptides in Autoimmune
Thrombotic Thrombocytopenic Purpura Fabian Verbij 107
2:00 PM 3:30 PM
Profiling the Autoantibody Repertoire of Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Patients By Single Cell Sorting and Deep Sequencing of Splenic B-Cells Magdalena Skowronska 108
Session Name: 622. Non-Hodgkin Lymphoma: Biology, excluding Therapy: Refining The Molecular Roadmap In NHL
2:00 PM 3:30 PM
Comprehensive MYC and BCL2 Genetic Profiling in De Novo Diffuse Large B-Cell Lymphoma
Demonstrates Clinically Relevant Genetic Alterations According to Cell of Origin Subtype Daisuke Ennishi 109
2:00 PM 3:30 PM
Discovery of Recurrent Mutations Associated with Chemo-Immunotherapy Relapse in Diffuse
Large B-Cell Lymphoma Jillian Wise 110
2:00 PM 3:30 PM
Genetic Alterations of Gα13 Signaling Pathway with BCL2 over-Expression Confers Lymphoma
Dissemination and Inferior Outcome in Germinal Center B Cell Diffuse Large B Cell Lymphoma Daisuke Ennishi 111
2:00 PM 3:30 PM
Analysis of 54 Follicular Lymphomas By Whole Exome Sequencing Identifies Multiple Novel
Recurrently Mutated Pathways Sami Malek 112
2:00 PM 3:30 PM
Frequent Activating Somatic Alterations in T-Cell Receptor / NF-κb Signaling in Adult T-Cell
Leukemia/Lymphoma Keisuke Kataoka 113
2:00 PM 3:30 PM Noninvasive Genotyping and Assessment of Treatment Response in Diffuse Large B Cell Lymphoma Florian Scherer 114
Session Name: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Novel Immunotherapeutics and the Impact of the Microenvironment
2:00 PM 3:30 PM
CAR-Engineered T Cells Specific for the Elotuzumab Target SLAMF7 Eliminate Primary Myeloma
Cells and Confer Selective Fratricide of SLAMF7+ Normal Lymphocyte Subsets Sophia Danhof 115

2:00 PM 3:30 PM
Bypassing the Constraint for Chimeric Antigen Receptor (CAR) Development in T-Cells Expressing
the Targeted Antigen: Improvement of Anti-CS1 CAR Activity in Allogenic TCRa/CS1 Double
Knockout T-Cells for the Treatment of Multiple Myeloma (MM) UCARTCS1 Cellectis Roman Galetto 116
2:00 PM 3:30 PM
Target Expression, Preclinical Activity and Mechanism of Action of EM801: A Novel First-in-Class
Bcma T-Cell Bispecific Antibody for the Treatment of Multiple Myeloma Anja Seckinger 117
2:00 PM 3:30 PM
A Novel Bispecific CD38 Antibody Eradicates Multiple Myeloma in a Mouse Model Following
Yttrium-90-DOTA Capture Damian Green 118
2:00 PM 3:30 PM
Anti-Sclerostin Treatment Prevents Multiple Myeloma Induced Bone Loss and Reduces Tumor
Burden Michaela Reagan 119
2:00 PM 3:30 PM Niche-Dependent Growth of Malignant and Pre-Neoplastic Plasma Cells in Humanized Mice Rituparna Das 120
Session Name: Contemporary Therapy of Lymphoma
2:00 PM 3:30 PM What Is the Role of Autologous Transplant for Lymphoma in the Current Era? Patrick Stiff
2:00 PM 3:30 PM Checkpoint Blockade in Lymphoma Philippe Armand
2:00 PM 3:30 PM B Cell Receptor Pathway Modulators in Non-Hodgkin Lymphoma Kristie Blum
Session Name: Cytosis: Too Much of a Good Thing
2:00 PM 3:30 PM Diagnosis and Management of Mastocytosis: An Emerging Challenge in Applied Hematology Peter Valent
2:00 PM 3:30 PM Eosinophilia: A Pragmatic Approach to Diagnosis and Treatment Amy Klion
2:00 PM 3:30 PM Lymphocytosis, Lymphadenopathy: Benign or Malignant? Jane Winter
Session Name: Genome Therapies for Hemoglobinopathies
2:00 PM 3:30 PM DNA Editing: A Primer Prashant Mali SCI-16
2:00 PM 3:30 PM Modified IPS Cells for Hemoglobinopathies Tim Townes SCI-17
2:00 PM 3:30 PM Bench to Bedside-Gene Therapy for Thalassemia and Sickle Cell Disease Philippe Leboulch SCI-18
Session Name: Hematopoiesis and the Niche
2:00 PM 3:30 PM Vascular Niche-Derived Angiocrine Factors Specify and Maintain Hematopoietic Stem Cells Shahin Rafii SCI-25
2:00 PM 3:30 PM
Noncellular Niche Influences: Nervous System Mediators, Metabolic Mediators, and
Hypoxia/Oxidative Stress Simón Méndez-Ferrer SCI-26
2:00 PM 3:30 PM Heterogeneity in the Making of Blood David Scadden SCI-27
Session Name: Inflammatory Signaling: Tailoring Myelopoiesis in Health and Disease
2:00 PM 3:30 PM Educating the Innate Immune System Scott Worthen SCI-28
2:00 PM 3:30 PM The Role of Receptor Interacting Protein Kinase in Myelopoiesis in Health and Disease John Silke SCI-29
2:00 PM 3:30 PM Reacting to Inflammatory Signals Nadia Carlesso SCI-30
Session Name: Junior-Faculty Development Education Session: Understanding the Basics of the Academic Job
2:00 PM 3:30 PM Strategies for Success for Junior Faculty Stephanie Lee
2:00 PM 3:30 PM Understanding Promotion and Tenure Robert Liem
2:00 PM 3:30 PM Looking for an Academic Job Janis Abkowitz
2:00 PM 3:30 PM Understanding Promotion and Tenure John Wagner
Session Name: Precision Medicine in Cancer Therapy: N-of-1
2:00 PM 3:30 PM Insights from the Study of Exceptional Responders Nikhil Wagle
2:00 PM 3:30 PM Precision Therapy of Lymphoma Inspired by Functional and Structural Genomics Louis Staudt
2:00 PM 3:30 PM Functional Screening to Guide Personalized Cancer Therapy Jeffrey Tyner

Session Name: Recent Advances in Histiocytic Neoplasms
2:00 PM 3:30 PM Treatment of Langerhans Cell Histiocytosis: Role of BRAF/MAPK Inhibition Sheila Weitzman
2:00 PM 3:30 PM
Biological and Clinical Significance of Somatic Mutations in Langerhans Cell Histiocytosis and
Related Histiocytic Neoplastic Disorders Donald Parsons
2:00 PM 3:30 PM
Uncommon Histiocyte Disorders: Rosai-Dorfman Disease, Juvenile Xanthogranuloma, and Erdheim-
Chester Disease Julien Haroche
Session Name: Send in the Clones: Stem Cell Succession, Replicative Stress and Somatic Reversion
2:00 PM 3:30 PM In vivo Stem Cell Clonal Dynamics Fernando Camargo SCI-40
2:00 PM 3:30 PM Stem Cell Replicative Stress and Clonal Exhaustion Michael Milsom SCI-41
2:00 PM 3:30 PM Clonal Reversion in Genetic Syndromes: Gene Therapy at Its Best Marjolijn Jongmans SCI-42
Session Name: Special Symposium on Quality
2:00 PM 3:30 PM
Quality Measures in Practice: The Medicare Access and CHIP Reauthorization Act of 2015
(MACRA)—What Clinicians Need to Know Lemeneh Tefera
2:00 PM 3:30 PM
Quality Measures, Quality Reporting, and Value-Based Remuneration: How Did We Get Here and
Where Are We Going? Helen Burstin
2:00 PM 3:30 PM Pay-for-Performance: Reviewing the Evidence Andrew Ryan
Session Name: Targeting the Tumor Microenvironment in Lymphoid Malignancies
2:00 PM 3:30 PM The Role of the Tumor Microenvironment in Lymphoid Malignancies Christian Steidl SCI-46
2:00 PM 3:30 PM Targeting the Immune Checkpoint Network in Lymphoid Malignancy Alan Ramsay SCI-47
2:00 PM 3:30 PM Re-educating the Tumor Microenvironment in the Clinic Stephen Ansell SCI-48
Session Name: Treatment of Acute Myeloid Leukemia: Moving Beyond 3 + 7
2:00 PM 3:30 PM Antigen-Specific Immunotherapies for Acute Myeloid Leukemia Roland Walter
2:00 PM 3:30 PM Molecularly Targeted Therapies for Acute Myeloid Leukemia Eytan Stein
2:00 PM 3:30 PM Newer Approaches to Transplantation in Acute Myeloid Leukemia Marcos de Lima
Session Name: Update on Diagnosis and Management of Thrombotic Thrombocytopenic Purpura
2:00 PM 3:30 PM
Beyond Simple Plasma Exchange: New Approaches in Treatment of Thrombotic Thrombocytopenic
Purpura Paul Coppo
2:00 PM 3:30 PM What’s New in the Diagnosis and Pathophysiology of Thrombotic Thrombocytopenic Purpura J. Evan Sadler
2:00 PM 3:30 PM Syndromes of Thrombotic Microangiopathy Associated with Pregnancy James George
Session Name: 321. Blood Coagulation and Fibrinolytic Factors: Biochemistry and Engineering of Coagulation Proteins
4:00 PM 5:30 PM
The X-Ray Structure of a Variant of Human Factor V Provides Structural Insights into the
Procofactor Activation Paradox Shekhar Kumar 121
4:00 PM 5:30 PM
Long-Range Allosteric Linkage Between Exosites Reciprocally Regulates the Zymogenicity of
Prothrombin Derivatives Joseph Micucci 122
4:00 PM 5:30 PM Bioengineering Coagulation Factor VIII through Ancestral Protein Reconstruction Philip Zakas 123
4:00 PM 5:30 PM
Prothrombin Membrane Binding and Gla-Dependent Function Are Not Required for Effective
Hemostasis In Vivo Lindsey Greene 124
4:00 PM 5:30 PM Tissue Factor Decryption By Oxidative Stress-Induced Lipid Peroxidation: Potential Mechanisms Shabbir Ansari 125
4:00 PM 5:30 PM
Engineered Factor Xa Variants Retain Procoagulant Activity Independent of Direct Factor Xa-
Inhibitors Daniël Verhoef 126

Session Name: 622. Non-Hodgkin Lymphoma: Biology, excluding Therapy: Clinical Implications of Genomic Studies of B-cell Lymphomas
4:00 PM 5:30 PM Liquid Biopsy As a Tool for Monitoring the Genotype of Diffuse Large B-Cell Lymphoma Silvia Rasi 127
4:00 PM 5:30 PM
Next Generation Sequencing Identifies a Distinct Transcriptional Profile, Including Isoform
Dysregulation That Segue with Genomic Alterations in Waldenstrom's Macoglobulinemia Zachary Hunter 128
4:00 PM 5:30 PM
Recurrent Mutations of the Exportin 1 Gene (XPO1) in Primary Mediastinal B-Cell Lymphoma: A
Lysa Study Fabrice Jardin 129
4:00 PM 5:30 PM
Dynamic Noninvasive Genomic Monitoring for Outcome Prediction in Diffuse Large B-Cell
Lymphoma David Kurtz 130
4:00 PM 5:30 PM Study of the Subclonal Mutations in Primary Diffuse Large B-Cell Lymphoma Yan Asmann 131
4:00 PM 5:30 PM
Characterization of MYD88 mutated Lymphoplasmacytic Lymphoma in Comparison to MYD88
mutated Chronic Lymphocytic Leukemia Constance Baer 132
Session Name: 632. Chronic Myeloid Leukemia: Therapy: Clinical Trials, Observations, and Molecular Monitoring
4:00 PM 5:30 PM
Personalized Daily Doses of Imatinib By Therapeutic Drug Monitoring Increase the Rates of
Molecular Responses in Patients with Chronic Myeloid Leukemia. Final Results of the Randomized
OPTIM Imatinib Study Philippe Rousselot 133
4:00 PM 5:30 PM
Combination of Dasatinib and Peg-Interferon Alpha 2b in Chronic Phase Chronic Myeloid Leukemia
(CP-CML) First Line: Preliminary Results of a Phase II Trial, from the French Intergroup of CML (Fi-
LMC) Lydia ROY 134
4:00 PM 5:30 PM
Quantification of BCR-ABL with Digital PCR Results in a Significantly Lower Rate of Deep Molecular
Responses When Compared to RT-qPCR in CML Patients Treated in the ENEST1st Trial Georg-Nikolaus Franke 135
4:00 PM 5:30 PM
International Scale (IS)-Standardized BCR-ABL1 Digital Polymerase Chain Reaction (dPCR) Assays
Using ABL1, BCR, and GUS Control Genes for Measuring Deep Molecular Response (MR) in Chronic
Myeloid Leukemia (CML) Alice Huang 136
4:00 PM 5:30 PM Osteoarticular Pain after Discontinuation of Tyrosine Kinase Inhibitors (TKI): A French Cohort Marc Berger 137
4:00 PM 5:30 PM
ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Exhibits Safety and Promising Single- Agent
Activity in a Phase I Study of Patients with CML with Failure of Prior TKI Therapy Novartis ABL-001 Oliver Ottmann 138
Session Name: 636. Myelodysplastic Syndromes – Basic and Translational Studies: Consequences of Splicing Factor Alterations and Genetic Instability
4:00 PM 5:30 PM
Different Mutant Splicing Factors Cause Distinct Missplicing Events and Give Rise to Different
Clinical Phenotypes in Myelodysplastic Syndromes Yusuke Shiozawa 139
4:00 PM 5:30 PM
Sf3b1 K700E Mutation Impairs Pre-mRNA Splicing and Definitive Hematopoiesis in a Conditional
Knock-in Mouse Model Annalisa Mupo 140
4:00 PM 5:30 PM
Integrative Genome-Wide Analysis of RNA Binding and Splicing Reveals Complex Loss and Gain of
Function Alterations By SRSF2 P95 Mutations in Myelodysplasia Kai Rejeski 141
4:00 PM 5:30 PM
Lineage-Specific Aberrant mRNA Splicing By U2AF1 Mutation Alters Erythroid and
Granulomonocytic Differentiation Bon Ham Yip 142
4:00 PM 5:30 PM
Ubiquitin Editing of a Spliceosome Auxiliary Factor By TRAF6 Links Chronic TLR Signaling with
Hematopoietic Defects and Myelodysplasia Jing Fang 143
4:00 PM 5:30 PM Inflammaging-Associated Metabolic Alterations Foster Development of the MDS Genotype Erika Eksioglu 144
Session Name: 702. Experimental Transplantation: Immune Function, GVHD, and Graft-versus-Tumor Effects: Novel Approaches to Dampen GVHD
4:00 PM 5:30 PM
Targeting the Human Notch 2-BCR Axis: A Driver of B-Cell Hyper-Responsiveness in Active Chronic
Graft-Versus Host Disease (cGVHD) Jonathan Poe 145
4:00 PM 5:30 PM Recipient Dendritic Cells Are Regulated By MiR-146a during Acute GvHD Kathrin Hanke 146
4:00 PM 5:30 PM
Loss of Programmed Death Ligand-1 Expression on Donor T Cells Lessens Acute Graft-Versus-Host
Disease Lethality Asim Saha 147
4:00 PM 5:30 PM NLRP6 in Host Target Tissues Exacerbates Graft-Versus-Host Disease Tomomi Toubai 148
4:00 PM 5:30 PM
ATG5 Dependent Autophagy Uncouples T Cell Functions and Modulates Experimental Graft-Versus-
Host Disease Katherine Oravecz-Wilson 149
4:00 PM 5:30 PM
Blockade of Interleukin 27 Signaling Attenuates Graft Versus Host Disease By Augmenting CD4+
and CD8+ Regulatory T Cell Reconstitution Ludovic Belle 150
Session Name: 732. Clinical Allogeneic Transplantation: Results II
4:00 PM 5:30 PM
Related Nonmyeloablative Haploidentical (mini-haplo) Blood or Marrow Transplantation (BMT)
with High-Dose Post-Transplant Cyclophosphmade (PTCy) for Acute Myeloid Leukemia (AML):
Donor Age Impacts Outcome Margaret Showel 151
4:00 PM 5:30 PM
Results of a Two-Arm Phase II Clinical Trial Using Post-Transplantation Cyclophosphamide for
Prevention of Graft-Versus-Host Disease in Haploidentical and Mismatched Unrelated Donors
Hematopoietic Stem-Cell Transplantation Sameh Gaballa 152
4:00 PM 5:30 PM
Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Inherited
or Acquired Bone Marrow Failure Disorders: A Report from Eurocord, Cord Blood Committee (CBC-
CTIWP) and Severe Aplastic Anemia Working Party (SAAWP) of the European Group of Blood and
Bone Marrow Transplantation (EBMT) Simona Pagliuca 153
4:00 PM 5:30 PM
The Impact of Donor Age on Outcome after Unrelated Bone Marrow Transplantation: Comparison
with Unrelated Cord Blood Transplantation Sachiko Seo 154
4:00 PM 5:30 PM
Reduced-Intensity Versus Myeloablative Conditioning for Unrelated Cord Blood Transplantation in
Adults with Acute Leukemia: A Report from Eurocord, the Acute Leukemia Working Party and the
Cord Blood Committee of the Cellular Therapy & Immunobiology Working Party of the European
Group for Blood and Marrow Transplantation Frederic Baron 155
4:00 PM 5:30 PM
HLA-Mismatched Microtransplantation Vs HLA-Matched Nonmyeloablative Transplantation for
Acute Myeloid Leukemia in Intermediate-Risk: Comparable Survival but Avoids of Gvhd Huisheng Ai 156
Session Name: ASH/ASCP Summit on Myelodysplastic Syndromes
4:00 PM 5:30 PM Treating MDS Outliers: Patients with Secondary Diseases and Comorbidities Rami Komrokji
4:00 PM 5:30 PM Prognostic Systems Masquerading As Default Staging Systems for Myelodysplastic Syndromes Mikkael Sekeres
4:00 PM 5:30 PM MDS Mimics: What You See Is Not Always What You Get Timothy Miley

Session Name: Evolving Issues in Anticoagulation
4:00 PM 5:30 PM Anticoagulation in Children and Adolescents Guy Young
4:00 PM 5:30 PM Monitoring and Reversal of Direct Oral Anticoagulants Adam Cuker
4:00 PM 5:30 PM Using Direct Oral Anticoagulants in Cancer and Other High-Risk Populations Harry Büller
Session Name: Genomically Engineered Stem Cells: A Brave New World for Therapeutics
4:00 PM 5:30 PM Making Hematopoietic Stem Cells: Backwards and Sideways Derrick Rossi
4:00 PM 5:30 PM Blood Cells from Pluripotent Stem Cells: The Platelet Story Mortimer Poncz
4:00 PM 5:30 PM Engineering Universal Donor Stem Cells David Russell
Session Name: Multiple Advances in Myeloma
4:00 PM 5:30 PM The Role of Maintenance Therapy for Multiple Myeloma in the Era of Novel Agents Thierry Facon
4:00 PM 5:30 PM When Does "Smoldering" Become an Open Flame? Evolving Diagnostic Criteria for Myeloma S. Vincent Rajkumar
4:00 PM 5:30 PM Novel Combination Approaches to Myeloma Sagar Lonial

Session Name: Hematology Course Directors' Workshop
Session Location: Manatee Springs (Hyatt Regency Orlando)
7:00 AM 9:00 AM Team-Based Learning Marc Kahn
7:00 AM 9:00 AM New Curricula in Medical Schools Jennifer Kesselheim
7:00 AM 9:00 AM LCME Transition Overview Marc Zumberg
7:00 AM 9:00 AM Flipping Classrooms Gowthami Arepally
Session Name: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Erythropoiesis
7:30 AM 9:00 AM
Conditional Knockout-First Gene Disruption of Dematin Causes Precipitous Loss of Erythrocyte
Membrane Stability and Severe Hemolytic Anemia Athar Chishti 157
7:30 AM 9:00 AM Mirna Regulation of Blood Group AB0 Genes
Romy Kronstein-
Wiedemann 158
7:30 AM 9:00 AM Distinct Roles of TET Proteins in the Regulation of Normal and Disordered Human Erythropoiesis Xiuli An 159
7:30 AM 9:00 AM
Pomalidomide Transcriptionally Reprograms Adult Erythroid Progenitors Independently of Ikaros
Proteasomal Degradation Celgene Pomalyst Brian Dulmovits 160
7:30 AM 9:00 AM
Mechanism Governing Heme Synthesis Reveals a GATA Factor-Heme Circuit That Controls
Differentiation Emery Bresnick 161
7:30 AM 9:00 AM
Staphylococcus Aureus Targets the Duffy Antigen Receptor for Chemokines (DARC) to Lyse
Erythrocytes Yves Colin Aronovicz 162
Session Name: 601. Chromosomal Rearrangements and DNA Repair: Genomic Instability and Clonal Evolution In Hematopoietic Malignancy
7:30 AM 9:00 AM Differential Regulation of c-Myc/Lin28 Discriminates Subclasses of Rearranged MLL Leukemia Lili Chen 163
7:30 AM 9:00 AM PI3Kdelta Inhibitors Increase Genomic Instability By Upregulating Aid Expression Mara Compagno 164
7:30 AM 9:00 AM
Crispr-Cas9 Induced MLL-Rearrangements Cause Clonal Outgrowth in CD34+ Hematopoietic Stem
Cells Dirk Heckl 165
7:30 AM 9:00 AM
Exposure to Inflammatory Immune Responses As Driver of Clonal Evolution in Childhood Acute
Lymphoblastic Leukemia Lars Klemm 166
7:30 AM 9:00 AM
Kinetics of CLL Subclonal Architecture: Spontaneous Disease Progression or Treatment-Induced
Selection? Ceri Oldreive 167
7:30 AM 9:00 AM
Models of Pluripotent and Somatic Stem Cells to Study Tissue-Specific Sensitivities in Fanconi
Anemia Timothy Chlon 168
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics and Molecular Markers in Diagnosis and Prognosis: Novel Genetic Lesions in AML – Insight from Genome Wide Characterization
7:30 AM 9:00 AM
Rearrangements in Nucleoporin Family of Genes in Childhood Acute Myeloid Leukemia: A Report
from Children Oncology Group and NCI/COG Target AML Initiative Fabiana Ostronoff 169
7:30 AM 9:00 AM
Comprehensive Genomic and Transcript Profiling of CBL Gene in Childhood AML: A Report from
Children's Oncology Group Studies AAML03P1, AAML0531 and COG/NCI Target AML Initiative Diana Chin 170
7:30 AM 9:00 AM
Next Generation Sequencing Identifies a Novel Subset of Non-Down Syndrome Acute
Megakaryoblastic Leukemia Characterized By Chimeric Transcripts Involving HOX Cluster Genes Jasmijn de Rooij 171
7:30 AM 9:00 AM
Predictors of Early Death in Childhood Acute Promyelocytic Leukemia: Results of an International
Retrospective Study Oussama Abla 172
7:30 AM 9:00 AM
Significance of Peri-Transplant Dynamics of Minimal Residual Disease (MRD) in Adults with Acute
Myeloid Leukemia (AML) in Morphological Remission Undergoing Myeloablative Allogeneic
Hematopoietic Cell Transplantation Yi Zhou 173
7:30 AM 9:00 AM
CSF3R Mutations Represent a Novel Therapeutic Target in Pediatric AML with a High Degree of
Overlap with CEBPA Mutations: a Report from COG AAML0531 and COG/NCI Target AML Initiative Julia Maxson 174
Session Name: 621. Hodgkin Lymphoma: Biology, excluding Therapy: Biological Insights and Clinical Impact
7:30 AM 9:00 AM
Prediction of Primary Treatment Outcome Using Gene Expression Profiling of Pre-Treatment
Biopsies Obtained from Childhood and Adolescent Hodgkin Lymphoma Patients Anja Mottok 175
7:30 AM 9:00 AM PD-L1 and PD-L2 Genetic Alterations Define Classical Hodgkin Lymphoma and Predict Outcome Margaretha Roemer 176
7:30 AM 9:00 AM
Disruption of Direct 3-Dimensional (3D) Telomere-TRF2 (Telomere Related Factor 2) Interaction Is
a Hallmark of Primary Hodgkin (H) and Reed-Sternberg (RS) Cells Hans Knecht 177
7:30 AM 9:00 AM Analysis of the Genomic Heterogeneity in Hodgkin Lymphoma Using Next Generation Sequencing Juan F García 178
7:30 AM 9:00 AM DNA Methylation Differences in Twins Discordant for Adolescent/Young Adult Hodgkin Lymphoma Wendy Cozen 179
7:30 AM 9:00 AM
TARC Predicts PET-Normalization and Event Free Surival in Relapsed/Refractory Hodgkin
Lymphoma Patients Treated with Brentuximab Vedotin Seattle Genetics Adcetris Alison Moskowitz 180
Session Name: 624. Lymphoma: Therapy with Biologic Agents, excluding Pre-Clinical Models: Novel Immunotherapy Strategies in Lymphoma
7:30 AM 9:00 AM
Multicenter Phase II Study of Lenalidomide in Patients with Relapsed Adult T-Cell Leukemia-
Lymphoma Celgene Revlimid Hiroshi Fujiwara 181
7:30 AM 9:00 AM
A Phase 1 Study of Denintuzumab Mafodotin (SGN-CD19A) in Relapsed/Refactory B-Lineage Non-
Hodgkin Lymphoma Seattle Genetics Denintuzumab Mafodotin Craig Moskowitz 182
7:30 AM 9:00 AM
Sustained Remissions Following Chimeric Antigen Receptor Modified T Cells Directed Against CD19
(CTL019) in Patients with Relapsed or Refractory CD19+ Lymphomas Novartis CTL019 Stephen Schuster 183
7:30 AM 9:00 AM
Anti-CD19 Chimeric Antigen Receptor-Modified T Cell Therapy for B Cell Non-Hodgkin Lymphoma
and Chronic Lymphocytic Leukemia: Fludarabine and Cyclophosphamide Lymphodepletion
Improves In Vivo Expansion and Persistence of CAR-T Cells and Clinical Outcomes Cameron Turtle 184
7:30 AM 9:00 AM Chimeric T Cells for Therapy of CD30+ Hodgkin and Non-Hodgkin Lymphomas Carlos Ramos 185
7:30 AM 9:00 AM Immunotherapy for Lymphoma Using T Cells Targeting Multiple Tumor Associated Antigens Ann Leen 186
Sunday December 6- Oral Presentations

Session Name: 653. Myeloma: Therapy, excluding Transplantation: Amyloidosis and Related Plasma Cell Disorders
7:30 AM 9:00 AM
Phase I/II Trial of the Efficacy and Safety of Combination Therapy with
Lenalidomide/Bortezomib/Dexamethasone (RVD) and Panobinostat in Transplant-Eligible Patients
with Newly Diagnosed Multiple Myeloma Novartis Farydak Jatin Shah 187
7:30 AM 9:00 AM
Results of Phase I Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients with
AL Amyloidosis Suzanne Lentzsch 188
7:30 AM 9:00 AM
Quadruplet Vs Sequential Triplet Induction Therapy Approaches to Maximise Response for Newly
Diagnosed, Transplant Eligible, Myeloma Patients Charlotte Pawlyn 189
7:30 AM 9:00 AM
Accurate Risk Stratification Identifies Patients with AL Amyloidosis Benefiting Most from Upfront
Bortezomib Combinations: A Study of Treatment Outcomes in 984 Patients Giovanni Palladini 190
7:30 AM 9:00 AM
Evaluation of Minimal Residual Disease (MRD) By Next Generation Sequencing (NGS) Is Highly
Predictive of Progression Free Survival in the IFM/DFCI 2009 Trial Herve Avet-Loiseau 191
7:30 AM 9:00 AM
A Prospective Phase II Study of Combination of Low Dose Lenalidomide and Dexamethasone for
Patients with Newly Diagnosed POEMS Syndrome Dao-bin Zhou 192
Session Name: 732. Clinical Allogeneic Transplantation: Results: Matched Related, Unrelated, and Alternative Donor Allograft Outcomes for Myeloid and Lymphoid Malignancies
7:30 AM 9:00 AM
Outcome of Patients 65 Years and Older with Myelodysplastic Syndrome (MDS) Receiving
Allogeneic Hematopoietic Stem Cell Transplantation Compared to Patients 55-64 Years of Age Ehab Atallah 193
7:30 AM 9:00 AM
Survival after T-Cell Replete Haploidentical Related Donor Transplant Using Post-Transplant
Cyclophosphamide Compared with Matched Unrelated Donor (MUD) Transplant for Lymphoid
Malignancies Alberto Mussetti 194
7:30 AM 9:00 AM
Children with Acute Leukemia Given Hematopoietic Stem Cell Transplantation (HSCT) from an HLA-
Compatible Sibling, or an Unrelated Donor (UD) or an HLA-Haploidentical Relative after
Alpha/Beta T-Cell Depletion Have a Comparable Outcome Alice Bertaina 195
7:30 AM 9:00 AM
Comparison of Matched-Sibling Donors Versus Unrelated Donors in Allogeneic Stem Cell
Transplantation (allo-SCT) for Primary Refractory Acute Myeloid Leukemia (PRF AML): A Report of
1041 Patients from the Acute Leukemia Working Party of the EBMT Eolia Brissot 196
7:30 AM 9:00 AM
Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation (allo-HCT) Provides Durable
Progression-Free Survival (PFS) in a Subset of Diffuse Large B-Cell Lymphoma (DLBCL) Patients
Relapsing after Autologous (auto-) HCT Timothy Fenske 197
7:30 AM 9:00 AM
Allogeneic Stem Cell Transplantation for Relapsed / Refractory (R/R) Follicular Lymphoma (FL). a
Joint Study Between the European Society for Blood and Marrow Transplantation (EBMT) and the
Center for International Blood and Marrow Transplant Research (CIBMTR) Anna Sureda 198
Session Name: 801. Gene Therapy and Transfer: Gene Therapy for Hemoglobinopathies and Inherited Bleeding Disorders
7:30 AM 9:00 AM
Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic
Factor VIII Levels in Non-Human Primates Xavier Anguela 199
7:30 AM 9:00 AM
ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of
Human FIX in Mice and Non-Human Primates Michael Holmes 200
7:30 AM 9:00 AM
Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for
Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced
Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product) bluebird bio LentiGlobin Mark Walters 201
7:30 AM 9:00 AM
Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via
Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta
AT87Q-Globin Vector bluebird bio LentiGlobin Marina Cavazzana 202
7:30 AM 9:00 AM
Genome Editing of the Bcl11A Erythroid Specific Enhancer in Bone Marrow Derived Hematopoietic
Stem and Progenitor Cells for the Treatment of Sickle Cell Disease Siyuan Tan 203
7:30 AM 9:00 AM
Clinical-Scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of the
Hemoglobinopathies Fyodor Urnov 204
Session Name: Anemia: When Something's Wrong with Iron
7:30 AM 9:00 AM Iron Deficiency: New Insights into Diagnosis and Treatment Clara Camaschella
7:30 AM 9:00 AM Understanding Anemia of Chronic Disease Paula Fraenkel
7:30 AM 9:00 AM
Diagnosis and Treatment of Sideroblastic Anemias: From Defective Heme Synthesis to Abnormal
RNA Splicing Mario Cazzola
Session Name: Guiding Hematologic Care with Genetic Testing
7:30 AM 9:00 AM Using Genetic Diagnostics in Hemophilia and von Willebrand Disease Paula James
7:30 AM 9:00 AM Sickle Cell Trait Diagnosis: Clinical and Social Implications Rakhi Naik
7:30 AM 9:00 AM Using Red Blood Cell Genomics in Transfusion Medicine Jill Johnsen
Session Name: Imaging and Treating the Clot: What's New?
7:30 AM 9:00 AM Incidental Pulmonary Embolism: What’s the Clinician to do? Casey O'Connell
7:30 AM 9:00 AM Imaging New and Recurrent Thrombosis Menno Huisman
7:30 AM 9:00 AM Clot Lysis and Stenting Suresh Vedantham
Session Name: Overcoming the Barrier of TP53 Dysfunction to Cure Blood Cancers
7:30 AM 9:00 AM Deconstructing p53 Transcriptional Networks in Tumor Suppression Laura Attardi
7:30 AM 9:00 AM Biology of TP53 Loss and Mutation Guillermina Lozano
7:30 AM 9:00 AM Circumventing TP53 Dysfunction: Targeting Cell Survival Pathways David Huang

Session Name: Palliative Care in Hematological Malignancies
7:30 AM 9:00 AM Spanning the Canyon Between Stem Cell Transplantation and Palliative Care Eric Roeland
7:30 AM 9:00 AM When and Why Should Patients with Hematological Malignancies See a Palliative Care Specialist? Thomas LeBlanc
7:30 AM 9:00 AM Palliative Care in Pediatric Patients with Hematologic Malignancies Tammy Kang
Session Name: 203. Lymphocytes, Lymphocyte Activation and Immunodeficiency, including HIV and Other Infections: Novel Immunodeficiencies and Natural Killer Cell Biology
9:30 AM 11:00 AM
Impairment of IL-17 Immunity to Candida and IFN-g Immunity to Mycobacterium in Humans with
Bi-Allelic Rorc mutations Satoshi Okada 205
9:30 AM 11:00 AM Dominant NFKB1 Mutations Cause Antibody Deficiency and Autoinflammatory Episodes Emma Haapaniemi 206
9:30 AM 11:00 AM Microrna 29b Mediates Immune Evasion of Natural Killer Cells in Acute Myeloid Leukemia Steven Scoville 207
9:30 AM 11:00 AM
Impact of Kindlin-3 in NK Cell-Mediated Anti-Tumor Cytotoxicity and Inflammatory Cytokine
Production Nathan Schloemer 208
9:30 AM 11:00 AM
mTORC1 Signaling and Metabolism Are Dynamically Regulated during Human NK Cell Maturation
and in Adaptive NK Cell Responses to Viral Infection Cichocki Frank 209
9:30 AM 11:00 AM
Modulation of Natural Killer Cell Activity in the Setting of Oncolytic Virotherapy and with a
Chimeric Antigen Receptor Chen Xilin 210
Session Name: 321. Blood Coagulation and Fibrinolytic Factors: Coagulation Proteins and Blood Vessels
9:30 AM 11:00 AM Bleeding Phenotype Observed in Murine Defect of VWF-Collagen 4 Interactions Veronica Flood 211
9:30 AM 11:00 AM
A Targeted Photochemical Microfluidic Vascular Injury Model for in Vitro Thrombosis Studies:
Usage in Heparin-Induced Thrombocytopenia (HIT) Ian Johnston 212
9:30 AM 11:00 AM
The Role of Putative Phosphatidylserine-Interactive Residues of Tissue Factor on Its Coagulant
Activity at the Cell Surface Shabbir Ansari 213
9:30 AM 11:00 AM
Von Willebrand Factor Protects Factor VIII from Rapid Endocytosis Via LDL Receptor-Related
Protein By Blocking the Initial Rate-Limiting Cell Surface Binding Step of Factor VIII Henriet Meems 214
9:30 AM 11:00 AM Clot Contraction-Mediated Erythrocyte Packing Is Significantly Altered in Sickle Cell Disease Hunter Strauss 215
9:30 AM 11:00 AM Coagulation Factor V Mediates Inhibition of Tissue Factor Signaling By Activated Protein C Hartmut Weiler 216
Session Name: 615. Acute Myeloid Leukemia: Commercially available Therapy, excluding Transplantation: New Approaches Using Older Drugs
9:30 AM 11:00 AM
North American Leukemia‚ Intergroup Phase III Randomized Trial of Single Agent Clofarabine As
Induction and Post-Remission Therapy‚ and Decitabine As Maintenance Therapy in Newly-
Diagnosed Acute Myeloid Leukemia in Older Adults (Age ≥60 Years): A Trial of the ECOG-ACRIN
Cancer Research Group (E2906) James Foran 217
9:30 AM 11:00 AM
Clofarabine-Based Consolidation Improves Relapse-Free Survival of Younger Adults with Non-
Favorable Acute Myeloid Leukemia (AML) in First Remission: Results of the Randomized ALFA-
0702/Clara Study (NCT 00932412) Xavier Thomas 218
9:30 AM 11:00 AM
Results of a Phase III Trial Including Arsenic Trioxide Consolidation for Pediatric Patients with Acute
Promyelocytic Leukemia (APL): A Report from the Children's Oncology Group Study AAML0631 Matthew Kutny 219
9:30 AM 11:00 AM
Tamibarotene Compared to All-Trans Retinoic Acid (ATRA) As Add-on to Arsenic Trioxide (ATO) in
Subjects with Relapsed Acute Promyelocytic Leukemia (APL) Jianxiang Wang 220
9:30 AM 11:00 AM
A Comparison of 1 or 2 Courses of High Dose Cytarabine As Consolidation in Younger Patients with
AML: First Results of the UK NCRI AML17 Trial Nigel Russell 221
9:30 AM 11:00 AM
Randomized Comparison of Intermediate-Dose Cytarabine Plus Mitoxantrone (IMA) Versus
Standard-Dose Cytarabine Plus Daunorubicin (DA) for Induction Therapy in AML Patients >60
Years. Results from the SAL 60+ Trial Christoph Röllig 222
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics and Molecular Markers in Diagnosis and Prognosis: Novel Molecular Markers for the Detection of Clonal Hematopoiesis and Minimal Residual Disease
9:30 AM 11:00 AM On the Origins of AML Relapse Liran Shlush 223
9:30 AM 11:00 AM Characteristics and Prognosis of AML Patients with or without a History of Clonal Hematopoiesis Felicitas Thol 224
9:30 AM 11:00 AM
High-Sensitivity Genomic Minimal Residual Disease Detection Reveals Multiclonal Hematopoiesis
and Is Associated with Survival in Adult AML Brian Parkin 225
9:30 AM 11:00 AM
Monitoring of Minimal Residual Disease (MRD) of DNMT3A Mutations (DNMT3Amut) in Acute
Myeloid Leukemia (AML): A Study of the AML Study Group (AMLSG) Verena Gaidzik 226
9:30 AM 11:00 AM
Clinical Relevance of Minimal Residual Disease Monitoring in NPM1 Mutated AML: A Study of the
AML Study Group (AMLSG) Silke Kapp-Schwoerer 227
9:30 AM 11:00 AM
Clinical Impact of Minimal Residual Disease (MRD) Monitoring in AML with PML-Rara, CBFB-
MYH11, and RUNX1-RUNX1T1: A Study on 600 Patients Torsten Haferlach 228
Session Name: Challenges in Hemophilia Care
9:30 AM 11:00 AM Emerging Genetic and Pharmacologic Therapies for Controlling Hemostasis Paul Monahan
9:30 AM 11:00 AM The Long and Short of It: Using the New Factor Products Amy Dunn
9:30 AM 11:00 AM Managing Older Patients with Hemophilia Suman Sood
Session Name: Hemophagocytic Lymphohistiocytosis in Adults
9:30 AM 11:00 AM Focus on Presentation and Diagnosis of Hemophagocytic Lymphohistiocytosis in Adults Nancy Berliner
9:30 AM 11:00 AM Pathophysiology and Epidemiology of Hemophagocytic Lymphohistiocytosis Carl Allen
9:30 AM 11:00 AM Treatment of Hemophagocytic Lymphohistiocytosis in Adults Paul La Rosée

Session Name: New Approaches in Chronic Lymphocytic Leukemia and Prolymphocytic Leukemia
9:30 AM 11:00 AM Management of Prolymphocytic Leukemia Claire Dearden
9:30 AM 11:00 AM
Patterns of Resistance to B-Cell Receptor Pathway Antagonists in Chronic Lymphocytic Leukemia
and Strategies for Management Jennifer Woyach
9:30 AM 11:00 AM
Emerging Prognostic Markers in Chronic Lymphocytic Leukemia and their Impact on Treatment
Strategies Stephan Stilgenbauer
Session Name: Optimizing Transfusion Therapy (AABB Session)
9:30 AM 11:00 AM Red Blood Cell Transfusion Goals in Hematologic Disease Brenda Grossman
9:30 AM 11:00 AM Implementing a Blood Management Program to Optimize Transfusion Utilization William Savage
9:30 AM 11:00 AM Platelet Transfusion Goals in Hematologic Disease Cassandra Josephson
Session Name: Patient-Reported Outcomes in Hematology
9:30 AM 11:00 AM Patient-Reported Outcomes and Drug Development Catherine Acquadro
9:30 AM 11:00 AM The Science Behind Developing a Patient-Reported Outcome Measure David Cella
9:30 AM 11:00 AM Patient-Reported Outcomes in Clinical Practice Julie Panepinto
9:30 AM 11:00 AM Patient-Reported Outcomes and Drug Development Virginia Kwitkowski
Session Name: 702. Experimental Transplantation: Immune Function, GVHD, and Graft-versus-Tumor Effects: Shifting the Balance Between GVH, GVT, and Post-transplant Immunity
9:30 AM 11:15 AM
Donor T Cells Intrinsic Responses to Damps Regulated By Siglec-G-CD24 Axis Mitigate Gvhd but
Maintain GVL in Experimental BMT Model Tomomi Toubai 229
9:30 AM 11:15 AM
R-spondin1 Promotes Paneth Cell Growth, Maintains Intestinal Microbial Ecology, and Ameliorates
GvHD Eiko Hayase 230
9:30 AM 11:15 AM
IL-33/ST2 Triggering of IL-9-Secreting T Cells Alters the Balance of Fatal Immunity and Tumor
Immunity Abdulraouf Ramadan 231
9:30 AM 11:15 AM
Trispecific Killer Engagers (TriKEs) that contain IL-15 to make NK cells antigen specific and to
sustain their persistence and expansion Jeffrey Miller 232
9:30 AM 11:15 AM
Programming of Donor T Cells Using Allogeneic Delta-like Ligand 4-Positive Dendritic Cells to
Reduce Gvhd but Retain GVL Activity Lijun Meng 233
9:30 AM 11:15 AM
Pharmacological Inhibition of Hsp90 Destabilizes the Histone Methyltransferase Ezh2 in
Alloreactive T Cells and Reduces Graft-Versus-Host Disease While Retaining Anti-Leukemic Effects
in Mice Qingrong Huang 234
9:30 AM 11:15 AM Production of BMP4 By Endothelial Cells Is Crucial for Endogenous Thymic Regeneration Jarrod Dudakov 637
Session Name: Continuing Conversations with the Speakers on Harnessing T cells in Therapy of Hematologic Malignancy
11:15 AM 12:15 PM Speaker Michel Sadelain
11:15 AM 12:15 PM Speaker Dirk Busch
11:15 AM 12:15 PM Speaker Crystal Mackall
Session Name: Continuing Conversations with the Speakers on Send in the Clones: Stem Cell Succession, Replicative Stress and Somatic Reversion
Session Location: Lecture Hall W300 (Orange County Convention Center)
11:15 AM 12:15 PM Speaker Michael Milsom
11:15 AM 12:15 PM Speaker Fernando Camargo
11:15 AM 12:15 PM Speaker Marjolijn Jongmans

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