62nd ASH Annual Meeting and Exposition: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster I

Oral and Poster Abstracts
Poster

Adult, Anemias, antibodies, aplasia, apoptosis, autoimmune disorders, Biological, Animal models, aplastic anemia, cell division, Diseases, Non-Biological, cell regulation, Therapies, Adverse Events, red blood cells, Biological Processes, Immune Disorders, PNH, Technology and Procedures, Cell Lineage, epigenetics, erythropoiesis, Study Population, Clinically relevant, genomics, hematopoiesis, pharmacology, immune mechanism, inflammation, NGS, pathogenesis

748

Roxadustat Lowers Risk of RBC Transfusion in Patients with Anemia of CKD

Steven Fishbane¹^*, Robert Provenzano²^*, Anjay Rastogi, MD, PhD³^*, Daniel Coyne⁴^*, Roberto Pecoits-Filho⁵^*, Chaim Charytan⁶^*, Maksym Pola⁷^*, Lona Poole⁸^*, Gopal Saha⁸^*, Willis Chou⁸^*, Tyson Lee⁹^* and Peony Yu, MD¹⁰^*

¹Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Great Neck, NY
²School of Medicine, Wayne State University, Detroit, MI
³David Geffen School of Medicine at UCLA, Los Angeles, CA
⁴Washington University School of Medicine, Saint Louis, MO
⁵Arbor Research Collaborative for Health, Ann Arbor, MI
⁶Nephrology Associates, New Rochelle, NY
⁷Astra Zeneca, Warsaw, Poland
⁸FibroGen, Inc., San Francisco, CA
⁹FibroGen, Inc., san francisco, CA
¹⁰FibroGen, Inc, San Francisco, CA

749

Pooled Efficacy and Cardiovascular Safety Results of Roxadustat Compared with Epoetin Alfa in the Treatment of Anemia in Chronic Kidney Disease Patients on Dialysis

Steven Fishbane¹^*, Robert Provenzano²^*, Lynda Szczech, MD³^*, Robert Leong⁴^*, Khalil Saikali⁵^*, Ming Zhong⁶^*, Tyson Lee⁷^*, Mark Houser⁸^*, Lars Frison⁸^*, John Houghton⁹^*, Peony Yu, MD¹⁰^* and Thomas Neff⁵^*

¹Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Great Neck, NY
²School of Medicine, Wayne State University, Detroit, MI
³FibroGen Inc.,, Durham, NC
⁴FibroGen Inc., San Francisco
⁵FibroGen, Inc., San Francisco, CA
⁶FibroGen, Inc., San Francisco
⁷FibroGen, Inc., san francisco, CA
⁸AstraZeneca, Gaithersburg, MD
⁹Astrazeneca, Gaithersburg, MD
¹⁰FibroGen, Inc, San Francisco, CA

750

Lncrna-Iκbα Promotes Erythroid Differentiation By Inhibiting NF-Κb Pathway As a Cerna Against Mir-24-3p in Polycythemia Vera

Zhigang Li¹^*, Xuemei Fu¹^*, Zhixiang Zuo, PhD²^*, Fangfang Sun¹^*, Xiaojun Zhang¹^*, Jieping Chen³^* and Yu Hou, PhD⁴

¹Southwest Hospital, Third Military Medical University (Army Medical University), ChongQing, China
²State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, China
³Southwest Hospital, Third Military Medical University (Army Medical University), Chongqing, China
⁴Department of Hematology, Southwest Hospital, Third Military Medical University (Army Medical University), CHONGQING, China

751

The Samd14-Capping Protein Complex Controls Cell Signaling in the Erythropoietic Stress Response

Kyle J Hewitt, PhD

Department of Genetics, Cell Biology and Anatomy, University of Nebraska, Omaha, NE

752

Fostamatinib for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (wAIHA): A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study

Nichola Cooper, MD¹, Robert P. Numerof, PhD²^*, Sandra Tong, MD²^* and David J. Kuter, MD³

¹Hammersmith Hospital, Imperial College London, London, United Kingdom
²Rigel Pharmaceuticals, Inc., South San Francisco, CA
³Hematology Division, Massachusetts General Hospital, Boston, MA

753

C3 Inhibition with Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the Paddock and Palomino Trials

Raymond S Wong, MBChB, MD¹, Kalina Ignatova²^*, Humphrey Pullon³^*, Jameela Sathar⁴^*, Pimjai Niparuck, MD⁵^*, Tontanai Numbenjapon⁶^*, Eric Tse, MBBS, PhD, FRCP, FRCPath⁷, Surapol Issaragrisil, MD⁸^*, Eloy Roman, MD⁹^*, Ismail Amine¹⁰^*, Lisa Tan¹¹^*, Carl Di Casoli¹²^*, Pascal Deschatelets, PhD¹²^*, Cedric Francois, MD, PhD¹²^*, Federico Grossi, MD¹²^* and Andrija Bogdanovic¹³^*

¹Sir YK Pao Centre for Cancer & Department of Medicine and Therapeutics, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong, Hong Kong
²National Specialized Hospital for Active Treatment of Hematologic Diseases, Sofia, Bulgaria
³Waikato Hospital, Hamilton, New Zealand
⁴Department of Hematology, Ampang Hospital, Selangor, Malaysia
⁵Ramathibodi Hospital, Bangkok, Thailand
⁶Phramongkutklao College of Medicine, Bangkok, Thailand
⁷Department of Medicine, The University of Hong Kong, Hong Kong, Hong Kong
⁸Faculty of Medicine, Siriraj Hospital, Bangkok, Thailand
⁹Lakes Research, Miami Lakes, FL
¹⁰Tokuda Hospital, Sofia, Bulgaria
¹¹Lisa Tan Pharma Consulting Ltd, Cambridge, United Kingdom
¹²Apellis Pharmaceuticals, Inc., Waltham, MA
¹³Clinic of Hematology, Clinical Center of Serbia, Faculty of Medicine, University of Belgrade, Belgrade, Serbia

754

GATA2 Mutation in Twins with Transient Aplastic Anemia after Chloramphenicol Exposure; A Follow up to Nagao and Mauer 1969

Zuhair Alam¹^* and Thomas Coyle, MD²

¹Good Samaritan Hospital, Cincinnati, OH
²SUNY Upstate Med. Univ., Cincinnati, OH

755

Management of Meningococcal Disease Risk in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) on Complement Inhibitors: 18 Years’ Experience from the UK National PNH Service in Leeds

Louise Arnold¹^*, Ray Borrow, PhD, FRCPath²^*, Kathryn Riley³^*, Talha Munir, MBBS⁴^*, Richard Kelly, BSc, MD³^*, Rachael Jones, BSc⁵^*, Jeanifer Gachev³^*, Briony Forrest³^*, Alexandra Pike, MBBS⁵^*, Petra Muus, MD, PhD³, Peter Hillmen, MBChB, PhD⁶^* and Morag Griffin, MBChB, FRCPath³^*

¹Department of Haematology, St James University Hospital, Leeds, LS97TF, United Kingdom
²Meningococcal Reference Unit,, Manchester, United Kingdom
³Department of Haematology, St James University Hospital, Leeds, United Kingdom
⁴St James University Hospital, Leeds, United Kingdom
⁵Department of Haematology, St James University Hospital, Leeds, ENG, United Kingdom
⁶St. James's University Hospital, Institute of Oncology, Leeds, United Kingdom

756

Phase 3 Study of Danicopan, an Oral Complement Factor D Inhibitor, As Add-on Therapy to a C5 Inhibitor in Patients with Paroxysmal Nocturnal Hemoglobinuria with Clinically Evident Extravascular Hemolysis

Austin G Kulasekararaj, MD, MBBS, MRCP, FRCPath¹, Antonio Risitano, MD, PhD², Jong Wook Lee, M.D., Ph.D.³, Mingjun Huang, PhD⁴^*, Jun-Ichi Nishimura, MD, PhD⁵, Alejandra Ramirez-Santiago⁴^* and Robert Brodsky⁶^*

¹King's College Hospital - NHS Foundation Trust, NIHR Wellcome King’s Clinical Research Facility, London and King’s College London, London, United Kingdom
²Hematology and BMT Unit, AORN Moscati Avellino, Avellino, Italy
³Seoul St. Mary’s Hospital, The Catholic University of Korea, Seoul, Korea, Republic of (South)
⁴Alexion Pharmaceuticals, New Haven, CT
⁵Osaka University, Osaka, Japan
⁶Division of Hematology, Johns Hopkins University School of Medicine, Baltimore, MD

757

Roxadustat Increases Hemoglobin in Anemic Non-Dialysis-Dependent (NDD) Chronic Kidney Disease (CKD) Patients Independent of Inflammation

Carol A. Pollock¹^*, Simon Roger²^*, Roberto Manllo-Karim³^*, Maksym Pola⁴^*, Stefan Tham⁵^*, Kin-Hung P Yu, MD⁶^* and Mohamed El-Shahawy⁷^*

¹The University of Sydney, Sydney, Australia
²Renal Unit, Gosford Hospital, Gosford, Australia
³Gamma Medical Research, McAllen, TX
⁴Clinical Research, AstraZeneca, Warsaw, Poland
⁵Clinical Research, AstraZeneca, Gothenburg, Sweden
⁶FibroGen Inc., San Francisco
⁷Keck-USC School of Medicine, Los Angeles, CA

758

Roxadustat Treatment Results in Consistent Improvements in Hemoglobin (Hb) Versus Placebo: An Analysis of 3 Multinational RCTs in Patients with Non-Dialysis-Dependent Chronic Kidney Disease (NDD-CKD)

Roberto Pecoits-Filho^1,2^*, Tak Mao Chan³^*, Elise Hardy⁴^*, Kin-Hung P Yu, MD⁵^* and Steven Fishbane⁶^*

¹Arbor Research Collaborative for Health, Ann Arbor, MI
²School of Medicine, Pontifical Catholic University of Parana, Curitiba, Brazil
³Division of Nephrology, Department of Medicine, Queen Mary Hospital, The University of Hong Kong, Pok Fu Lam, Hong Kong
⁴Clinical Research, AstraZeneca, Gaithersburg, MD
⁵FibroGen Inc., San Francisco
⁶Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Great Neck, NY

759

Inflammation and Fatigue in Patients with Cold Agglutinin Disease (CAD): Analysis from the Phase 3 Cardinal Study

Ilene C. Weitz¹, Yasutaka Ueda², Frank Shafer³^*, Jun Su⁴, Meredith Foster³^*, Michael Storek³^* and Parija Patel³^*

¹Keck School of Medicine, University of Southern California, Los Angeles, CA
²Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan
³Sanofi, Cambridge, MA
⁴Medical Affairs, Sanofi, Cambridge, MA

760

Incomplete Complement Inhibition in Patients with PNH on Eculizumab - 5 Year Experience from the National PNH Service Leeds

Rachael Jones, BSc^*, Petra Muus, MD, PhD, Talha Munir, MBBS^*, Alexandra Pike, MBBS^*, Louise Arnold^*, Jeanifer Gachev^*, Briony Forrest^*, Peter Hillmen, MBChB, PhD^* and Morag Griffin, MBChB, FRCPath^*

Department of Haematology, St James University Hospital, Leeds, United Kingdom

761

Roxadustat Treatment Corrects Anemia to Hemoglobin (Hb) Values ≥10 g/dL in the Majority of Patients with Non-Dialysis-Dependent Chronic Kidney Disease (NDD-CKD)

Anjay Rastogi, MD, PhD¹^*, Roberto Pecoits-Filho²^*, Steven Fishbane³^*, Dustin Little⁴^*, Elise Hardy⁴^*, Kin-Hung P Yu, MD⁵^* and Robert Provenzano⁶^*

¹David Geffen School of Medicine at UCLA, Los Angeles, CA
²Arbor Research Collaborative for Health, Ann Arbor, MI
³Department of Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Great Neck, NY
⁴Clinical Research, AstraZeneca, Gaithersburg, MD
⁵FibroGen Inc., San Francisco, CA
⁶Department of Nephrology, Hypertension & Transplantation St. John Hospital & Medical Center Detroit, Detroit, MI

762

Identification of a Warm Autoimmune Hemolytic Anemia (wAIHA) Population Using Predictive Analytics of a Known Clinically Profiled Cohort

Keith R. McCrae, MD^1,2, Jennifer Beachell³^*, Tricia Gooljarsingh, PhD⁴^*, Mary Lee Tjoa, PhD⁵^*, Graham K Jones, PhD⁶^* and Julieanna K Gubitosa⁷^*

¹Taussig Cancer Institute, Cleveland Clinic Lerner College of Medicine, Cleveland Clinic, Cleveland, OH
²Cardiovascular & Metabolic Sciences, Cleveland Clinic, Cleveland, OH
³Momenta Pharmaceuticals, Inc, Cambridge, MA
⁴Medical Affairs and Communications, Cambridge, MA
⁵Director of Medical Affairs, Momenta Pharmaceuticals, Boston, MA
⁶Director of Client Engagement, IPM.ai, Cambridge, MA
⁷Director of Integration and Analytics, IPM.ai, Cambridge, MA

763

Detection of Myeloid and Clonal Expansion Related Gene Mutations By Whole-Exome Sequencing in Patients with Paroxysmal Nocturnal Hemoglobinuria

Fangfei Chen^*, Bing Han, MD/PhD^* and Jian Li, MD

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China

764

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

Alexander Röth, MD¹, Britta Hoechsmann²^*, Morag Griffin, MBChB, FRCPath³^*, Carlos M. de Castro, MD⁴, Jeffrey Szer, MBBS⁵, Kensuke Usuki, MD, PhD⁶, Juliette Soret, MD⁷^*, Mohamed Hamdani, MS⁸^*, Temitayo Ajayi⁸^*, Sujata P. Sarda, PhD⁸^* and Jens Panse, MD⁹

¹Department of Hematology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
²University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Institute of Transfusion Medicine, Ulm, Germany
³Department of Haematology, St James University Hospital, Leeds, United Kingdom
⁴Duke University School of Medicine, Durham, NC
⁵Department of Clinical Haematology, Royal Melbourne Hospital, Melbourne, VIC, Australia
⁶Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan
⁷Centre d'Investigations Cliniques, Hôpital Saint-Louis; Assistance Publique – Hôpitaux de Paris; Université de Paris, Paris, France
⁸Apellis Pharmaceuticals, Inc., Waltham, MA
⁹Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen, Germany

765

Sutimlimab, a Complement C1s Inhibitor, Improves Quality of Life in Patients with Cold Agglutinin Disease: Patient-Reported Outcomes Results of the Phase 3 Cardinal Study

Alexander Röth, MD¹, Wilma Barcellini²^*, Tor Henrik Anderson Tvedt³^*, Yoshitaka Miyakawa⁴^*, David J. Kuter, MD⁵, William Hobbs⁶, Jun Su⁶, Xiaoyu Jiang⁶^*, Jaime Morales Arias⁶^* and Ilene C. Weitz⁷^*

¹Department of Hematology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
²Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
³Section for Hematology, Department of Medicine, Haukeland University Hospital, Bergen, Norway
⁴Thrombosis and Hemostasis Center, Saitama Medical University Hospital, Saitama, Japan
⁵Hematology Division, Massachusetts General Hospital, Boston, MA
⁶Sanofi, Cambridge, MA
⁷Jane Anne Nohl Division of Hematology, Department of Medicine, University of Southern California – Keck School of Medicine, Los Angeles, CA

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^*signifies non-member of ASH

101.P1.48 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster I