61st Annual Meeting and Exposition (December 7-10, 2019): Late-Breaking Abstracts Session

General Sessions

Hall D, Level 2 (Orange County Convention Center)

Lecture Title:
Late Breaking Abstracts

Co-chairs:

Elisabeth Battinelli, MD, PhD, Brigham and Women's Hospital and Jason Gotlib, MD, MS, Stanford Cancer Center

Disclosures:

Gotlib: Seattle Genetics: Research Funding; Pharmacyclics: Research Funding; Promedior: Research Funding; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Incyte: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Gilead: Membership on an entity's Board of Directors or advisory committees, Research Funding; Deceiphera: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Blueprint Medicines: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Allakos: Honoraria, Membership on an entity's Board of Directors or advisory committees.

This highly anticipated session highlights the Program Committee's selections of the highest-impact abstracts, featuring substantive, novel, and groundbreaking data that were not available by the general abstract submission deadline and would otherwise not be presented at the ASH annual meeting.

LBA-1

A Randomized Phase 3 Trial of Blinatumomab Vs. Chemotherapy As Post-Reinduction Therapy in High and Intermediate Risk (HR/IR) First Relapse of B-Acute Lymphoblastic Leukemia (B-ALL) in Children and Adolescents/Young Adults (AYAs) Demonstrates Superior Efficacy and Tolerability of Blinatumomab: A Report from Children’s Oncology Group Study AALL1331

Patrick A. Brown, MD¹, Lingyun Ji, PhD²^*, Xinxin Xu, MS³^*, Meenakshi Devidas, PhD⁴^*, Laura Hogan, MD⁵, Michael J. Borowitz, MD, PhD⁶, Elizabeth A. Raetz, MD⁷, Gerhard Zugmaier⁸, Elad Sharon, MD, MPH⁹^*, Lia Gore, MD¹⁰, James A. Whitlock, MD¹¹, Michael A. Pulsipher, MD¹², Stephen P. Hunger, MD¹³ and Mignon L. Loh, MD¹⁴

¹Division of Pediatric Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD
²Department of Preventative Medicine, Keck School of Medicine, University of Southern California, Los Angeles
³Children's Oncology Group, Monrovia, CA
⁴Department of Global Pediatric Medicine, St. Jude Children’s Research Hospital, Memphis, TN
⁵Stony Brook Children's, Stony Brook, NY
⁶Departments of Pathology and Oncology, Johns Hopkins University, Baltimore, MD
⁷Pediatric Hematology/Oncology, Laura and Isaac Perlmutter Cancer Cent at NYU Langone, New York, NY
⁸Amgen Research (Munich) GmbH, Munchen, Germany
⁹Division of Cancer Treatment and Diagnosis, National Cancer Institute, Cancer Therapy Evaluation Program, Bethesda, MD
¹⁰Peds Heme/Onc/BMT-CT, University of Colorado, Children's Hospital Colorado, Aurora, CO
¹¹Hospital For Sick Children, Toronto, ON, CAN
¹²Children’s Center for Cancer and Blood Diseases, Blood and Marrow Transplantation Section, Children’s Hospital Los Angeles, Los Angeles, CA
¹³Department of Pediatrics and the Center for Childhood Cancer Research, Children's Hospital of Philadelphia and The Perelman School of Medicine at The University of Pennsylvania, Philadelphia, PA
¹⁴Department of Pediatrics, University of California Benioff Children’s Hospital, San Francisco, CA

LBA-2

Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase 3 Cardinal Study

Alexander Roeth, MD¹, Wilma Barcellini²^*, Shirley D'Sa³^*, Yoshitaka Miyakawa⁴, Catherine M Broome, MD⁵, Marc Michel⁶^*, David J. Kuter⁷, Bernd Jilma⁸, Tor Henrik Anderson Tvedt⁹, Stella Lin¹⁰^*, Xiaoyu Jiang¹⁰^*, Caroline Reuter¹⁰^*, William Hobbs¹⁰ and Sigbjørn Berentsen¹¹^*

¹Department of Hematology, West German Cancer Center, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
²Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
³UCLH Centre for Waldenström’s Macroglobulinemia and Related Conditions, University College London Hospitals NHS Foundation Trust, London, United Kingdom
⁴Thrombosis and Hemostasis Center, Saitama Medical University Hospital, Saitama, Japan
⁵Division of Hematology, MedStar Georgetown University Hospital, Washington, DC
⁶Henri-Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, UPEC, Créteil, France
⁷Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA
⁸Department of Clinical Pharmacology, Medical University of Vienna, Vienna, Austria
⁹Section for Hematology, Department of Medicine, Haukeland University Hospital, Bergen, Norway
¹⁰Sanofi, Waltham, MA
¹¹Department of Research and Innovation, Haugesund Hospital, Haugesund, Norway

LBA-3

The QUAZAR AML-001 Maintenance Trial: Results of a Phase III International, Randomized, Double-Blind, Placebo-Controlled Study of CC-486 (Oral Formulation of Azacitidine) in Patients with Acute Myeloid Leukemia (AML) in First Remission

Andrew H. Wei, MBBS, FRACP, FRCPA, PhD^1,2^*, Hartmut Döhner, MD³, Christopher Pocock, MBBS⁴, Pau Montesinos, MD, PhD^5,6^*, Boris Afanasyev, MD, DSci⁷^*, Hervé Dombret, MD^8,9,10, Farhad Ravandi, MD¹¹, Hamid Sayar, MD, MSc¹², Jun Ho Jang, MD, PhD¹³, Kimmo Porkka, MD¹⁴, Dominik Selleslag, MD¹⁵, Irwindeep Sandhu, MD¹⁶^*, Mehmet Turgut, MD¹⁷^*, Valentina Giai, MD¹⁸^*, Yishai Ofran, MD^19,20, Merih Kizil Cakar, MD²¹^*, Aida Botelho de Sousa, MD²²^*, Justyna Rybka, MD, PhD²³^*, Chiara Frairia, MD²⁴^*, Lorenza Borin, MD²⁵^*, Germana Beltrami, MD²⁶^*, Jaroslav Cermak, MD, PhD²⁷^*, Gert J. Ossenkoppele, MD, PhD²⁸, Ignazia La Torre, MD²⁹^*, Barry Skikne, MD²⁹^*, Keshava Kumar, PhD²⁹^*, Qian Dong, DrPH²⁹^*, CL Beach, PharmD²⁹ and Gail J. Roboz, MD^30,31

¹The Alfred Hospital, Melbourne, AUS
²Monash University, Melbourne, Australia
³Ulm University Hospital, Ulm, Germany
⁴Kent & Canterbury Hospital, Canterbury, United Kingdom
⁵CIBERONC, Instituto Carlos III, Madrid, Spain
⁶Hospital Universitari i Politècnic La Fe, Valenica, Spain
⁷Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology, Hematology and Transplantation, Pavlov First Saint Petersburg State Medical University, St. Petersburg, Ru, Russian Federation
⁸Hematology, Hôpital Saint-Louis, Paris, France
⁹Institut de Recherche Saint Louis, Paris, France
¹⁰Université de Paris, Paris, France
¹¹Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
¹²Indiana University Cancer Center, Indianapolis, IN
¹³Samsung Medical Centre, Seoul, Korea, Republic of (South)
¹⁴Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center, Helsinki, Finland
¹⁵AZ Sint-Jan Brugge-Oostende AV, Bruges, Belgium
¹⁶University of Alberta Hospital, Edmonton, Canada
¹⁷Ondokuz Mayis University, Samsun, Turkey
¹⁸Antonio e Biagio e Cesare Arrigo Hospital, Alessandria, Italy
¹⁹Rambam Medical Center, Haifa, Israel
²⁰Faculty of Medicine Technion, Haifa, Israel
²¹Dr. Abdurrahman Yurtaslan Ankara Oncology Training and Research Hospital, Ankara, Turkey
²²Hospital dos Capuchos (CHLC), Lisboa, Portugal
²³Wroclaw Medical University, Wroclaw, Poland
²⁴Città della Salute e della Scienza, Torino, Italy
²⁵Ospedale San Gerardo Monza, Monza, Italy
²⁶Ospedale Policlinico San Martino, Genoa, Italy
²⁷Ústav Hematologie a Krevní Transfuze, Prague, Czech Republic
²⁸Amsterdam UMC, Location VUMC, Amsterdam, Netherlands
²⁹Celgene Corporation, Summit, NJ
³⁰Weill Cornell Medicine, New York, NY
³¹New York Presbyterian Hospital, New York, NY

LBA-4

Integrated Transcriptomic and Genomic Sequencing Identifies Prognostic Constellations of Driver Mutations in Acute Myeloid Leukemia and Myelodysplastic Syndromes

Ilaria Iacobucci, PhD¹, Manja Meggendorfer, PhD², Niroshan Nadarajah²^*, Stanley Pounds, PhD³^*, Lei Shi, PhD³^*, Chunxu Qu, PhD¹^*, Constance Baer, PhD², Claudia Haferlach, MD², Wolfgang Kern, MD^2,4, Charles G. Mullighan, MBBS, MD ¹ and Torsten Haferlach, MD²

¹Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN
²MLL Munich Leukemia Laboratory, Munich, Germany
³Biostatistics, St. Jude Children's Research Hospital, Memphis, TN
⁴Munich Leukemia Laboratory, Munich, Germany

LBA-5

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD¹, Maureen Achebe, MD, MPH², Myriam Armant, PhD³^*, Pablo Bartolucci, MD, PhD⁴^*, Marioara Felicia Ciuculescu, MD⁵^*, Heather Daley⁶^*, Colleen Dansereau, MSN, RN, CPN⁷^*, Giuseppe Di Caprio, PhD⁸^*, Bronner Goncalves, MD, PhD⁹^*, Nicolas Hebert^10,11^*, Matthew M Heeney, MD¹², John M. Higgins, MD, PhD¹³^*, Leslie E. Lehmann, MD¹⁴, John P Manis, MD¹⁵, Olivier Negre, PhD¹⁶^*, Sarah Nikiforow, MD, PhD¹⁷, Ethan Schonbrun, PhD¹⁸^*, David K. Wood, PhD¹⁹^* and David A. Williams, MD²⁰

¹Children's Hospital Boston, Boston, MA
²Division of Hematology, Brigham and Women's Hospital/ Harvard Medical School, Boston, MA
³TransLab, Boston Children's Hospital, Harvard Medical School, Boston, MA
⁴Service de Médecine Interne, Hôpital Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris 12, Créteil, France
⁵Gene Therapy for Rare Diseases, Boston Children’s Hospital, Boston
⁶Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
⁷Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
⁸Center for Systems Biology, Massachusetts General Hospital/ Harvard Medical School, Boston, MA
⁹Centers for Systems Biology, Massachusetts General Hospital/ Harvard Medical School, Boston, MA
¹⁰Université Paris-Est Créteil, Créteil, France
¹¹INSERM-U955, EFS, Institut Mondor, Université Paris-Est Créteil, Team 2 Transfusion et maladies du globule rouge, Laboratoire d´Excellence GR-Ex, Créteil, France
¹²Division of Pediatric Hematology/Oncology, Boston Children's Hospital/ Harvard Medical School, Boston, MA
¹³Department of Systems Biology, Harvard Medical School, Boston
¹⁴Division of Pediatric Hematology/Oncology/Stem Cell Transplant Program, Dana-Farber/Boston Children’s Cancer & Blood Disorders Center, Boston, MA
¹⁵Department of Laboratory Medicine, Boston Children's Hospital, Boston, MA
¹⁶bluebird bio, inc., Cambridge, MA
¹⁷Cell Manipulation Core Facilities, Dana-Farber Cancer Institute, Boston, MA
¹⁸Center for Systems Biology, Massachusetts General Hospital, Boston
¹⁹Biomedical Engineering, University of Minnesota, Minneapolis, MN
²⁰Division of Hematology/Oncology, Dana-Farber/Boston Children’s Cancer & Blood Disorders Center, Boston, MA

LBA-6

Carfilzomib, Dexamethasone, and Daratumumab Versus Carfilzomib and Dexamethasone for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma (RRMM): Primary Analysis Results from the Randomized, Open-Label, Phase 3 Study Candor (NCT03158688)

Saad Z. Usmani, MD, MBBS¹, Hang Quach, MD², María-Victoria Mateos³, Ola Landgren, MD, PhD⁴, Xavier Leleu, MD, PhD⁵, David S. Siegel⁶, Katja Weisel⁷^*, Hui Yang⁸^*, Zandra K. Klippel, MD⁸, Anita Zahlten-Kumeli⁸ and Meletios A. Dimopoulos, MD⁹

¹Atrium Health, Charlotte, NC
²St. Vincent's Hospital, Melbourne, Australia
³University Hospital Salamanca/IBSAL, Salamanca, Spain
⁴Memorial Sloan Kettering Cancer Center, New York, NY
⁵Hopital Claude Huriez, Lille, France
⁶John Theurer Cancer Center at Hackensack University Medical Center, Hackensack, NJ
⁷Department of Oncology, Hematology and Bone Marrow Transplantation with Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
⁸Amgen, Inc., Thousand Oaks, CA
⁹National and Kapodistrian University of Athens, School of Medicine, Athens, Greece

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