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American Society of Hematology Annual Meeting UCSF HELEN DILLER FAMILY COMPREHENSIVE CANCER CENTER December 1-4, 2018 San Diego, CA UCSF Presentation Brochure

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Page 1: American Society of Hematology Annual Meetingcancer.ucsf.edu/_docs/UCSFCancerCenter_ASH2018_Brochure.pdf · 1 American Society of Hematology Annual Meeting UCSF HELEN DILLER FAMILY

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American Society of Hematology Annual Meeting

UCSF

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December 1-4, 2018San Diego, CAUCSF Presentation Brochure

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Page 3: American Society of Hematology Annual Meetingcancer.ucsf.edu/_docs/UCSFCancerCenter_ASH2018_Brochure.pdf · 1 American Society of Hematology Annual Meeting UCSF HELEN DILLER FAMILY

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For the past three years, it has been my privilege to work with UCSF’s team of exceptional cancer scientists who are helping to transform cancer into a manageable disease, and hopefully one day, a curable disease. Among our many programs, UCSF faculty are driving efforts to overcome drug resistance, a key limitation of targeted therapy; developing drugs against previously undruggable proteins; and making significant advances in cryo-electron microscopy that is allowing scientists to visualize a broad spectrum of proteins at resolutions approaching crystal structure resolutions. We are leading the way on the next generation of cellular therapies, with novel approaches to addressing specificity and efficacy, while minimizing side effects. Also, we’ve expanded our state-of-the-art cancer imaging program to more rapidly assess disease progression and treatment response, creating time to move patients to other therapies when first-line therapies fail. All of us are working in an exciting new era of collaboration. We recognize that bringing advancements to patients is best accomplished by working in partnership with the broader life science industry. As an NCI-designated comprehensive cancer center, UCSF is recognized for our outstanding science, extensive resources, depth and breadth of our research in basic, clinical, and population sciences, as well as cutting edge research that bridges these scientific areas. UCSF is home to many of the world’s finest oncology clinicians and scientists who understand the power of partnerships. This searchable abstract book of UCSF research presented at ASH is a resource for potential partners interested in identifying world-class faculty engaged in basic science and clinical oncology research. I invite you to learn more about our work and expertise by reaching out to our faculty during this meeting. If you have additional questions or would like assistance with your outreach, please contact the Director of Strategic Alliances for the Cancer Center: Cammie Edwards ( ). I wish you a very productive meeting, and we look forward to future discussions and collaborations.

Alan Ashworth, PhD, FRS

EXCEPTIONAL PEOPLE EXTRAORDINARY SCIENCE

Alan Ashworth, PhD, FRS

President, UCSF Helen Diller Family Comprehensive Cancer Center

Senior Vice President for Cancer Services,UCSF Health

Professor of Medicine, Division of Hematology/Oncology, Department of Medicine

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OUR SUCCESS IS DRIVEN BY OUR FACULTYHDFCCC Membership: 455 Members & Affiliate Members

2 Nobel Laureates3 Albert Lasker Award winners8 Howard Hughes Medical Investigators16 Members of the National Academy of Sciences20 Members of the Institute of Medicine23 Fellows of the American Academy of Arts and Sciences6 Fellows of the Royal Society

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UCSF IN THE NEWS (Click on link to read story)

UCSF Medical Center Is Best Hospital in California for 2018-19Continues to Rank Among Nation’s Top Hospitals in Adult Specialty Carehttp://cancer.ucsf.edu/news/2018/08/14/ucsf-medical-center-is-best-hospital-in-california-for-2018-19.9076

‘Orphan’ RNAs Make Cancer Deadlier, But Potentially Easier to Diagnose Newly Discovered Regulatory Molecule Promotes Metastasis but May Lead to Non-Invasive Diagnoseshttp://cancer.ucsf.edu/news/2018/11/07/orphan-rnas-make-cancer-deadlier-but-potentially-easier-to-diagnose.9271

The First Genome Surgeons: Scientists Are Preparing to Bring DNA-Editing Tools to the Clinichttp://cancer.ucsf.edu/news/2018/10/24/the-first-genome-surgeons-scientists-are-preparing-to-bring-dna-editing-tools-to-the-clinic.9230

Genetic Study of Familial Leukemia Solves 30-Year Medical Mystery http://cancer.ucsf.edu/news/2018/07/27/genetic-study-of-familial-leukemia-solves-30-year-medical-mystery.9052

T Cell Engineering Breakthrough Sidesteps Need for Viruses in CRISPR Gene-Editing With Faster, Cheaper, More Precise Technique, Authors Say It’s ‘Off to the Races’ Toward New Cell Therapies http://cancer.ucsf.edu/news/2018/07/11/t-cell-engineering-breakthrough-sidesteps-need-for-viruses-in-gene-editing.9034

Mutations, Drugs Drive Cancer by Blurring Growth Signals Novel Technique Lets Researchers Control Common Cancer Pathway in the Lab with Pulses of Light http://cancer.ucsf.edu/news/2018/09/06/mutations-drugs-drive-cancer-by-blurring-growth-signals.9150

Chronic Inflammation Can Trigger Cancers Via Newly Discovered Mechanism Research on Severe Inherited Childhood Skin Disorder Reveals Insights About How Tissue Injury Can Lead to Cancerhttp://cancer.ucsf.edu/news/2018/08/23/chronic-inflammation-can-trigger-cancers-via-newly-discovered-mechanism.9128

Present at Creation of Nobel-Winning ‘Checkpoint Inhibitor’ Therapies, Immunologist Looks to the Future Nobel Prize Win Recognizes Cancer Immunotherapy’s Coming of Age http://cancer.ucsf.edu/news/2018/10/02/present-at-creation-of-nobel-winning-checkpoint-inhibitor-therapies-immunologist-looks-to-the-future.9194

UCSF Health and John Muir Health to Build East Bay Cancer NetworkCancer Collaboration Will Offer State-of-the-Art Care to Patients in East Bay http://cancer.ucsf.edu/news/2018/10/01/ucsf-health-and-john-muir-health-to-build-east-bay-cancer-network.9192

Feature: Cancer Imaging Research Corehttp://cancer.ucsf.edu/news/2018/10/16/feature-cancer-imaging-research-core.9158

Catherine Smith, MD Named 2018 Damon Runyon Clinical Investigatorhttp://cancer.ucsf.edu/news/2018/08/14/blakely-and-smith-named-2018-damon-runyon-clinical-investigators.9085

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BLOOD MALIGNANCIES AND DISEASESUCSF has over 50 scientists and clinicians working in the areas of myelodysplastic syndromes,

myeloproliferative disorders, lymphomas, leukemias, myelomas, blood and marrow transplant,

hemophilia, and amyloidosis. With our growing programs, combined expertise, and access to

resources, UCSF faculty continue to make significant strides in understanding the biology of

hematological diseases and improving patient outcomes with advanced clinical care.

WORKING TOGETHER ADVANCING THE UNDERSTANDING AND TREATMENT OF CANCER

NCI - Supported Research Programs (click on link to get more information)

Additional Cancer Research (click on link to get more information)

Key Initiatives (click on link to get more information)

• Cancer Risk

• Gastrointestinal Oncology

• Gynecologic Oncology

• Melanoma

• Multiple Myeloma

• Pancreas Cancer

• Pediatric Brain Tumor Research

• Thoracic Oncology

• Cancer Immunotherapy

• Center for BRCA Research

• Global Cancer

• Molecular Oncology

• Precision Cancer Medicine Building

• The San Francisco Cancer Initiative (SF CAN)

• University of California Cancer Consortium

• Breast Oncology

• Cancer Control

• Cancer Genetics

• Cancer Immunology

• Experimental Therapeutics

• Hematopoietic Malignancies

• Neurologic Oncology

• Pediatric Malignancies

• Prostate Cancer

• Tobacco Control

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CORE CAPABILITIES SUPPORTING OUR PROGRAMS (click on link to get more information)

Biorepository and Tissue Biomarker TechnologyProvides optimal acquisition, processing, and storage of human tissue biospecimens, as well as state-of-the-art biomarker histologic detection and/or image analyses for both human and mouse tissue biospecimens.

BiostatisticsProvides statistical expertise and collaboration to the UCSF cancer research community on all phases of basic science, translational, clinical, epidemiological, and prevention research.

Cancer Imaging ResearchProvides technical capabilities and scientific expertise for integrating cutting-edge, multi-modality imaging into basic, translational, and clinical research.

Computational Biology and InformaticsProvides computational biology and computational infrastructure support to the UCSF cancer research community.

Laboratory for Cell AnalysisProvides cytometric, microscopic, and genomic support and services for the UCSF cancer research community.

Preclinical TherapeuticsOffers a complete set of preclinical services and in vivo imaging devices for cancer investigators.

Small Molecule DiscoveryCollaborates with academics, government labs, and pharmaceutical companies to develop unique chemical probes and drug leads that address unmet medical needs in cancer.

Tobacco BiomarkersServes as an analytical chemistry resource for the UCSF tobacco control and cancer research community.

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FRIDAY | NOVEMBER 30, 2018

TriLeukeVax: A Next Generation Autologous AML Vaccine for Transplant Ineligible Older Patients

Authors*: Karin L. Gaensler, MD

Abstract #: Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper120613.htmlPresentation Date/Time: Friday, November 30, 2018, 2:00 PM-3:00 PMLocation: Room 33B (San Diego Convention Center) Presentation Type: Scientific Workshop

Gaensler Research Interests: The Gaensler lab has pursued gene transfer and stem cell transplantation approaches for the treatment of hematologic disorders. Translational studies now focus on immunotherapeutic approaches to increase progression-free and overall survival in older individuals with acute myelogenous leukemia (AML). Compelling evidence for the efficacy of immunotherapy in eliminating minimal residual disease (MRD) is provided by the superior outcomes of allogeneic hematopoietic stem cell transplants (HSCT) due to the leukemia-specific immune responses of donor cells. However, patients > 60 yo are often ineligible for allo-HSCT. Two vaccine approaches are being developed as part of a collaborative consortium at UCSF, UCLA, UCSD and King’s College London. The first is an engineered autologous vaccine approach for which pre-IND studies are now underway. The second vaccine approach incorporates a combined molecular adjuvant with leukemia associated antigens for future “off the shelf” vaccines.

http://cancer.ucsf.edu/people/profiles/gaensler_karin.3516

_______________________________________________________________________

PRESENTATIONS*UCSF authors in bold

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Pediatric Clinical Malignant Heme

Authors*: Inga Hofmann, MD, Mignon L. Loh, MD and Sarah K. Tasian, MD

Abstract #: Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper120493.htmlPresentation Date/Time: Friday, November 30, 2018, 3:30 PM-4:00 PMLocation: Section 124 -The Porch (Petco Park)Presentation Type: Trainee Session

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

_______________________________________________________________________

Enhancing Immunotherapy Efficacy for Aging Myeloma Patients Ineligible for Transplant

Authors*: Arun P Wiita, MD, PhD

Abstract #: Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper120608.htmlPresentation Date/Time: Friday, November 30, 2018, 4:25 PM-5:25 PMLocation: Room 33B (San Diego Convention Center) Presentation Type: Scientific Workshop

Wiita Research Interests: Significant effort in cancer research has been directed towards understanding the composition of the cancer genome and transcriptome. Less is known, however, about how genomic alterations and therapeutic perturbations remodel the cancer proteome, despite the fact that proteins actually carry out all biological function. To attempt to bridge this knowledge gap, the Wiita Lab uses a combination of genome engineering, chemical biology, and quantitative mass spectrometry-based proteomics to discover basic biology and new therapeutic targets in hematologic malignancies. In addition, the Wiita Lab also includes the UCSF Stephen and Nancy Grand Multiple Myeloma Translational Initiative laboratory (MMTI Lab). The MMTI Lab works with academic and industry partners to perform preclinical evaluation of new small molecules and immunotherapies across a suite of in vitro, in vivo, and patient ex vivo models, with the goal of moving compounds into the clinic to benefit myeloma patients.

http://labmed.ucsf.edu/wiita/

_______________________________________________________________________

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SATURDAY | DECEMBER 1, 2018

Platelet Biogenesis in the Lung Circulation

Authors*: Mark Looney, MD

Abstract #: SCI-22Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109544.htmlPresentation Date/Time: Saturday, December 1, 2018, 7:30 AM-9:00 AMLocation: Room 31B (San Diego Convention Center)Presentation Type: Scientific Program

Looney Research Interests: My lab is broadly focused on innate immune biology in the lung. Thematic areas include neutrophil and platelet biology as applied to a variety of pulmonary disease states including acute lung injury (ALI)/acute respiratory distress syndrome (ARDS), primary graft dysfunction after lung transplantation, and cystic fibrosis. A major interest is the application of multiphoton intravital lung microscopy as a discovery tool to aid in the study of lung biology, including novel studies on the role of the pulmonary circulation in platelet biogenesis and the hematopoietic potential of the lung. Our overall goal is to identify new mechanisms responsible for lung inflammation and injury and to develop novel therapies to combat lung disease.

http://looneylab.ucsf.edu

_______________________________________________________________________

Triple Intrathecal Therapy (Methotrexate/Hydrocortisone/Cytarabine) Does Not Improve Disease-Free Survival Versus Intrathecal Methotrexate Alone in Children with High Risk B-Lymphoblastic Leukemia: Results of Children’s Oncology Group Study AALL1131

Authors*: Wanda L. Salzer, MD, Michael J. Burke, MD, Meenakshi Devidas, PhD, Lia Gore, MD, Joanne M. Hilden, MD, Eric Larsen, MD, Karen R. Rabin, MD, PhD, Patrick A. Zweidler-McKay, MD, PhD, Michael J. Borowitz, MD, PhD, Brent Wood, MD, PhD, Nyla A. Heerema, PhD, Andrew J. Carroll, PhD, Naomi Winick, MD, William L. Carroll, MD, Elizabeth A. Raetz, MD, Mignon L. Loh, MD and Stephen P. Hunger, MD

Abstract #: 35Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116180.htmlPresentation Date/Time: Saturday, December 1, 2018: 8:30 AMLocation: Pacific Ballroom 20 (Marriott Marquis San Diego Marina)Presentation Type: Oral

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

_______________________________________________________________________

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Low Hemoglobin Increases Risk for Stroke, Kidney Disease, Elevated Estimated Pulmonary Artery Systolic Pressure, and Premature Death in Sickle Cell Disease: A Systematic Literature Review and Meta-Analysis

Authors*: Kenneth I. Ataga, MBBS, Victor R. Gordeuk, MD, I. Elaine Allen, PhD, Jennifer Colby, PharmD, Kimberly Gittings, PharmD and Irene Agodoa, MD

Abstract #: 12Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117261.htmlPresentation Date/Time: Saturday, December 1, 2018: 8:45 AMLocation: Room 25B (San Diego Convention Center)Presentation Type: Oral

Allen Research Interests: Isabel Elaine Allen expertise is in biostatistics and data science. She uses these techniques to link cognitive, clinical, imaging, and demographic data to understand the diagnosis and underlying relationships in neurodegenerative diseases and other mental and physical health issues related to cognitive decline.

http://profiles.ucsf.edu/elaine.allen

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Axicabtagene Ciloleucel (Axi-cel) CD19 Chimeric Antigen Receptor (CAR) T-Cell Therapy for Relapsed/Refractory Large B-Cell Lymphoma: Real World Experience

Authors*: Loretta J. Nastoupil, MD, Michael D. Jain, MD, PhD, Jay Y. Spiegel, MD, FRCPC, Armin Ghobadi, MD, Yi Lin, MD, PhD, Saurabh Dahiya, MD, Matthew A. Lunning, DO, Lazaros J. Lekakis, MD, Patrick M. Reagan, MD, Olalekan O. Oluwole, MBBS, MPH, Joseph P. McGuirk, DO, Abhinav Deol, MD, Alison R. Sehgal, MD, Andre Goy, MD, Brian T. Hill, MD, PhD, Charalambos Andreadis, MD, Javier Munoz, MD, Jason R. Westin, MD, Julio C. Chavez, MD, Amanda F Cashen, MD, Nora N Bennani, MD, Aaron P. Rapoport, MD, Julie M. Vose, MD, MBA, David B. Miklos, MD, PhD, Sattva S. Neelapu, MD and Frederick L. Locke, MD

Abstract #: 91Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper114152.htmlPresentation Date/Time: Saturday, December 1, 2018: 9:30 AMLocation: Pacific Ballroom 20 (Marriott Marquis San Diego Marina)Presentation Type: Oral

Andreadis Research Interests: Dr. Andreadis studies the interplay of cancer genetics and traditional pharmacogenetics (germline) as it pertains to prognosis and treatment response in patients with cancer, and especially hematologic malignancies. His clinical experience combined with his molecular epidemiology statistical expertise have been the primary drivers for this work. Another important focus of his research centers on targeted and immune therapies for lymphoma, specifically Diffuse Large B-cell Lymphoma.

http://cancer.ucsf.edu/people/profiles/andreadis_babis.3784

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Ixazomib-Lenalidomide-Dexamethasone (IRd) Consolidation Following Autologous Stem Cell Transplantation in Patients with Newly Diagnosed Multiple Myeloma: A Large Multi-Center Phase II Trial

Authors*: Ravi Vij, MBBS, Nitya Nathwani, MD, Thomas G. Martin III, MD, Mark A. Fiala, Abhinav Deol, MD, Francis K. Buadi, MB, CHB, Jonathan L Kaufman, MD, Craig C Hofmeister, MD, MPH, Tara Gregory, MD, Jesus G. Berdeja, MD, Ajai Chari, MD and Ashley E. Rosko, MD

Abstract #: 123Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109920.htmlPresentation Date/Time: Saturday, December 1, 2018: 10:00 AMLocation: Grand Hall C (Manchester Grand Hyatt San Diego)Presentation Type: Oral

Martin Research Interests: Dr. Thomas Martin is a leading expert in hematological malignancies and has been the principal investigator (PI) on over 25 MM clinical trials. His clinical research also includes translational studies designed to address the genetics of MM, the role of the microenvironment as well as discovery of biomarkers for patient selection and response to anti-MM therapeutics.

http://cancer.ucsf.edu/research/multiple-myeloma/mmti/mmti_team#martin

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Bortezomib Maintenance (BM) or Consolidation (BC) Following Aggressive Immunochemotherapy and Autologous Stem Cell Transplant (ASCT) for Untreated Mantle Cell Lymphoma (MCL): 8 Year Follow up of CALGB 50403 (Alliance)

Authors*: Lawrence D Kaplan, MD, Matthew J. Maurer, MS, Wendy Stock, MD, Nancy L. Bartlett, MD, Noreen Fulton, BS, Adam Pettinger, John C. Byrd, MD, Kristie A. Blum, MD, Ann S. LaCasce, MD, MSc, David Duane Hurd, MD, Amy S. Ruppert, MAS, PhD, Francisco J. Hernandez-Ilizaliturri, MD, John P. Leonard, MD and Bruce Cheson, MD

Abstract #: 146Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112655.htmlPresentation Date/Time: Saturday, December 1, 2018: 12:15 PMLocation: Pacific Ballroom 20 (Marriott Marquis San Diego Marina)Presentation Type: Oral

_______________________________________________________________________

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The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

Authors*: Maria Domenica Cappellini, MD, Vip Viprakasit, MD, DPhil, Ali Taher, MD PhD, Pencho Georgiev, MD, Kevin H. M. Kuo, MD, MSc, FRCPC, Thomas D. Coates, MD, Ersi Voskaridou, MD, Hong Keng Liew, Idit Pazgal-Kobrowski, Gianluca Forni, Silverio Perrotta, Abderrahim Khelif, Ashutosh Lal, MD, Antonis Kattamis, Efthymia Vlachaki, Raffaella Origa, MD, Yesim Aydinok, Mohamed Bejaoui, P. Joy Ho, Lee Ping Chew, Ping Chong Bee, Soo Min Lim, Meng-Yao Lu, Adisak Tantiworawit, Penka Ganeva, Liana Gercheva, Farrukh Shah, Ellis J. Neufeld, Abderrahmane Laadem, MD, Jeevan K. Shetty, Jun Zou, MD PhD, Dimana Miteva, PhD, Tatiana Zinger, PhD, Peter G. Linde, Matthew L. Sherman, Olivier Hermine, MD PhD, John Porter, MA MD FRCP FRCPath and Antonio Piga

Abstract #: 163Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112435.htmlPresentation Date/Time: Saturday, December 1, 2018: 2:00 PMLocation: Room 30D (San Diego Convention Center)Presentation Type: Oral

_______________________________________________________________________

Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-Risk Leukemias: A Report from the LEAP Consortium

Authors*: Yana Pikman, MD, Sarah K. Tasian, MD, Maria Luisa Sulis, MD, Todd M Cooper, DO, Melinda Pauly, MD, Kelly W. Maloney, MD, Michael J. Burke, MD, Patrick Brown, MD, Nathan Gossai, MD, Peter Cole, MD, Jennifer McNeer, MD, MS, Neerav Shukla, MD, Beth Apsel Winger, MD, PhD, Asmani A Adhav, Traci M. Blonquist, MS, Amy Conway, Andrew E. Place, MD, PhD, Lia Gore, MD, Stephen P. Hunger, MD, Katherine Janeway, MD, MMSc, Lewis B. Silverman, MD, Jeffrey W. Tyner, PhD, Marian H. Harris, MD, PhD, Mignon L. Loh, MD and Kimberly Stegmaier, MD

Abstract #: 261Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116948.htmlPresentation Date/Time: Saturday, December 1, 2018: 4:30 PMLocation: Room 7B (San Diego Convention Center)Presentation Type: Oral

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

_______________________________________________________________________

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Phlebitis after Parenteral Iron Sucrose Administration in Postpartum Women

Authors*: Julia Ramos, MD, PhD, Melissa Rosenstein, MD and Andrew Leavitt, MD

Abstract #: 1052Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper113182.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Active Calpain Promotes Fibrin Clot Contraction By Strengthening the Coupling of Fibrin-Bound αIIbβ3 to the Platelet Cytoskeleton

Authors*: Rustem I. Litvinov, Karen Pei Yi Fong, PhD, Oleg V. Kim, PhD, Kathleen I. Molnar, PhD, Paul C. Billings, PhD, Alex Sternisha, James A. Wells, PhD, John W. Weisel, PhD, William F. DeGrado, Ph.D. and Joel S. Bennett, MD

Abstract #: 1128Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper113063.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

_______________________________________________________________________

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Genetics of Factor VIII Inhibitor Development in Hemophilia Patients: Novel Statistical Approaches in the PATH Study

Authors*: Vincent P. Diego, PhD1, Marcio A. Almeida, PhD, Bernadette W. Luu, BS, MT/CLS, Karin Haack, PhD, Meera B. Chitlur, MD, Afshin Ameri, MD, Long V. Dinh, PhD, Raja Rajalingam, PhD, Jerry S. Powell, MD, John Blangero, PhD, Laura Almasy, PhD, Shelley Cole, PhD and Tom E. Howard

Abstract #: 1199Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper120021.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Raja Research Interests: Natural killer (NK) have been implicated in control and clearance of malignant and virally infected cells, regulation of adaptive immune responses, rejection of bone marrow transplants and autoimmunity. Human NK cells largely use a family of Human Leukocyte Antigen (HLA)-specific Killer cell Immunoglobulin-like Receptors (KIR) to recognize and respond to unhealthy target cells. KIR and HLA loci are both polygenic and substantially polymorphic, and map to distinct human chromosomes. KIR and HLA gene families segregate independently, yielding many individuals who express KIR receptors for which they lack HLA class I ligands, and vice versa, thus creating human diversity in the number and type of KIR-HLA inherited gene pairs, which potentially influences the health and disease status of a given individual. The goal of our research is to understand the diversity of KIR receptors, HLA ligands, and KIR-HLA gene combinations in populations and their relevance in human health and disease.

https://itl.surgery.ucsf.edu/people/director/rajalingam-raja,-phd,-d(abhi).aspx

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On-Demand Treatment of Bleeding Episodes in Patients with Hereditary Factor X Deficiency Using a High-Purity Factor X Concentrate: Data from 2 Clinical Trials and a Data-Collection Study

Authors*: James N. Huang, MD, Chioma Akanezi, BSc, PgDip and Amy Shapiro

Abstract #: 1215Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111397.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Huang Research Interests: Dr. James Huang, director of Pediatric Hematology and co-director of the Hemophilia Treatment Center at UCSF Benioff Children’s Hospital, is an expert in treating children with blood disorders such as hemophilia, thrombophilia and hemoglobinopathies including thalassemias and sickle cell disease as well as addressing issues related to bone marrow failure. His research focuses on bleeding disorders, hemoglobinopathies and stem cell transplantation to treat blood disorders. He has a particular interest in Shwachman-Diamond Syndrome, an inherited bone marrow failure syndrome associated with pancreatic dysfunction that also predisposes children to leukemia.

http://profiles.ucsf.edu/james.huang#toc-id1

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Ras-Driven B-Cell Transformation Targets Developmental Rewiring of Cytokine to Pre-B Cell Receptor Signaling

Authors*: Lai N Chan, Christian Hurtz, PhD, Huimin Geng, PhD, Franziska Auer, PhD, Zhengshan Chen, MD-PhD, Gang Xiao, PhD, Jae-Woong Lee, PhD, Kadriye Nehir Cosgun, PhD, B Hilda Ye, PhD and Markus Muschen, MD

Abstract #: 1336Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper115514.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Geng Research Interests: Dr. Geng’s research is focused on functional genomics and epigenomics of lymphoma and leukemia using computational approaches on genome-wide array and deep sequencing data, including RNA-seq, Whole Exome-Seq, ChIP-seq, miRNA-seq and DNA methylation eRRBS-seq. Applying bioinformatic methods coupled with in vitro and in vivo experiments, they are interested in identifying and evaluating new prognostic and disease-classification biomarkers and novel therapeutic targets for different forms of lymphoma and leukemia.

http://profiles.ucsf.edu/huimin.geng

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Induction Toxicities Are More Frequent in Young Adults Compared to Children Treated on the Children’s Oncology Group (COG) First Relapse B-Lymphoblastic Leukemia Clinical Trial AALL1331

Authors*: Laura Brook Hogan, MD, Teena Bhatla, MD, Sue Rheingold, MD, Lingyun Ji, PhD, Xinxin Xu, Elizabeth A. Raetz, MD, Stephen P. Hunger, MD, Mignon L. Loh, MD and Patrick Brown, MD

Abstract #: 1382Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112269.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Phase I Study of the Selinexor in Relapsed/Refractory Childhood Acute Leukemia

Authors*: Andrew E. Place, MD, PhD, Traci M. Blonquist, MS, Elliot Stieglitz, MD, Todd M Cooper, DO, Lia Gore, MD, Richard Aplenc, MD, PhD, Mignon L. Loh, MD, Melinda Pauly, MD, Rachel E. Rau, MD, Michael J. Burke, MD, Julia Etchin, PhD, A. Thomas Look, MD, Maria Luisa Sulis, MD and Lewis B. Silverman, MD

Abstract #: 1405Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116689.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Impact of Minimal Residual Disease and Achievement of Complete Remission/Complete Remission with Partial Hematologic Recovery (CR/CRh) on Overall Survival Following Treatment with Gilteritinib in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) with FLT3 Mutations

Authors*: Mark J. Levis, MD, Alexander E. Perl, MD, Jessica K. Altman, MD, Jorge E. Cortes, MD, Catherine C. Smith, MD, Maria R. Baer, MD, David F. Claxton, MD, Joseph G. Jurcic, MD, Ellen K. Ritchie, MD, Stephen A. Strickland, MD, Raoul Tibes, MD, PhD, Jason E. Hill, PhD, Matt Rosales and Erkut Bahceci, MD

Abstract #: 1458Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper110971.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Smith Research Interests: The Smith laboratory focuses on identification of therapeutic resistance mechanisms and novel treatment strategies for acute myeloid leukemia (AML), particularly AML associated with mutations in Fms-like Tyrosine Kinase-3 (FLT3). FLT3 is the most frequently mutated gene in AML, with constitutively activating FLT3 internal tandem duplication (ITD) mutations conferring a poor prognosis. They employ a prototypical “bedside to bench and back” approach to the problem of cancer drug resistance, founded on the belief that the ultimate pathway to improved cancer therapy begins with translational studies that utilize samples from patients who have undergone therapy in real time. This strategy allows them to interrogate how tumors can evolve under the selective pressure of cancer therapy and allows them to devise ways to circumvent these evolutionary adaptations.

http://cancer.ucsf.edu/people/profiles/smith_catherine.3369

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Longitudinal Monitoring of AML Tumors with High-Throughput Single-Cell DNA Sequencing Reveals Rare Clones Prognostic for Disease Progression and Therapy Response

Authors*: Dennis J. Eastburn, PhD, Christine M. McMahon, MD, Robert Durruthy-Durruthy, PhD, Martin Carroll, MD, Catherine C. Smith, MD and Alexander E. Perl, MD

Abstract #: 1476Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119869.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Smith Research Interests: The Smith laboratory focuses on identification of therapeutic resistance mechanisms and novel treatment strategies for acute myeloid leukemia (AML), particularly AML associated with mutations in Fms-like Tyrosine Kinase-3 (FLT3). FLT3 is the most frequently mutated gene in AML, with constitutively activating FLT3 internal tandem duplication (ITD) mutations conferring a poor prognosis. They employ a prototypical “bedside to bench and back” approach to the problem of cancer drug resistance, founded on the belief that the ultimate pathway to improved cancer therapy begins with translational studies that utilize samples from patients who have undergone therapy in real time. This strategy allows them to interrogate how tumors can evolve under the selective pressure of cancer therapy and allows them to devise ways to circumvent these evolutionary adaptations.

http://cancer.ucsf.edu/people/profiles/smith_catherine.3369

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Enhanced DNA Repair and Genomic Stability in HIV(+) Diffuse Large B Cell Lymphoma of Germinal Center Origin

Authors*: Alanna Maguire, PhD, MSc, Xianfeng Chen, BSc, Lee Wisner, Colleen Ramsower, Betty Glinsmann-Gibson, MS, Michael S. McGrath, MD, PhD and Lisa M. Rimsza, MD

Abstract #: 1570Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116571.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

McGrath Research Interests: My lab studies the role of macrophages in the pathogenesis of a wide variety of diseases, including cancer. The primary model systems for these studies revolved around the study of HIV-infected macrophages and their influence on the brain, as well as in lymphoma. Through the study of HIV, we have gained important insight into the pathogenesis of other non-HIV diseases that seem to operate in a similar manner to HIV infection. Since 1994, I have been PI of ACSR, a central NCI-sponsored program. Access to well-preserved tissues from patients with a wide variety of AIDS-related conditions has allowed the discovery of fundamental mechanisms of disease. Through study of ACSR specimen sets, we have discovered that HIV-infected macrophages persist in tissues long after the blood has been cleared of infectious HIV. In fact, recent evidence suggests that infected macrophages play central roles in the pathogenesis of major post-cART diseases, such as cancer and heart disease.

http://cancer.ucsf.edu/people/profiles/mcgrath_michael.3373

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Sustained Disease Control for Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma: An Updated Analysis of Juliet, a Global Pivotal Phase 2 Trial of Tisagenlecleucel

Authors*: Stephen J. Schuster, MD, Michael R. Bishop, MD, Constantine Tam, MD, PhD, Peter Borchmann, MD, Ulrich Jaeger, MD, Edmund K. Waller, MD, Harald Holte, MD, PhD, Joseph P. McGuirk, DO8, Samantha Jaglowski, MD, MPH, Kensei Tobinai, MD, PhD, Charalambos Andreadis, MD, MSCE, Isabelle Fleury, Stephan Mielke, MD, Takanori Teshima, Jason R. Westin, MD, Veronika Bachanova, MD, PhD, Stephen Ronan Foley, MD, FRCPC, P Joy Ho, MBBS(Syd) DPhil(Oxon) FRACP FRCPA FFSc(RCPA), John M. Magenau, MD, Nina D. Wagner-Johnston, MD, Koji Kato, MD, PhD, Marie Jose Kersten, MD, PhD, Koen Van Besien, MD, PhD, Jufen Chu, PhD, Aline Jary, MS, Özlem Anak, MD, Gilles Salles, MD, PhD and Richard Thomas T. Maziarz, MD

Abstract #: 1684Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper115252.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Andreadis Research Interests: Dr. Andreadis studies the interplay of cancer genetics and traditional pharmacogenetics (germline) as it pertains to prognosis and treatment response in patients with cancer, and especially hematologic malignancies. His clinical experience combined with his molecular epidemiology statistical expertise have been the primary drivers for this work. Another important focus of his research centers on targeted and immune therapies for lymphoma, specifically Diffuse Large B-cell Lymphoma.

http://cancer.ucsf.edu/people/profiles/andreadis_babis.3784

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Identifying Factors in Multiple Myeloma Controlling Response to B-Cell Maturation Antigen (BCMA)-Targeted Immunotherapy Using CRISPR-Based Functional Genomics

Authors*: Poornima Ramkumar, PhD, Jaime Leong, Meghan Seyler, Stratton J Georgoulis, Axel Hyrenius Wittsten, PhD4, Priya Choudhry, PhD, Kole Roybal, PhD, Arun P Wiita, MD, PhD and Martin Kampmann, PhD

Abstract #: 1926Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper115368.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Kampmann Research Interests: The Kampmann lab develops and uses innovative technologies to understand cellular and molecular mechanisms of human diseases, and to discover new therapeutic strategies. A major focus of our research are diseases associated with protein misfolding. We ask how cells maintain their proteins in a functional and balanced state. In human cells, this is accomplished by the proteostasis network. Our goal is to understand how this network functions, and how it is challenged and rewired in disease states. Our functional genomics technology, which integrates CRISPR/Cas9-based control of gene function and large-scale genetic interaction maps, enables us to elucidate dynamic networks and to pinpoint nodes that are potential therapeutic targets. We use biochemistry, biophysics and cell biology to “zoom in” on individual nodes of the network and to reveal their mechanism of action.

https://kampmannlab.ucsf.edu/welcome-kampmann-lab

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Cytomegalovirus Reactivation in Haploidentical Blood or Marrow Transplant (BMT) Using Post-Transplant Cyclophosphamide (PTCy) Is Associated with Higher Non-Relapse-Related Mortality, Despite Rarity of Disease

Authors*: Maria Veronica Dioverti, MD, Chiung-Yu Huang, PhD, Megan Morales, MD, Na Lu, Willa Cochran, NP, Douglas Gladstone, MD, Darin Ostrander, PhD, Robin Avery, MD, Richard J. Jones, MD, Richard F Ambinder, MD and Kieren Marr, MD

Abstract #: 2096Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119603.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Huang Research Interests: Dr. Huang’s research interest is in general biostatistics methodology and its application to the biomedical sciences. She has extensive experience in the statistical analysis of survival outcomes, recurrent events, competing risks, longitudinal measurements, missing data, and monitoring of clinical trials. In particular, she has developed novel nonparametric and semiparametric approaches for analyzing recurrent event data and panel count data under informative censoring, where a dependent terminal event, such as death, precludes further occurrence of recurrent events of interest. She also developed efficient estimation procedures to combine auxiliary subgroup survival or covariate effect information in the analysis of survival data, with the goal to improve risk prediction in cancer patients. Recently, she has developed efficient statistical methods for evaluating vaccine efficacy in clinical trials with active surveillance by incorporating information about the number of founder virus.

http://cancer.ucsf.edu/people/profiles/huang_chiungyu.8706

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Thalassemia Management Checklists: Quick Reference Guides to Reduce Disparities in the Care of Patients with Transfusion-Dependent Thalassemia

Authors*: Ashutosh Lal, MD, Sujit Sheth, MD, Sandra Gilbert, Ph.D. and Janet L. Kwiatkowski, MD

Abstract #: 2233Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109945.htmlPresentation Date/Time: Saturday, December 1, 2018, 6:15 PM-8:15 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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SUNDAY | DECEMBER 2, 2018

Related and Unrelated Donor Transplantation for β Thalassemia Major: Results of an International Survey

Authors*: Chunfu Li, MD, Vikram Mathews, MD, Biju George, DM, Soyoung Kim, Kyle Hebert, Hua Jiang, MD, Changgang Li, MD, Yiping Zhu, Daniel A Keesler, Rajni Agarwal, MD, Jaap Jan Boelens, MD, PhD, Christopher C. Dvorak, MD, Jeffery Auletta, MD, Rakesh Kumar Goyal, MBBS, MD, MRCP, Rabi Hanna, MD, Kimberly A. Kasow, DO, David Margolis, MD, Shalini Shenoy, MD, Mark C. Walters, MD and Mary Eapen, MBBS, MS

Abstract #: 308Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111152.html Presentation Date/Time: Sunday, December 2, 2018: 7:45 AMLocation: Grand Hall A (Manchester Grand Hyatt San Diego)Presentation Type: Oral

Dvorak Research Interests: Dr. Dvorak’s research interests are the supportive care aspects of pediatric hematopoietic cell transplantation (HCT), with a goal of decreasing treatment-related morbidity and mortality (especially infections), and optimizing HCT regimens for treatment of infant leukemias. Dr. Dvorak is the former Chair of the international Pediatric Blood and Marrow Transplant Consortium’s (PBMTC) Supportive Care Strategy Group and Chair of the Children’s Oncology Group (COG)’s Cancer Control & Supportive Care Committee. Among his clinical trials, he was the national PI of COG trial ACCL1131: An Open-Label Phase III Trial of Caspofungin vs. Azole Prophylaxis for Patients at High-Risk for Invasive Fungal Infections Undergoing Allogeneic HCT and the COG trial ASCT1221: A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation for Children with Juvenile Myelomonocytic Leukemia (JMML).

http://cancer.ucsf.edu/people/profiles/dvorak_christopher.3611

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Patient Satisfaction of Care in the Treatment of Vaso-Occlusive Crises: A Comparison of Emergency Department and Infusion Centers in the Escaped Study

Authors*: Nebras Abu Al Hamayel, MBBS, MPH, Jasmine Brooks, Hang Wang, PhD, Rebecca Seufert, Carlton Haywood Jr., PhD, MA, Ravi Varadhan, PhD, Mustapha Saheed, MD, Jodi Segal, MD, Chiung-Yu Huang, PhD, Joshua J. Field, MD, Steven Frymark, Jane Little, MD, Brandi Griffin, BSN, RN, Nicole Arnold, Joseph Ryan Shows, MD, Allie Piehet, BS, Marc Proudford, Derek Robertson, MBA, JD, Marcus Wallace, MD, Adrienne Kincaid, Charles Green, Lorri Burgess, and Sophie Lanzkron, MD

Abstract #: 314Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111544.htmlPresentation Date/Time: Sunday, December 2, 2018: 7:45 AMLocation: Room 28D (San Diego Convention Center)Presentation Type: Oral

Huang Research Interests: Dr. Huang’s research interest is in general biostatistics methodology and its application to the biomedical sciences. She has extensive experience in the statistical analysis of survival outcomes, recurrent events, competing risks, longitudinal measurements, missing data, and monitoring of clinical trials. In particular, she has developed novel nonparametric and semiparametric approaches for analyzing recurrent event data and panel count data under informative censoring, where a dependent terminal event, such as death, precludes further occurrence of recurrent events of interest. She also developed efficient estimation procedures to combine auxiliary subgroup survival or covariate effect information in the analysis of survival data, with the goal to improve risk prediction in cancer patients. Recently, she has developed efficient statistical methods for evaluating vaccine efficacy in clinical trials with active surveillance by incorporating information about the number of founder virus.

http://cancer.ucsf.edu/people/profiles/huang_chiungyu.8706

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Using Genomics to Define Pediatric Blood Cancers and Inform Practice

Authors*: Mignon L. Loh, MD

Abstract #: Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109553.htmlPresentation Date/Time: Sunday, December 2, 2018, 9:30 AM-11:00 AMLocation: San Diego Ballroom B (Marriott Marquis San Diego Marina)Presentation Type: Education Program

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Increased Disease Burden Among Black Children Compared to White Children with Newly Diagnosed Acute Myeloid Leukemia

Authors*: Lena E. Winestone, MD, MSHP, Kelly D Getz, PhD, Yimei Li, PhD, Evanette Burrows, MPH, Michael Scheurer, MPH, PhD, M. Monica Gramatges, MD, PhD, Jennifer J. Wilkes, MD, Tamara P. Miller, MD, MSc, Alix E. Seif, MD, Robert W. Grundmeier, MD3, Karen R. Rabin, MD, PhD, Brian T. Fisher, DO and Richard Aplenc, MD, PhD

Abstract #: 369Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper120076.html Presentation Date/Time: Sunday, December 2, 2018: 10:00 AMLocation: Room 25B (San Diego Convention Center)Presentation Type: Oral

Winestone Research Interests: Dr. Lena Winestone is clinically focused on pediatric blood and marrow transplant. Dr. Winestone’s research explores racial, ethnic, and socioeconomic disparities in access to care and outcome of leukemia and lymphoma treatment. She has studied access to care across the continuum of cancer from diagnosis and clinical trial enrollment through treatment and relapse to salvage therapies. Her research has shown that African American children with acute lymphoblastic leukemia, acute myeloid leukemia, and mature B cell lymphoma are two times more likely to present with organ failure at the time of a new diagnosis. Her ongoing work investigates access to highly specialized and complex therapies such as stem cell transplant, chimeric antigen receptor T cells, and MIBG therapy.

http://cancer.ucsf.edu/people/profiles/winestone_lena.9218

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Platelet Biogenesis in the Lung Circulation

Authors*: Mark Looney, MD

Abstract #: SCI-22Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109544.htmlPresentation Date/Time: Sunday, December 2, 2018, 9:30 AM-11:00 AMLocation: Room 31B (San Diego Convention Center)Presentation Type: Scientific Program

Looney Research Interests: My lab is broadly focused on innate immune biology in the lung. Thematic areas include neutrophil and platelet biology as applied to a variety of pulmonary disease states including acute lung injury (ALI)/acute respiratory distress syndrome (ARDS), primary graft dysfunction after lung transplantation, and cystic fibrosis. A major interest is the application of multiphoton intravital lung microscopy as a discovery tool to aid in the study of lung biology, including novel studies on the role of the pulmonary circulation in platelet biogenesis and the hematopoietic potential of the lung. Our overall goal is to identify new mechanisms responsible for lung inflammation and injury and to develop novel therapies to combat lung disease.

http://looneylab.ucsf.edu

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Germline GAB2 Mutations in Childhood Acute Lymphoblastic Leukemia

Authors*: Adam J de Smith, PhD, Geneviève Lavoie, PhD, Kyle M Walsh, PhD, Sumeet Aujla, BSc, Erica Evans, BSc, Helen M Hansen, BSc, Ivan Smirnov, PhD, Alice Y Kang, MPH, Martin Zenker, M.D., John J Ceremsak, BSc, Elliot Stieglitz, MD, Roberta McKean-Cowdin, PhD, Catherine Metayer, MD, PhD, Philippe P Roux, PhD and Joseph L Wiemels, PhD

Abstract #: 388Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119235.htmlPresentation Date/Time: Sunday, December 2, 2018: 12:45 PMLocation: Room 25B (San Diego Convention Center)Presentation Type: Oral

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Integrating Phosphoproteomics and Transcriptional Classifiers Reveals “Hidden Signaling” in Multiple Myeloma Including Differential KRAS and NRAS Mutant Effects

Authors*: Yu-Hsiu T. Lin, Gergory P. Way, PhD, Margarette C. Mariano, MS, Makeba Marcoulis, Ian Ferguson, Casey S. Greene, PhD and Arun P Wiita, MD, PhD

Abstract #: 469Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper113683.htmlPresentation Date/Time: Sunday, December 2, 2018: 4:30 PMLocation: Grand Ballroom 7 (Marriott Marquis San Diego Marina)Presentation Type: Oral

Wiita Research Interests: Significant effort in cancer research has been directed towards understanding the composition of the cancer genome and transcriptome. Less is known, however, about how genomic alterations and therapeutic perturbations remodel the cancer proteome, despite the fact that proteins actually carry out all biological function. To attempt to bridge this knowledge gap, the Wiita Lab uses a combination of genome engineering, chemical biology, and quantitative mass spectrometry-based proteomics to discover basic biology and new therapeutic targets in hematologic malignancies. In addition, the Wiita Lab also includes the UCSF Stephen and Nancy Grand Multiple Myeloma Translational Initiative laboratory (MMTI Lab). The MMTI Lab works with academic and industry partners to perform preclinical evaluation of new small molecules and immunotherapies across a suite of in vitro, in vivo, and patient ex vivo models, with the goal of moving compounds into the clinic to benefit myeloma patients.

http://labmed.ucsf.edu/wiita/

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Initial Results from a Phase 1 Clinical Study of bb21217, a Next-Generation Anti-BCMA CAR T Therapy

Authors*: Nina Shah, MD, Melissa Alsina, MD, David S Siegel, MD, PhD, Sundar Jagannath, MD, Deepu Madduri, MD, Jonathan L Kaufman, MD, Ashley Turka, Lyh Ping Lam, PharmD, RPh, Monica Massaro, MPH, Kristen Hege, MD, Fabio Petrocca, MD, Jesus G. Berdeja, MD and Noopur Raje, MD

Abstract #: 488Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116953.htmlPresentation Date/Time: Sunday, December 2, 2018: 4:45 PMLocation: Room 6B (San Diego Convention Center)Presentation Type: Oral

Shah Research Interests: Dr. Nina Shah is an expert in cellular therapy for multiple myeloma. She has developed a novel natural killer cell platform using umbilical cord blood and conducted a first-in-human clinical trial for cord blood derived natural killer cells in the setting of high dose chemotherapy and autologous stem cell transplantation. She is also interested in dendritic cell-based vaccines, adoptive T cell therapies and novel immunomodulatory combinations for myeloma.

http://cancer.ucsf.edu/people/profiles/shah_nina.8258

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Impact of Prophylaxis Usage on Bleeding Rates Among Persons with Hemophilia A: Evidence from Longitudinal Analyses in the USA

Authors*: Michael B. Nichol, PhD, Randall Curtis, MBA, Yuchen Ding, PhD, Elmar R. Aliyev, Marion A. Koerper, MD, Mimi Lou, MS, Megan M. Ullman, MA, MPH, Duc Quang Tran Jr., MD, Judith Baker, DrPH, MHSA, Brenda Riske, MBA, MS and Joanne Wu, MD, MS

Abstract #: 494Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119215.htmlPresentation Date/Time: Sunday, December 2, 2018: 4:45 PMLocation: Room 24B (San Diego Convention Center)Presentation Type: Oral

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TNF-Induced Vaso-Occlusive and Inflammatory Processes in Mice with Sickle Cell Anemia Are Abrogated By the Platelet Activation Inhibitor, Prasugrel

Authors*: Hanan Chweih, M.Sc., Juliete A.F. Silva, Ph.D., Erica M.F. Gotardo, Ph.D., Pamela L. Brito, B.Sc, Guilherme G.O. Barbosa, Ph.D., Flavia Garcia, Ph.D., Wilson A. Ferreira Jr., Ph.D., Flavia C. Leonardo, Ph.D., Carla F. Franco-Penteado, Ph.D., Fernando F. Costa, M.D., Ph.D. and Nicola Conran, PhD

Abstract #: 2354Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper118633.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Optimized Beta-Globin Expression and Enucleation from Induced Red Blood Cells for In Vitro Modeling of Sickle Cell Disease

Authors*: Tolulope O Rosanwo, Melissa Kinney, PhD, Martha A Clark, PhD, Linda T Vo, PhD, R. Grant Rowe, MD, PhD, Natasha M. Archer, MD, Matthew M Heeney, MD, Thorsten Schlaeger, PhD, Daniel E. Bauer, MD, PhD Mark D. Fleming, MD, DPhil, Manoj T Duraisingh, PhD, Stuart H. Orkin, MD, Carlo Brugnara, MD, Trista North, PhD and George Q. Daley, MD, PhD

Abstract #: 2359Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper114136.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Vo Research Interests: My research career is focused on the development of novel prognostic and predictive biomarkers and incorporation of these biomarkers into new clinical trials, working towards more effective and less toxic therapies in children and young adults with solid tumors.

http://cancer.ucsf.edu/people/profiles/vo_kieuhoa.7395

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Whole Exome Sequencing and Extended Thrombophilia Testing in Patients with Venous Thromboembolism

Authors*: Lauren Marsh Shevell, MD, MPH, Eun-Ju Lee, MD, Rahul Dhodapkar, MBBS, Daniel Dykas, BS, MBA, Andreea Popa, MD, PhD, Deqiong Ma, MD, PhD, Noffar Bar, MD5, Audrey Baluha, RN, Adrienne J. Burns, MMS, PA-C, Cassius Ilya Ochoa Chaar, MD, Anne Dupont, RN, Sean Gu, MD, PhD, Stephanie Halene, MD, Randy Luciano, MD, PhD, Rebecca Marien, RN, Natalia Neparidze, MD, Terri L Parker, MD, Xiaopan Yao, PhD, Rodney M. Camire, PhD, Eduard Ebberink, PhD, Pablo Garcia de Frutos, Kavitha Gnanasambandan, PhD, Peter P. Sayeski, PhD, James A. Huntington, PhD, Steven R. Lentz, MD, PhD, Koen Mertens, PhD, Christopher Parish, PhD, Ray Rezaie, PhD, Jean M. Connors, MD, Andrew Leavitt, MD, Allen Bale, MD and Alfred Ian Lee, MD, PhD

Abstract #: 2506Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper115529.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Application of Hyperpolarized 13C Magnetic Resonance Imaging to Detect Target Inhibition of NFkB Activation in Preclinical Patient-Derived Models of CNS Lymphoma

Authors*: Huimin Geng, PhD, Brice Tiret, Ph.D., Hua-Xin Gao, Ph.D., Cigall Kadoch, Ph.D., Ming Lu, Ph.D., Lingjing Chen, Ph.D., Ilwoo Park, Ph.D., Gerd Melkus, Ph.D., Adam Elkhaled, Ph.D., John Kurhanewicz, Ph.D., Lisa Drew, PhD, Sebastien Degorce, Ph.D., Michele Mayo, PhD, Keith Dillman, Ph.D., Rana Anjum, Ph.D., Andrew Bloecher, Ph.D., Myriam Chaumeil, Ph.D. and James Rubenstein, M.D., Ph.D.

Abstract #: 2840Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117625.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Rubenstein Research Interests: The lab of Dr. James Rubenstein, Department of Medicine, works in the field immunotherapy and cancer. Their major interests are in the identification of genetic factors associated with relapse, in tumor cell tropism to the brain, and in defining the tumor microenvironment in order to improve the anti-tumor immune response. They are simultaneously involved in leading phase I and II trials in patients, in conducting correlative studies of the immune response in patients treated with immunotherapy, and in the development of novel preclinical models to understand disease mechanisms.

https://bms.ucsf.edu/directory/faculty/james-rubenstein-md-phd

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Short Diagnosis to Treatment Interval (DTI) Is Associated with Inferior Outcome in Newly Diagnosed Patients with Mantle Cell Lymphoma, a MER/LEO and Alliance Collaboration

Authors*: Matthew J Maurer, MS, Amy S. Ruppert, MAS, PhD, Brian K. Link, MD, Lawrence D Kaplan, MD, David J Inwards, MD, Andrew L. Feldman, MD, Eric D. Hsi, MD, Adam Pettinger, Jonathon B. Cohen, MD, Peter Martin, FRCPC, MD, Brad S Kahl, MD, Nancy L. Bartlett, MD, James R. Cerhan, MD, PhD, John P. Leonard, MD and Thomas M. Habermann, MD

Abstract #: 2878Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper110558.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Long Term Follow-up of a Phase 2 Study Examining Intratumoral G100 Alone and in Combination with Pembrolizumab in Patients with Follicular Lymphoma

Authors*: Christopher R Flowers, MD, MS, Carlos Panizo, MD PhD, Iris Isufi, MD, Alex F. Herrera, MD, Craig Okada, MD, PhD, Elizabeth H. Cull, MD, Bela Kis, MD, Jorge M. Chaves, MD, Nancy L. Bartlett, MD, Weiyun Ai, MD, PhD, Luis de la Cruz-Merino, MD, PhD, Locke J. Bryan, MD, Roch Houot, MD, PhD, Kim Linton, MBChB PhD, Javier Briones, MD, Ian Chau, MD, Gottfried R. von Keudell, MD, PhD, Hailing Lu, PhD, Frank J Hsu, MD and Ahmad S Halwani, MD

Abstract #: 2892Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117932.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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5-Hydroxymethylcytosines of Circulating Cell-Free DNA and Prognosis in Diffuse Large B-Cell Lymphoma

Authors*: Brian C.-H. Chiu, PhD, Zhou Zhang, PhD2, Qiancheng You, Elizabeth Stepniak, MPH, Paige Bracci, PhD, Rudyard I Chiu, Xu Zhang, PhD, Girish Venkataraman, MD, MBBS, Sonali M. Smith, MD, Chuan He, PhD and Wei Zhang, PhD

Abstract #: 2985Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper114375.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Multiparameter Single Cell Analysis to Identify Immune Cell States That May Contribute to Successful Treatment Discontinuation in Patients with Chronic Myeloid Leukemia

Authors*: Kibeom Jang, PhD and Neil P. Shah, MD, PhD

Abstract #: 2999Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119267.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Shah Research Interests: The Shah lab is interested in advancing targeted therapeutics for hematologic malignancies through basic studies of in vitro and in vivo model systems to gain a better understanding of the critical vulnerabilities of malignant cells, and through translational/clinical studies of samples obtained from patients participating in early phase monotherapy clinical studies to identify, validate and override mechanisms of resistance to these agents.

http://shah.ucsf.edu/

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A Phase 1 Trial of Ibrutinib (IBR) and Azacitidine (AZA) for the Treatment of Higher-Risk Myelodysplastic Syndromes (HR-MDS): Updated Results of University of California Hematologic Malignancies Consortium (UCHMC) Study 1503

Authors*: Brian A. Jonas, MD, PhD, Peter T. Curtin, MD, Gary J. Schiller, MD, Deepa Jeyakumar, MD, Matthew J. Wieduwilt, MD, PhD, Mehrdad Abedi, MD, Rafael Bejar, MD, PhD, Helen Chow, MD, Leslie G. Oesterich, MD and Aaron C. Logan, MD, PhD

Abstract #: 3088Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119237.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Logan Research Interests: Dr. Aaron Logan is a member of the Adult Hematology and Blood and Marrow Transplantation program. His clinical research encompasses treatments for acute leukemias, myelodysplastic syndrome, post-transplant infectious complications, and graft-versus-host disease (GVHD). In addition to a clinical research program, Dr. Logan is Director of UCSF Hematologic Malignancies Tissue Bank (HMTB), which captures and archives tissue samples from blood cancer patients at UCSF upon diagnosis and during treatment. Dr. Logan has an independent research program that seeks to establish methods to quantify B and T cell reconstitution kinetics after myeloablative and reduced intensity conditioning (RIC) allogeneic hematopoietic cell transplantation (allo-HCT) to yield insights into the effect of conditioning intensity on recovery of diversity in the adaptive immune system, and to determine whether immunologic repertoire diversity correlates with clinical outcomes, including relapse, GVHD, infections, and mixed donor chimerism.

http://cancer.ucsf.edu/people/profiles/logan_aaron.4577

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DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia: An International Collaborative Analysis and Development of a Common Diagnostic Platform

Authors*: Maximilian Schoenung, Elliot Stieglitz, Hideki Muramatsu, MD, PhD, Norihiro Murakami, MD, Mark Hartmann, Manuel Wiesenfarth, Yusuke Okuno, MD, Ph.D, Adam B. Olshen, PhD, Christoph Plass, Mignon L. Loh, MD, Charlotte Niemeyer, MD9, Christian Flotho and Daniel B. Lipka

Abstract #: 3093Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper114918.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Novel Allosteric Inhibitors of Heat Shock Protein 70 As Agents to Probe Protein Homeostasis and Overcome Proteasome Inhibitor Resistance in Multiple Myeloma

Authors*: Ian Ferguson, Christine Lam, Margarette C. Mariano, MS, Donghui Wang, Paul Phojanakong, Sami Toumivaara, PhD, Hao Shao, PhD, Byron C Hann, MD, PhD, Jason Gestwicki, PhD and Arun P Wiita, MD, PhD

Abstract #: 3212Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117401.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Wiita Research Interests: Significant effort in cancer research has been directed towards understanding the composition of the cancer genome and transcriptome. Less is known, however, about how genomic alterations and therapeutic perturbations remodel the cancer proteome, despite the fact that proteins actually carry out all biological function. To attempt to bridge this knowledge gap, the Wiita Lab uses a combination of genome engineering, chemical biology, and quantitative mass spectrometry-based proteomics to discover basic biology and new therapeutic targets in hematologic malignancies. In addition, the Wiita Lab also includes the UCSF Stephen and Nancy Grand Multiple Myeloma Translational Initiative laboratory (MMTI Lab). The MMTI Lab works with academic and industry partners to perform preclinical evaluation of new small molecules and immunotherapies across a suite of in vitro, in vivo, and patient ex vivo models, with the goal of moving compounds into the clinic to benefit myeloma patients.

http://labmed.ucsf.edu/wiita/

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Retrospective Analysis of Modified Hyper-CVAD Therapy Combined with Proteasome Inhibition for Patients with Multiple Myeloma and Renal Insufficiency or Renal Failure

Authors*: Derek Galligan, MD, Rupa Narayan, MD, Sarah Kim, PharmD, Ann Lazar, PhD, MS, Marisela Tan, PharmD, Richard Fong, PharmD, Mimi Lo, PharmD, Nina Shah, MD, Shagun Arora, MD, Sandy W. Wong, MD, Thomas G. Martin III, MD and Jeffrey Lee Wolf, MD

Abstract #: 3236Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111625.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Wolf Research Interests: Dr. Wolf’s clinical practice is primarily focused on plasma cell disorders and his research interests are in the areas of Phase 1/2 drug development, autologous stem cell transplant (ASCT) and minimal residual disease (MRD) methodologies and their clinical application. In Phase I/2 clinical development, Dr. Wolf has a large practice supporting Phase 1/2 clinical trials evaluating new treatment modalities for myeloma, particularly first-in-class therapies. Dr. Wolf’s clinical research focuses on optimizing ASCT in the context of the new and emerging myeloma therapies. In MRD, Dr. Wolf works with collaborators evaluating technologies for MRD detection and optimizing use for practice in a clinical setting.

http://cancer.ucsf.edu/research/multiple-myeloma/mmti/mmti_team#wolf

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Cost of Care at NCI-Designated Comprehensive Cancer Centers Vs. Other Treatment Sites for Young Adults with Newly-Diagnosed Acute Lymphoblastic Leukemia (ALL)

Authors*: Julie A. Wolfson, MD, MS, Smita Bhatia, MD, MPH, Jill Ginsberg, MD, Laura Becker, MS, David Bernstein, Henry (Joe) Henk, PhD, Gary H. Lyman, Paul Nathan, MD, Diane Puccetti, MD, Jennifer J. Wilkes, MD, Lena E. Winestone, MD and Kelly Kenzik, PhD

Abstract #: 3531Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper110649.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Winestone Research Interests: Dr. Lena Winestone is clinically focused on pediatric blood and marrow transplant. Dr. Winestone’s research explores racial, ethnic, and socioeconomic disparities in access to care and outcome of leukemia and lymphoma treatment. She has studied access to care across the continuum of cancer from diagnosis and clinical trial enrollment through treatment and relapse to salvage therapies. Her research has shown that African American children with acute lymphoblastic leukemia, acute myeloid leukemia, and mature B cell lymphoma are two times more likely to present with organ failure at the time of a new diagnosis. Her ongoing work investigates access to highly specialized and complex therapies such as stem cell transplant, chimeric antigen receptor T cells, and MIBG therapy.

http://cancer.ucsf.edu/people/profiles/winestone_lena.9218

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A Novel Approach to Identifying Septic Shock (SS) in Children with Acute Lymphoblastic Leukemia (ALL) Using Pediatric Health Information System (PHIS) Data: Methods Validation and Incidence Estimation in a National Cohort

Authors*: Viviane C. Cahen, Yimei Li, PhD, Evanette Burrows, MPH, Caitlin W Elgarten, MD, Amanda M. DiNofia, MD, Jennifer J. Wilkes, MD, Lena E. Winestone, MD, Yuan-Shung V. Huang, MS, Julie C Fitzgerald, MD, M. Monica Gramatges, MD, PhD, Karen R. Rabin, MD, PhD, Brian T. Fisher, D, Kelly D Getz, PhD, Richard Aplenc, MD, PhD and Alix E. Seif, MD

Abstract #: 3597Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper114235.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Winestone Research Interests: Dr. Lena Winestone is clinically focused on pediatric blood and marrow transplant. Dr. Winestone’s research explores racial, ethnic, and socioeconomic disparities in access to care and outcome of leukemia and lymphoma treatment. She has studied access to care across the continuum of cancer from diagnosis and clinical trial enrollment through treatment and relapse to salvage therapies. Her research has shown that African American children with acute lymphoblastic leukemia, acute myeloid leukemia, and mature B cell lymphoma are two times more likely to present with organ failure at the time of a new diagnosis. Her ongoing work investigates access to highly specialized and complex therapies such as stem cell transplant, chimeric antigen receptor T cells, and MIBG therapy.

http://cancer.ucsf.edu/people/profiles/winestone_lena.9218

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Functional Status Is Associated with Outcomes after Allogeneic Stem Cell Transplantation for Older Patients with Hematologic Malignancies

Authors*: Li-Wen Huang, MD, Chiung-Yu Huang, PhD, Charalambos Andreadis, MD, Aaron C. Logan, MD, PhD, Gabriel N. Mannis, MD, Catherine C. Smith, MD, Karin L. Gaensler, MD, Thomas G. Martin III, MD, Lloyd E. Damon, MD, Michael A. Steinman, MD and Rebecca L. Olin, MD

Abstract #: 3599Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112025.htmlPresentation Date/Time: Sunday, December 2, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Olin Research Interests: Dr. Rebecca Olin specializes in adult bone marrow transplantation (BMT) and the treatment of leukemia, lymphoma, myelodysplasia and myeloproliferative disorders, and multiple myeloma. In her research, she is particularly interested in the process of decision making in cancer treatment and the impact of cancer treatment on patient quality of life. She also studies how best to treat blood cancers in older patients. http://cancer.ucsf.edu/people/profiles/olin_rebecca.3372

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UPDATE OF PRESENTATIONS FOR MONDAY, DECEMBER 3, 2018

MONDAY | DECEMBER 3, 2018

Using Genomics to Define Pediatric Blood Cancers and Inform Practice

Authors*: Mignon L. Loh, MD

Abstract #: Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109553.htmlPresentation Date/Time: Monday, December 3, 2018, 7:00 AM-8:30 AMLocation: Pacific Ballroom 21 (Marriott Marquis San Diego Marina)Presentation Type: Education Program

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease

Authors*: Elliott Vichinsky, MD, Carolyn Hoppe, MD, Jo Howard, MD, Kenneth I. Ataga, Videlis Nduba, Amal El-Beshlawy, David L. Diuguid, MD, Salam Al-Kindi, Clark Brown, Hoda Hassab, MD, Paul Telfer, Dimitris A. Tsitsikas, Selma Unal, Julie Kanter, MD, Miguel R. Abboud, Victor R. Gordeuk, MD, Joshua Lehrer-Graiwer, Claire Sherman, Margaret Tonda, PharmD, Allison Intondi, Yifah Yaron and Russell E. Ware, MD, PhD

Abstract #: 505Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper118508.htmlPresentation Date/Time: Monday, December 3, 2018: 7:00 AMLocation: Room 28D (San Diego Convention Center)Presentation Type: Oral

_______________________________________________________________________

*UCSF authors in bold

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Lgr5 Enables Positive B-Cell Selection and Tumor-Initiation in B-Cell Malignancies

Authors*: Kadriye Nehir Cosgun, PhD, Gauri Deb, MS, PhD, Xin Yang, MD, Gang Xiao, PhD, Teresa Sadras, PhD, Franziska Auer, PhD, Jaewoong Lee, PhD, Anthony Abarientos, BSc, Maurizio Mangolini, PhD, Ali Aghajanirefah, PhD, Huimin Geng, PhD, Hassan Jumaa, PhD, Andrew G. Polson, PhD, Hans Clevers, MD, PhD and Markus Muschen, MD

Abstract #: 547Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116956.htmlPresentation Date/Time: Monday, December 3, 2018: 7:00 AMLocation: Room 10 (San Diego Convention Center)Presentation Type: Oral

Geng Research Interests: Dr. Geng’s research is focused on functional genomics and epigenomics of lymphoma and leukemia using computational approaches on genome-wide array and deep sequencing data, including RNA-seq, Whole Exome-Seq, ChIP-seq, miRNA-seq and DNA methylation eRRBS-seq. Applying bioinformatic methods coupled with in vitro and in vivo experiments, they are interested in identifying and evaluating new prognostic and disease-classification biomarkers and novel therapeutic targets for different forms of lymphoma and leukemia.

http://profiles.ucsf.edu/huimin.geng

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Characterization of Novel Subtypes in B Progenitor Acute Lymphoblastic LeukemiaClinically Relevant Abstract

Authors*: Zhaohui Gu, PhD, Michelle L. Churchman, PhD, Kathryn G. Roberts, PhD, Ian Moore, MS, Xin Zhou, PhD, Joy Nakitandwe, PhD, Kohei Hagiwara, MD, Stephane Pelletier, PhD, Sebastian Gingras, PhD, Hartmut Berns, PhD, Debbie Payne-Turner, Ashley Hill, BS, Ilaria Iacobucci, PhD, Lei Shi, PhD, Stanley Pounds, PhD, Cheng Cheng, PhD, Deqing Pei, MS, Chunxu Qu, PhD, Meenakshi Devidas, PhD, Yunfeng Dai, PhD, Shalini C. Reshmi, PhD, Julie Gastier Foster, PhD, Elizabeth A. Raetz, MD, Michael J. Borowitz, MD, PhD, Brent Wood, MD, PhD, William L. Carroll, MD, Patrick Zweider McKay, MD, PhD, Karen R. Rabin, MD, PhD, Leonard A. Mattano, MD, Kelly W. Maloney, MD, Alessandro Rambaldi, MD, PhD, Orietta Spinelli, PhD, Jerald Radich, MD, Mark D. Minden, MD, PhD, Jacob M. Rowe, MD, Selina Luger, MD, Mark R. Litzow, MD, Martin S. Tallman, MD, Janis Recevskis, PhD, Yanming Zhang, Ravi Bhatia, MD, Jessica Kohlschmidt, PhD, Krzysztof Mrózek, MD, PhD, Clara D. Bloomfield, MD, Wendy Stock, MD, Steven M. Kornblau, MD, Hagop M. Kantarjian, MD, Marina Y. Konopleva, MD, PhD, William E. Evans, PharmD, Sima Jeha, MD, Ching-Hon Pui, MD, Jun J. Yang, PhD, Elisabeth M. Paietta, PhD, James R. Downing, MD, Mary V Relling, PharmD, Jinghui Zhang, Mignon L. Loh, MD, Stephen P. Hunger, MD and Charles G. Mullighan, MBBS, MD

Abstract #: 565Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111219.htmlPresentation Date/Time: Monday, December 3, 2018: 7:00 AMLocation: Room 25B (San Diego Convention Center)Presentation Type: Oral

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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A Phase 2 Study of Ruxolitinib with Chemotherapy in Children with Philadelphia Chromosome-like Acute Lymphoblastic Leukemia (INCB18424-269/AALL1521): Dose-Finding Results from the Part 1 Safety Phase

Authors*: Sarah K. Tasian, MD, Albert Assad, MD, Deborah S Hunter, PhD, Yining Du, PhD and Mignon L. Loh, MD

Abstract #: 555Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper110221.htmlPresentation Date/Time: Monday, December 3, 2018: 7:30 AMLocation: Ballroom 20A (San Diego Convention Center)Presentation Type: Oral

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Ivosidenib (AG-120) Induced Durable Remissions and Transfusion Independence in Patients with IDH1-Mutant Untreated AML: Results from a Phase 1 Dose Escalation and Expansion Study

Authors*: Gail J. Roboz, MD, Courtney D. DiNardo, MD, MSc, Eytan M. Stein, MD, Stephane de Botton, MD, PhD, Alice S. Mims, MD, Gabrielle T. Prince, MD, Jessica K. Altman, MD, Martha L. Arellano, MD, William B. Donnellan, MD, Harry P. Erba, MD, PhD, Gabriel N. Mannis, MD, Daniel A Pollyea, MD, Anthony S. Stein, MD, Geoffrey L. Uy, MD, Justin M. Watts, MD, Amir T. Fathi, MD, Hagop M. Kantarjian, MD, Martin S. Tallman, MD, Sung Choe, PhD, David Dai, PhD, Bin Fan, PhD, Hongfang Wang, PhD, Vickie Zhang, PhD, Katharine E. Yen, PhD, Stephanie M. Kapsalis, OTR, Denice Hickman, RN, Hua Liu, PhD, Samuel V. Agresta, MD, Bin Wu, PhD, Eyal C. Attar, MD and Richard M. Stone, MD

Abstract #: 561Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper110595.htmlPresentation Date/Time: Monday, December 3, 2018: 7:30 AMLocation: Seaport Ballroom F (Manchester Grand Hyatt San Diego)Presentation Type: Oral

Mannis Research Interests: Dr. Mannis is a clinical/translational investigator whose research aims to improve outcomes for patients via more personalized treatment strategies, including the study of novel immunotherapeutic approaches and molecularly targeted agents. Accordingly, his research includes a diverse array of hematologic malignancies, most commonly acute leukemias, myelodysplastic syndromes and myeloproliferative neoplasms.

http://profiles.ucsf.edu/gabriel.mannis

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Divergent Evolutionary Trajectories of Erk- and Stat5-Activating Lesions in Acute Lymphoblastic Leukemia

Authors*: Lai N Chan, Seyedmehdi Shojaee, PhD, Christian Hurtz, PhD, Franziska Auer, PhD, Zhengshan Chen, MD-PhD, Kadriye Nehir Cosgun, PhD, Huimin Geng, PhD, Teresa Sadras, PhD, Deborah L White, PhD and Markus Muschen, MD

Abstract #: 568Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper115536.htmlPresentation Date/Time: Monday, December 3, 2018: 7:45 AMLocation: Room 25B (San Diego Convention Center)Presentation Type: Oral

Geng Research Interests: Dr. Geng’s research is focused on functional genomics and epigenomics of lymphoma and leukemia using computational approaches on genome-wide array and deep sequencing data, including RNA-seq, Whole Exome-Seq, ChIP-seq, miRNA-seq and DNA methylation eRRBS-seq. Applying bioinformatic methods coupled with in vitro and in vivo experiments, they are interested in identifying and evaluating new prognostic and disease-classification biomarkers and novel therapeutic targets for different forms of lymphoma and leukemia.

http://profiles.ucsf.edu/huimin.geng

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Efficacy and Safety of 1500mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease

Authors*: Clark Brown, Carolyn Hoppe, MD, Adlette Inati, Miguel R. Abboud, Winfred Wang, MD, Robert Liem, MD, Gerald Woods, Lewis L. Hsu, Victor R. Gordeuk, MD, Connie Piccone, Richard A. Drachtman, Erica Fong, Sandra Dixon, Margaret Tonda, PharmD, Carla Washington and Joshua Lehrer-Graiwer

Abstract #: 509Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117510.htmlPresentation Date/Time: Monday, December 3, 2018: 8:00 AMLocation: Room 28D (San Diego Convention Center)Presentation Type: Oral

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IFITM3-Mediated Regulation of Cell Membrane Dynamics Is Essential for Malignant B-Cell Transformation

Authors*: Jae-Woong Lee, PhD, Huimin Geng, PhD, Derek S Dinson, Gang Xiao, PhD, Kadriye Nehir Cosgun, PhD, Lai N Chan, Zhengshan Chen, MD-PhD, Michael Farzan, MD-PhD, Jae U Jung, PhD, Arun P. Wiita, MD, PhD and Markus Muschen, MD

Abstract #: 552Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117472.htmlPresentation Date/Time: Monday, December 3, 2018: 8:15 AMLocation: Room 10 (San Diego Convention Center)Presentation Type: Oral

Wiita Research Interests: Significant effort in cancer research has been directed towards understanding the composition of the cancer genome and transcriptome. Less is known, however, about how genomic alterations and therapeutic perturbations remodel the cancer proteome, despite the fact that proteins actually carry out all biological function. To attempt to bridge this knowledge gap, the Wiita Lab uses a combination of genome engineering, chemical biology, and quantitative mass spectrometry-based proteomics to discover basic biology and new therapeutic targets in hematologic malignancies. In addition, the Wiita Lab also includes the UCSF Stephen and Nancy Grand Multiple Myeloma Translational Initiative laboratory (MMTI Lab). The MMTI Lab works with academic and industry partners to perform preclinical evaluation of new small molecules and immunotherapies across a suite of in vitro, in vivo, and patient ex vivo models, with the goal of moving compounds into the clinic to benefit myeloma patients.

http://labmed.ucsf.edu/wiita/

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Using Administrative Data to Identify Relapse and Hematopoietic Stem Cell Transplantation (HSCT) in Children with Acute Lymphoblastic Leukemia (ALL): Validation at Two Centers and Incidence Estimation in a National Cohort

Authors*: Viviane C. Cahen, Yimei Li, PhD, Caitlin W Elgarten, MD, Amanda M. DiNofia, MD, Jennifer J. Wilkes, MD, Lena E. Winestone, MD, Yuan-Shung V. Huang, MS, M. Monica Gramatges, MD, PhD, Karen R. Rabin, MD, PhD, Brian T. Fisher, DO, Kelly D Getz, PhD, Richard Aplenc, MD, PhD and Alix E. Seif, MD

Abstract #: 624Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper114228.htmlPresentation Date/Time: Monday, December 3, 2018: 8:15 AMLocation: Room 24B (San Diego Convention Center)Presentation Type: Oral

Winestone Research Interests: Dr. Lena Winestone is clinically focused on pediatric blood and marrow transplant. Dr. Winestone’s research explores racial, ethnic, and socioeconomic disparities in access to care and outcome of leukemia and lymphoma treatment. She has studied access to care across the continuum of cancer from diagnosis and clinical trial enrollment through treatment and relapse to salvage therapies. Her research has shown that African American children with acute lymphoblastic leukemia, acute myeloid leukemia, and mature B cell lymphoma are two times more likely to present with organ failure at the time of a new diagnosis. Her ongoing work investigates access to highly specialized and complex therapies such as stem cell transplant, chimeric antigen receptor T cells, and MIBG therapy. http://cancer.ucsf.edu/people/profiles/winestone_lena.9218

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Allogeneic Hematopoietic Stem Cell Transplantation (alloHSCT) for Children and Young Adults with T-Cell Acute Lymphoblastic Leukemia (T-ALL) Treated at Investigator Discretion: A Report from Children’s Oncology Group (COG) AALL0434

Authors*: Stuart S. Winter, MD, Meenakshi Devidas, PhD, Brent Wood, MD, PhD, Zhiguo Chen, MS, Natia Esiashvili, MD, Barbara L Asselin, MD, Mignon L. Loh, MD, Karen R. Rabin, MD, PhD, Elizabeth A. Raetz, MD, Patrick A. Zweidler-McKay, MD, PhD, Kirk R. Schultz, MD, Naomi Winick, MD, William L. Carroll, MD, Stephen P. Hunger, MD and Kimberly P. Dunsmore, MD

Abstract #: 659Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112146.htmlPresentation Date/Time: Monday, December 3, 2018: 11:30 AMLocation: Room 6B (San Diego Convention Center)Presentation Type: Oral

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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CD25-Dependent Feedback Control of the B-Cell Receptor and Its Oncogenic Mimics in B-Cell Malignancies

Authors*: Jae-Woong Lee, PhD, Kohei Kume, PhD, Zhengshan Chen, MD-PhD, Gang Xiao, PhD, Kadriye Nehir Cosgun, PhD, Lai N Chan, Chun-Wei Chen, PhD, Raju Pillai, MD, Wing C Chan, MD, Stephen J Forman, Larry W Kwak, MD, PhD, Francesca Zammarchi, PhD, Patrick Van Berkel, PhD, David M Weinstock, MD, Ari M Melnick, MD, Vu N Ngo, PhD, Huimin Geng, PhD, Selina Luger, MD, Mark R. Litzow, MD, Alexandre Belot, MD-PhD, Gulbu Uzel, MD, Michael T McManus, PhD, Elisabeth M. Paietta, PhD, Eric Meffre, PhD and Markus Muschen, MD

Abstract #: 776Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117553.htmlPresentation Date/Time: Monday, December 3, 2018: 3:00 PMLocation: Room 28D (San Diego Convention Center)Presentation Type: Oral

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Safety, Efficacy, Pharmacokinetic (PK) and Biomarker Analyses of BCL2 Inhibitor Venetoclax (Ven) Plus MDM2 Inhibitor Idasanutlin (idasa) in Patients (pts) with Relapsed or Refractory (R/R) AML: A Phase Ib, Non-Randomized, Open-Label Stud

Authors*: Naval G. Daver, MD, Daniel A Pollyea, MD, Jacqueline S. Garcia, Brian A. Jonas, MD, PhD, Karen W.L. Yee, MD, Pierre Fenaux, Sarit Assouline, MD, Norbert Vey, MD, Rebecca Olin, Gail J. Roboz, MD, Agostino Tafuri, Giuseppe Visani, Whitney Kirschbrown, Cherie Green, Connie Ma, Monique Dail, Jue Wang, Marion G Ott1, Mehrdad Mobasher, MD, MPH, Patrick Phuong, PharmD, Wan-Jen Hong, MD, Marina Y. Konopleva, MD, PhD and Michael Andreeff, MD, PhD

Abstract #: 767Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116013.htmlPresentation Date/Time: Monday, December 3, 2018: 3:45 PMLocation: Seaport Ballroom F (Manchester Grand Hyatt San Diego)Presentation Type: Oral

Olin Research Interests: Dr. Rebecca Olin specializes in adult bone marrow transplantation (BMT) and the treatment of leukemia, lymphoma, myelodysplasia and myeloproliferative disorders, and multiple myeloma. In her research, she is particularly interested in the process of decision making in cancer treatment and the impact of cancer treatment on patient quality of life. She also studies how best to treat blood cancers in older patients.

http://cancer.ucsf.edu/people/profiles/olin_rebecca.3372

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A Five Fold Decrease in Admissions for Uncomplicated Vaso-Occlusive Crisis and Other Benefits from Care in Infusion Clinics: Results from the Escaped Trial

Authors*: Sophie Lanzkron, MD, Jane Little, MD, Joshua J. Field, MD, Carlton Haywood Jr., PhD, MA, Ravi Varadhan, PhD, Mustapha Saheed, MD, Marc Proudford, Derek Robertson, MBA, JD, Adrienne Kincaid, Lorri Burgess, Charles Green, Hang Wang, PhD, Rebecca Seufert, Jasmine Brooks, Allie Piehet, Brandi Griffin, BSN, RN, Nicole Arnold, Steven Frymark, Chiung- Yu Huang, PhD, Marcus Wallace, MD, Nebras Abu Al Hamayel, MBBS, MPH and Jodi Segal, MD

Abstract #: 853Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111484.htmlPresentation Date/Time: Monday, December 3, 2018: 4:30 PMLocation: Room 25B (San Diego Convention Center)Presentation Type: Oral

Huang Research Interests: Dr. Huang’s research interest is in general biostatistics methodology and its application to the biomedical sciences. She has extensive experience in the statistical analysis of survival outcomes, recurrent events, competing risks, longitudinal measurements, missing data, and monitoring of clinical trials. In particular, she has developed novel nonparametric and semiparametric approaches for analyzing recurrent event data and panel count data under informative censoring, where a dependent terminal event, such as death, precludes further occurrence of recurrent events of interest. She also developed efficient estimation procedures to combine auxiliary subgroup survival or covariate effect information in the analysis of survival data, with the goal to improve risk prediction in cancer patients. Recently, she has developed efficient statistical methods for evaluating vaccine efficacy in clinical trials with active surveillance by incorporating information about the number of founder virus.

http://cancer.ucsf.edu/people/profiles/huang_chiungyu.8706

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PRC2 Inactivation Induces Resistance to Chemotherapy-Induced Apoptosis By Upregulating the TRAP1 Mitochondrial Chaperone in T-ALL

Authors*: Kimberly Bodaar, MD, Ingrid M. Aries, PhD, MSc, Salmaan Karim, Triona Ni Chonghaile, PhD, BSc, Melissa A. Burns, MD, Laura Hinze, Maren Pfirrmann, James Degar, Jack Landrigan, Sebastian Balbach, Sofie Peirs, PhD, Bjorn Menten, PhD, Kristen E. Stevenson, MS, Donna S Neuberg, ScD, Meenakshi Devidas, PhD, Mignon L. Loh, MD, Stephen P. Hunger, MD, David T Teachey, MD, Karen R. Rabin, MD, PhD, Stuart S. Winter, MD, Kimberly P. Dunsmore, MD, Brent Wood, MD, PhD, Lewis B. Silverman, MD, Stephen E. Sallan, MD, Pieter Van Vlierberghe, PhD, Stuart H. Orkin, MD, Birgit Knoechel, MD PhD, Anthony Letai, MD, PhD and Alejandro Gutierrez, MD

Abstract #: 889Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper113518.htmlPresentation Date/Time: Monday, December 3, 2018: 4:30 PMLocation: Room 29C (San Diego Convention Center)Presentation Type: Oral

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Glucocorticoids Paradoxically Induce Intrinsic Steroid Resistance through a STAT5-Mediated Survival Mechanism in T-Cell Acute Lymphoblastic Leukemia

Authors*: Lauren K. Meyer, Benjamin Huang, MD, Ritu Roy, PhD, Aaron Hechmer, PhD, Anica Wandler, PhD, Cristina Delgado-Martin, PhD, Adam B. Olshen, PhD, Terzah M. Horton, MD, PhD, David T Teachey, MD, Kevin M. Shannon, MD and Michelle L. Hermiston, MD, PhD

Abstract #: 913Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119930.htmlPresentation Date/Time: Monday, December 3, 2018: 4:30 PMLocation: Room 11B (San Diego Convention Center)Presentation Type: Oral

Hermiston Research Interests: Dr. Hermiston’s research interests are focused on defining the signaling networks involved in the development of lymphoid malignancies, including leukemia and lymphoma. Her team also studies how these signaling networks mediate chemotherapy resistance and how targeted therapies may restore chemosensitivity.

http://profiles.ucsf.edu/michelle.hermiston

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Fibrinogen in Neurologic Diseases: Mechanism, Imaging, and Therapeutics

Authors*: Katerina Akassoglou, PhD

Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109525.htmlPresentation Date/Time: Monday, December 3, 2018, 4:30 PM-6:30 PMLocation: Room 30D (San Diego Convention Center)Presentation Type: Special Scientific Symposia

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Bcor Mutations Cooperate with Tet2 Deficiency to Promote RAS Transformation of Mouse Hematopoietic Progenitors

Authors*: Iman Fares, PhD, Rahul S. Vedula, MD, Shabbir M. Vahanvaty, Christopher S Waters, Marlise R. Luskin, MD, Priti Kumari, M.S., Marie McConkey, PhD, Anne Charles, Kevin Copson, MA, Gregory A. Abel, MD, MPH, Richard M. Stone, MD, Benjamin L. Ebert, MD, PhD and R. Coleman Lindsley, MD, PhD

Abstract #: 888Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper120096.htmlPresentation Date/Time: Monday, December 3, 2018: 5:45 PMLocation: Room 10 (San Diego Convention Center)Presentation Type: Oral

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Updated Phase 1 Results of Zuma-3: Kte-C19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia

Authors*: William G. Wierda, MD, PhD, Michael R. Bishop, MD, Olalekan O. Oluwole, MBBS, MPH, Aaron C. Logan, MD, PhD, Maria R. Baer, MD, William B. Donnellan, MD, Kristen M. O’Dwyer, MD, Januario E. Castro, MD, Gary J. Schiller, MD, Houston Holmes, MD, Mehrdad Abedi, MD, Armin Ghobadi, MD, Martha L. Arellano, MD, Adriana K. Malone, MD, John M. Pagel, MD, PhD, Armen Mardiros, PhD, Tong Shen, MS, Remus Vezan, MD, PhD, Rajul K. Jain, MD and Bijal D. Shah, MD

Abstract #: 897Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper111531.htmlPresentation Date/Time: Monday, December 3, 2018: 5:00 PMLocation: Room 6A (San Diego Convention Center)Presentation Type: Oral

Logan Research Interests: Dr. Aaron Logan is a member of the Adult Hematology and Blood and Marrow Transplantation program. His clinical research encompasses treatments for acute leukemias, myelodysplastic syndrome, post-transplant infectious complications, and graft-versus-host disease (GVHD). In addition to a clinical research program, Dr. Logan is Director of UCSF Hematologic Malignancies Tissue Bank (HMTB), which captures and archives tissue samples from blood cancer patients at UCSF upon diagnosis and during treatment. Dr. Logan has an independent research program that seeks to establish methods to quantify B and T cell reconstitution kinetics after myeloablative and reduced intensity conditioning (RIC) allogeneic hematopoietic cell transplantation (allo-HCT) to yield insights into the effect of conditioning intensity on recovery of diversity in the adaptive immune system, and to determine whether immunologic repertoire diversity correlates with clinical outcomes, including relapse, GVHD, infections, and mixed donor chimerism.

http://cancer.ucsf.edu/people/profiles/logan_aaron.4577

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Children’s Oncology Group (COG) AALL0434: Successful Disease Control without Cranial Radiation in Newly Diagnosed T Lymphoblastic Lymphoma (T-LL)

Authors*: Robert James Hayashi, Stuart S. Winter, MD, Kimberly P. Dunsmore, MD, Meenakshi Devidas, PhD, Brent Wood, MD, PhD, Michelle L. Hermiston, MD, PhD, David T Teachey, MD, Sherrie L. Perkins, MD, PhD, Rodney R. Miles, MD, PhD, Elizabeth A. Raetz, MD, Mignon L. Loh, MD, Naomi Winick, MD, William L. Carroll, MD, Stephen P. Hunger, MD, Megan S. Lim, M.D., Ph.D., Catherine M. Bollard, MD and Thomas G. Gross, MD, PhD

Abstract #: 1000Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper113481.htmlPresentation Date/Time: Monday, December 3, 2018: 7:00 PMLocation: Room 6F (San Diego Convention Center)Presentation Type: Oral

Hermiston Research Interests: Dr. Hermiston’s research interests are focused on defining the signaling networks involved in the development of lymphoid malignancies, including leukemia and lymphoma. Her team also studies how these signaling networks mediate chemotherapy resistance and how targeted therapies may restore chemosensitivity.

http://profiles.ucsf.edu/michelle.hermiston

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Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Authors*: Franco Locatelli, Mark C. Walters, MD, Janet L. Kwiatkowski, MD, John Porter, MA MD FRCP FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD, PhD, Ashutosh Lal, MD, Adrian J. Thrasher, MD, PhD, FMedSci, Evangelia Yannaki, MD, Heidi Elliot, Ge Tao, PhD, Mohammed Asmal, MD, PhD and Alexis A. Thompson, MD, MPH

Abstract #: 1025Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112667.htmlPresentation Date/Time: Monday, December 3, 2018: 7:15 PMLocation: Room 6B (San Diego Convention Center)Presentation Type: Oral

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Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 StudyClinically Relevant Abstract

John F. Tisdale, MD, Julie Kanter, MD, Markus Y. Mapara, MD, PhD, Janet L. Kwiatkowski, MD, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey Jr., Weiliang Shi, PhD, Jean-Antoine Ribeil, MD, PhD, Mohammed Asmal, MD, PhD, Alexis A. Thompson, MD, MPH and Mark C. Walters, MD

Abstract #: 1026Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper113480.htmlPresentation Date/Time: Monday, December 3, 2018: 7:30 PMLocation: Room 6B (San Diego Convention Center)Presentation Type: Oral

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The Potential of Plant-Based Compounds As Iron Chelators

Authors*: Sarah Lane, Farzam Viand, Kayla Bolduc, Juergen Ehlting, MSc, PhD and Patrick B Walter, MSc, PhD

Abstract #: 3631Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117528.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Upregulation of Bcl-2 Confers Resistance to FLT3 Inhibition in FLT3-ITD AML with Secondary Acquired Mutations

Authors*: Kotoko Yamatani, MD, Yoko Tabe, MD, PhD, Kaori Saito, Haeun Yang, Yuko Murakami-Tonami, MD, PhD, Koya Suzuki, PhD, Weiguo Zhang, MD, PhD, Sonoko Kinjo, PhD, Kazuho Ikeo, PhD, Kaoru Mogushi, PhD, Masaki Hosoya, PhD, Shigeo Yamaguchi, MD.PhD, Hironori Harada, MD, PhD, Takashi Miida, MD, PhD, Neil P. Shah, MD, PhD, Marina Y. Konopleva, MD, PhD, Yoshihide Hayashizaki, MD, PhD and Michael Andreeff, MD, PhD

Abstract #: 3944Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper109957.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Shah Research Interests: The Shah lab is interested in advancing targeted therapeutics for hematologic malignancies through basic studies of in vitro and in vivo model systems to gain a better understanding of the critical vulnerabilities of malignant cells, and through translational/clinical studies of samples obtained from patients participating in early phase monotherapy clinical studies to identify, validate and override mechanisms of resistance to these agents.

http://shah.ucsf.edu/

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Significant In Vivo Sensitivity to Aurora Kinase Inhibition in TCF3-Hlf rearranged Acute Lymphoblastic Leukemia

Authors*: Bill H. Chang, MD, PhD, Jessica Leonard, MD, Joelle Wolf, Michelle Degnin, BS, Kyle Lenz, MD, Beth Wilmot, PhD, Charles G. Mullighan, MBBS, MD, Mignon L. Loh, MD, Stephen P. Hunger, MD, Jeffrey W. Tyner, PhD and Brian J. Druker, MD

Abstract #: 4026Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper115706.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Loh Research Interests: The Loh lab has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. Their work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, Dr. Loh established JMML CLIA molecular diagnostic testing, which is now utilized as standard testing for patients suspected of having JMML. Dr. Loh is currently Chair of the Children’s Oncology Group (COG) ALL committee starting in April 2015 and responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults.

http://cancer.ucsf.edu/people/profiles/loh_mignon.3407

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Low-Dose Lenalidomide Maintenance after Induction Therapy in Older Patients with Primary CNS Lymphoma

Authors*: Khoan Vu, MD, James Rubenstein, MD, PhD, Gabriel N. Mannis, MD, Jimmy Hwang, PhD and Huimin Geng, PhD

Abstract #: 4230Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112767.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Geng Research Interests: Dr. Geng’s research is focused on functional genomics and epigenomics of lymphoma and leukemia using computational approaches on genome-wide array and deep sequencing data, including RNA-seq, Whole Exome-Seq, ChIP-seq, miRNA-seq and DNA methylation eRRBS-seq. Applying bioinformatic methods coupled with in vitro and in vivo experiments, they are interested in identifying and evaluating new prognostic and disease-classification biomarkers and novel therapeutic targets for different forms of lymphoma and leukemia.

http://profiles.ucsf.edu/huimin.geng

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Comparison of the Transcriptomic Signature of Pediatric Vs. Adult CML and Normal Bone Marrow Stem Cells

Authors*: Hee-Don Chae, PhD, Lara C. Murphy, MD, Michele Donato, PhD, Alex G. Lee, PhD, E. Alejandro Sweet-Cordero, MD, PhD, Parveen Abidi, PhD, Henrique Bittencourt, MD, PhD, Norman J. Lacayo, MD, Gary Van Houten Dahl, MD, Catherine Aftandilian, MD, Kara L. Davis, DO, Min Huang, PhD, Nathan Sumarsono, Michele Redell, MD, PhD, Cecilia H. Fu, MD, I-Ming L. Chen, DVM, MS, Todd A Alonzo, PhD, Elizabeth A. Eklund, MD, Jason R. Gotlib, MD, MS, Purvesh Khatri, PhD, Nobuko Hijiya, MD and Kathleen M. Sakamoto, MD, PhD

Abstract #: 4246Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119974.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Sweet-Cordero Research Interests: My lab works to identify novel therapeutic approaches for cancer that target the genetic mutations and altered signaling networks specific to cancer cells. We use functional genomics applied to mouse and human systems (genetically engineered models, patient derived xenografts) to understand the transcriptional networks that regulate the outcome of specific oncogenic mutations and to understand how cancers become resistant to chemotherapy. We have two primary disease interests: lung cancer and pediatric sarcomas. Our lab has identified novel regulators of chemoresistance in lung cancer. We have used functional genomics in mouse and human models to identify a novel role for Wt1 in mediating KRAS-driven oncogenesis. We have identified and characterized the role of tumor-propagating cells in NSCLC and identified a key role for Notch3 as a self-renewal pathway in mouse and human NSCLC. In our sarcoma work, we are interested in mechanisms driving osteosarcoma and Ewing sarcoma progression.

http://cancer.ucsf.edu/people/profiles/sweetcordero_alejandro.8106

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Updated 18-Month Results from Dasfree: A Study Evaluating Dasatinib Discontinuation in Patients (Pts) with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) and Deep Molecular Response (DMR)

Authors*: Neil P. Shah, MD, PhD, Jose Valentín García Gutiérrez, MD, PhD, Antonio Jiménez-Velasco, MD, Sarah Larson, MD, Susanne Saussele, MD, Delphine Rea, MD, François-Xavier Mahon, MD, PhD, Moshe Yair Levy, MD, María Teresa Gómez-Casares, MD, PhD, Fabrizio Pane, MD, Franck-Emmanuel Nicolini, MD, PhD, Michael J. Mauro, MD, Oumar Sy, PhD, Patricia Martin Regueira, MD and Jeffrey H. Lipton, MD, PhD

Abstract #: 4253Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper116563.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Shah Research Interests: The Shah lab is interested in advancing targeted therapeutics for hematologic malignancies through basic studies of in vitro and in vivo model systems to gain a better understanding of the critical vulnerabilities of malignant cells, and through translational/clinical studies of samples obtained from patients participating in early phase monotherapy clinical studies to identify, validate and override mechanisms of resistance to these agents.

http://shah.ucsf.edu/

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Meta-Analysis Illustrates Role of Interferon-γ Signaling in Multiple Myeloma Pathogenesis

Authors*: Jihad Aljabban, MMSc, Nabeal Aljabban, Mohamad Mukhtar, Saad Syed, Ross Wanner, Francesca Cottini, MD, Tiffany Hughes, PhD, Kareem Saleh,, Evan C Catton, Taylor Gonzalez, Maryam Panahiazar, PhD, Dexter Hadley, MD, PhD and Don M. Benson Jr., MD, PhD

Abstract #: 4510Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper112265.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Disparities in Cancer-Related Mortality and Long-Term Survival in Adolescent and Young Adults with Hodgkin Lymphoma: A Population-Level Analysis across the United States

Authors*: Justine M. Kahn, MD, MSc, Theresa H.M. Keegan, PhD, MS, Elysia Alvarez, MD, MPH, Lori S Muffly, MD, Helen Parsons, Lena E. Winestone, MD and Archie Bleyer, M.D.

Abstract #: 4866Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper119382.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

Winestone Research Interests: Dr. Lena Winestone is clinically focused on pediatric blood and marrow transplant. Dr. Winestone’s research explores racial, ethnic, and socioeconomic disparities in access to care and outcome of leukemia and lymphoma treatment. She has studied access to care across the continuum of cancer from diagnosis and clinical trial enrollment through treatment and relapse to salvage therapies. Her research has shown that African American children with acute lymphoblastic leukemia, acute myeloid leukemia, and mature B cell lymphoma are two times more likely to present with organ failure at the time of a new diagnosis. Her ongoing work investigates access to highly specialized and complex therapies such as stem cell transplant, chimeric antigen receptor T cells, and MIBG therapy.

http://cancer.ucsf.edu/people/profiles/winestone_lena.9218

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A Phase II Study of Pegylated Asparaginase, Cyclophosphamide, Rituximab, and Dasatinib Added to the UCSF 8707 (Linker 4-drug) Regimen with Liposomal Cytarabine CNS Prophylaxis for Adults with Newly Diagnosed Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LBL): University of California Hematologic Malignancies Consortium Study (UCHMC) 1401

Authors*: Matthew J. Wieduwilt, MD, PhD, Brian A. Jonas, MD, Gary J. Schiller, MD, Lin Liu, PhD, Carolyn Mulroney, MD, Gabriel N. Mannis, MD, Dimitrios Tzachanis, MD, PhD, Januario E. Castro, MD, Edward D Ball, MD, Peter T. Curtin, MD, Charalambos Andreadis, MD, Aaron C. Logan, MD, PhD, Jesika Reiner, MPH and Lloyd E. Damon, MD

Abstract #: 4018 Abstract link: https://ash.confex.com/ash/2018/webprogram/Paper117469.htmlPresentation Date/Time: Monday, December 3, 2018, 6:00 PM-8:00 PMLocation: Hall GH (San Diego Convention Center)Presentation Type: Poster

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Weiyun Ai, MD2892 Long Term Follow-up of a Phase 2 Study Examining Intratumoral G100 Alone and in Combination with Pembrolizumab in Patients with Follicular Lymphoma

I. Ellen Allen, PhD12 Low Hemoglobin Increases Risk for Stroke, Kidney Disease, Elevated Estimated Pulmonary Artery Systolic Pressure, and Premature Death in Sickle Cell Disease: A Systematic Literature Review and Meta-Analysis

Charalambos Andreadis, MD91 Axicabtagene Ciloleucel (Axi-cel) CD19 Chimeric Antigen Receptor (CAR) T-Cell Therapy for Relapsed/Refractory Large B-Cell Lymphoma: Real World Experience

1684 Sustained Disease Control for Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma: An Updated Analysis of Juliet, a Global Pivotal Phase 2 Trial of Tisagenlecleucel

Guilherme G. O. Barbosa, PhD2354 TNF-Induced Vaso-Occlusive and Inflammatory Processes in Mice with Sickle Cell Anemia Are Abrogated By the Platelet Activation Inhibitor, Prasugrel

Paige Bracci, PhD2985 5-Hydroxymethylcytosines of Circulating Cell-Free DNA and Prognosis in Diffuse Large B-Cell Lymphoma

Lloyd E. Damon, MD4018 A Phase II Study of Pegylated Asparaginase, Cyclophosphamide, Rituximab, and Dasatinib Added to the UCSF (Linker 4-drug) Regimen with Liposomal Cytarabine CNS Prophylaxis for Adults with Newly Diagnosed Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LBL): University of California Hematologic Malignancies Consortium Study (UCHMC) 1401

Christopher C. Dvorak308 Related and Unrelated Donor Transplantation for β Thalassemia Major: Results of an International Survey

Karin L. Gaensler— TriLeukeVax: A Next Generation Autologous AML Vaccine for Transplant Ineligible Older Patients

SUMMARY OF ABSTRACTS BY FACULTY MEMBER

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Huimin Geng, PhD1336 Ras-Driven B-Cell Transformation Targets Developmental Rewiring of Cytokine to Pre-B Cell Receptor Signaling

568 Divergent Evolutionary Trajectories of Erk- and Stat5-Activating Lesions in Acute Lymphoblastic Leukemia

Michelle L. Herminston, MD1000 Children’s Oncology Group (COG) AALL0434: Successful Disease Control without Cranial Radiation in Newly Diagnosed T Lymphoblastic Lymphoma (T-LL)

Carolyn Hoppe, MD509 Efficacy and Safety of 1500mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease

James N Huang, MD1215 On-Demand Treatment of Bleeding Episodes in Patients with Hereditary Factor X Deficiency Using a High-Purity Factor X Concentrate: Data from 2 Clinical Trials and a Data-Collection Study

Chiung-Yu Huang, PhD2096 Cytomegalovirus Reactivation in Haploidentical Blood or Marrow Transplant (BMT) Using Post-Transplant Cyclophosphamide (PTCy) Is Associated with Higher Non-Relapse-Related Mortality, Despite Rarity of Disease

314 Patient Satisfaction of Care in the Treatment of Vaso-Occlusive Crises: A Comparison of Emergency Department and Infusion Centers in the Escaped Study

Martin Kampmann, PhD1926 Identifying Factors in Multiple Myeloma Controlling Response to B-Cell Maturation Antigen (BCMA)-Targeted Immunotherapy Using CRISPR-Based Functional Genomics

Lawrence D. Kaplan, MD146 Bortezomib Maintenance (BM) or Consolidation (BC) Following Aggressive Immunochemotherapy and Autologous Stem Cell Transplant (ASCT) for Untreated Mantle Cell Lymphoma (MCL): 8 Year Follow up of CALGB 50403 (Alliance)

2878 Short Diagnosis to Treatment Interval (DTI) Is Associated with Inferior Outcome in Newly Diagnosed Patients with Mantle Cell Lymphoma, a MER/LEO and Alliance Collaboration

Marion A. Koerper, MD494 Impact of Prophylaxis Usage on Bleeding Rates Among Persons with Hemophilia A: Evidence from Longitudinal Analyses in the USA

Ashutosh Lal, MD163 The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

2233 Thalassemia Management Checklists: Quick Reference Guides to Reduce Disparities in the Care of Patients with Transfusion-Dependent Thalassemia

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Andrew Leavitt, MD1052 Phlebitis after Parenteral Iron Sucrose Administration in Postpartum Women

2506 Whole Exome Sequencing and Extended Thrombophilia Testing in Patients with Venous Thromboembolism

Aaron Logan, MD, PhD3088 A Phase 1 Trial of Ibrutinib (IBR) and Azacitidine (AZA) for the Treatment of Higher-Risk Myelodysplastic Syndromes (HR-MDS): Updated Results of University of California Hematologic Malignancies Consortium (UCHMC) Study 1503

Mignon L. Loh, MD— Pediatric Clinical Malignant Heme

35 Triple Intrathecal Therapy (Methotrexate/Hydrocortisone/Cytarabine) Does Not Improve Disease-Free Survival Versus Intrathecal Methotrexate Alone in Children with High Risk B-Lymphoblastic Leukemia: Results of Children’s Oncology Group Study AALL1131

261 Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-Risk Leukemias: A Report from the LEAP Consortium

1382 Induction Toxicities Are More Frequent in Young Adults Compared to Children Treated on the Children’s Oncology Group (COG) First Relapse B-Lymphoblastic Leukemia Clinical Trial AALL1331

1405 Phase I Study of the Selinexor in Relapsed/Refractory Childhood Acute Leukemia

— Using Genomics to Define Pediatric Blood Cancers and Inform Practice

3093 DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia: An International Collaborative Analysis and Development of a Common Diagnostic Platform

— Using Genomics to Define Pediatric Blood Cancers and Inform Practice

565 Characterization of Novel Subtypes in B Progenitor Acute Lymphoblastic LeukemiaClinically Relevant Abstract

555 A Phase 2 Study of Ruxolitinib with Chemotherapy in Children with Philadelphia Chromosome-like Acute Lymphoblastic Leukemia (INCB18424-269/AALL1521): Dose-Finding Results from the Part 1 Safety Phase

4026 Significant In Vivo Sensitivity to Aurora Kinase Inhibition in TCF3-Hlf rearranged Acute Lymphoblastic Leukemia

Mark Looney, MDSCI-22 Platelet Biogenesis in the Lung Circulation

Gabriel Mannis, MD561 Ivosidenib (AG-120) Induced Durable Remissions and Transfusion Independence in Patients with IDH1-Mutant Untreated AML: Results from a Phase 1 Dose Escalation and Expansion Study

Thomas G. Martin, MD123 Ixazomib-Lenalidomide-Dexamethasone (IRd) Consolidation Following Autologous Stem Cell Transplantation in Patients with Newly Diagnosed Multiple Myeloma: A Large Multi-Center Phase II Trial

Michael S. McGrath, PhD1570 Enhanced DNA Repair and Genomic Stability in HIV(+) Diffuse Large B Cell Lymphoma of Germinal Center Origin

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Rebecca L. Olin, MD3599 Functional Status Is Associated with Outcomes after Allogeneic Stem Cell Transplantation for Older Patients with Hematologic Malignancies

Rajalingam Raja, PhD1199 Genetics of Factor VIII Inhibitor Development in Hemophilia Patients: Novel Statistical Approaches in the PATH Study

James Rubenstein, MD2840 Application of Hyperpolarized 13C Magnetic Resonance Imaging to Detect Target Inhibition of NFkB Activation in Preclinical Patient-Derived Models of CNS Lymphoma

Nina Shah, MD488 Initial Results from a Phase 1 Clinical Study of bb21217, a Next-Generation Anti-BCMA CAR T Therapy

Neil P. Shah, MD2999 Multiparameter Single Cell Analysis to Identify Immune Cell States That May Contribute to Successful Treatment Discontinuation in Patients with Chronic Myeloid Leukemia

4253 Updated 18-Month Results from Dasfree: A Study Evaluating Dasatinib Discontinuation in Patients (Pts) with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) and Deep Molecular Response (DMR)

Catherine C. Smith, MD1458 Impact of Minimal Residual Disease and Achievement of Complete Remission/Complete Remission with Partial Hematologic Recovery (CR/CRh) on Overall Survival Following Treatment with Gilteritinib in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) with FLT3 Mutations

1476 Longitudinal Monitoring of AML Tumors with High-Throughput Single-Cell DNA Sequencing Reveals Rare Clones Prognostic for Disease Progression and Therapy Response

Elliot Stieglitz, MD388 Germline GAB2 Mutations in Childhood Acute Lymphoblastic Leukemia

Elliot Vichinsky, MD505 Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease

Linda T. Vo, PhD2359 Optimized Beta-Globin Expression and Enucleation from Induced Red Blood Cells for In Vitro Modeling of Sickle Cell Disease

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Patrick B Walter, MSc, PhD3631 The Potential of Plant-Based Compounds As Iron Chelators

Mark C. Walters, MD1025 Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

1026 Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 StudyClinically Relevant Abstract

Arun P Wiita, MD, PhD— Enhancing Immunotherapy Efficacy for Aging Myeloma Patients Ineligible for Transplant

469 Integrating Phosphoproteomics and Transcriptional Classifiers Reveals “Hidden Signaling” in Multiple Myeloma Including Differential KRAS and NRAS Mutant Effects

3212 Novel Allosteric Inhibitors of Heat Shock Protein 70 As Agents to Probe Protein Homeostasis and Overcome Proteasome Inhibitor Resistance in Multiple Myeloma

James A. Wells, PhD1128 Active Calpain Promotes Fibrin Clot Contraction By Strengthening the Coupling of Fibrin-Bound αIIbβ3 to the Platelet Cytoskeleton

Lena E. Winestone, MD, MSHP369 Increased Disease Burden Among Black Children Compared to White Children with Newly Diagnosed Acute Myeloid Leukemia

3531 Cost of Care at NCI-Designated Comprehensive Cancer Centers Vs. Other Treatment Sites for Young Adults with Newly-Diagnosed Acute Lymphoblastic Leukemia (ALL)

3597 A Novel Approach to Identifying Septic Shock (SS) in Children with Acute Lymphoblastic Leukemia (ALL) Using Pediatric Health Information System (PHIS) Data: Methods Validation and Incidence Estimation in a National Cohort

624 Using Administrative Data to Identify Relapse and Hematopoietic Stem Cell Transplantation (HSCT) in Children with Acute Lymphoblastic Leukemia (ALL): Validation at Two Centers and Incidence Estimation in a National Cohort

4866 Disparities in Cancer-Related Mortality and Long-Term Survival in Adolescent and Young Adults with Hodgkin Lymphoma: A Population-Level Analysis across the United States

Jeffrey L. Wolf, MD3236 Retrospective Analysis of Modified Hyper-CVAD Therapy Combined with Proteasome Inhibition for Patients with Multiple Myeloma and Renal Insufficiency or Renal Failure

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