57th ASH Annual Meeting and Exposition (December 5-8, 2015): 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Heme and Anemia

Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron Oral and Poster Abstracts
Oral

W414AB, Level 4 (Orange County Convention Center)

Moderators:

Laurie A Steiner, MD, University of Rochester and Lionel Blanc, PhD, The Feinstein Institute for Medical Research

Disclosures:

No relevant conflicts of interest to declare.

4:30 PM

271

Mitochondrial Protein Kinase A Regulates Heme Biosynthesis

Jacky Chung, Ph.D.¹, Johannes G. Wittig¹^*, Daniel E. Bauer, M.D., Ph.D.², Joshua J. Coon, Ph.D.³^*, Dave J. Pagliarini, Ph.D.⁴^*, Harry A. Dailey, Ph.D.⁵^*, Harvey F. Lodish, Ph.D.⁶ and Barry H. Paw, M.D., Ph.D.¹

¹Division of Hematology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA
²Division of Hematology-Oncology, Dana-Farber Cancer Institute, Boston Children's Hospital, Harvard Medical School, Boston, MA
³Department of Chemistry, University of Wisconsin, Madison, WI
⁴Department of Biochemistry, University of Wisconsin, Madison, WI
⁵Departments of Microbiology, Biochemistry & Molecular Biology, University of Georgia, Athens, GA
⁶Massachusetts Institute of Technology, Whitehead Institute for Biomedical Research, Cambridge, MA

4:45 PM

272

Biallelic Mutations in PARP4 Are Linked to a Variant Form of Congenital Dyserythropoietic Anemia

Paola Bianchi, PhD¹^*, Elisa Fermo, PhD¹^*, Jennifer C Eng^2,3^*, Jacob C Ulirsch^3,4^*, Cristina Vercellati, BSc¹^*, Paola Braidotti, MD⁵^*, Gordon Hildick-Smith^6,7^*, Timothy J Satchwell, PhD⁸^*, Stephanie Pellegrin, PhD⁸^*, Alberto Zanella, M.D.¹, Wilma Barcellini, MD⁹^*, Barry H. Paw, M.D., Ph.D.^6,7, Ashley Mark Toye, PhD^8,10^* and Vijay G Sankaran, MD, PhD^3,4

¹Oncohematology Unit - Physiopathology of Anemias Unit, Foundation IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy
²Division of Hematology/Oncology, Manton Center for Orphan Disease Research, Boston Children’s Hospital, Boston, MA
³Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
⁴Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA
⁵Pathology Department, S. Paolo Hospital, Milan, Italy
⁶Division of Hematology/Oncology, Boston Children’s Hospital, Harvard Medical School, Boston, MA
⁷Division of Hematology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA
⁸School of Biochemistry, University of Bristol School of Medical Sciences, Clifton, Bristol, United Kingdom
⁹Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
¹⁰Bristol Institute of Transfusion Sciences, NHSBT, Filton, United Kingdom

5:00 PM

273

ALK2 Inhibition Via TP-0184 Abrogates Inflammation-Induced Hepcidin Expression and Is a Potential Therapeutic for Anemia of Chronic Disease

Peter Peterson, PhD¹^*, Katherine K. Soh¹^*, Ye Sol Lee²^*, Wontak Kim¹^*, Clifford J. Whatcott, PhD¹^*, Adam Siddiqui-Jain, PhD³^*, David J. Bearss, PhD³ and Steven L. Warner, PhD³

¹Discovery Biology, Tolero Pharmaceuticals, Inc., Lehi, UT
²Discovery Biology, Tolero Pharmaceuticals, Lehi, UT
³Tolero Pharmaceuticals, Inc., Lehi, UT

5:15 PM

274

Complement Inhibition with Eculizumab in Patients with Cold Agglutinin Disease (CAD): Results from a Prospective Phase II Trial (DECADE Trial)

Alexander Röth, MD¹, Martin Bommer, MD²^*, Andreas Hüttmann, MD³, Dörte Herich-Terhürne¹^*, Nils Kuklik, PhD⁴^*, Lenz Veronika⁵^*, Hubert Schrezenmeier, MD⁶ and Ulrich Dührsen, MD¹

¹Department of Hematology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
²Department of Internal Medicine III, University Hospital Ulm, Ulm, Germany
³Department of Haematology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
⁴Center for Clinical Trials, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
⁵Institute of Transfusion Medicine, University Hospital Essen, University of Duisburg-Essen, Essen, Germany
⁶Institute of Clinical Transfusion Medicine and Immunogenetics, University of Ulm, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, Ulm, Germany

5:30 PM

275

Small Molecule Factor D Inhibitors Block Complement Activation in Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome

Eleni Gavriilaki, MD¹, Jane A Thanassi, BS²^*, Guangwei Yang²^*, Xuan Yuan, MD, MSc¹, Mngjun Huang²^* and Robert A. Brodsky, MD¹

¹Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD
²Achillion Pharmaceuticals, New Haven, CT

5:45 PM

276

Long-Term Follow up of the Beneficial Effects and of Issues in Subtotal Splenectomy for Hereditary Spherocytosis

Thomas Pincez¹^*, Corinne Guitton²^*, Frederic Gauthier³^*, Guenolee Lambert⁴^*, Veronique Picard, PhD, MD⁵^*, Madeleine feneant Thibault⁶^*, Narla Mohandas, DSc⁷, Gil Tchernia, MD⁸^* and Loic Garcon^9,10^*

¹Service de Pédiatrie Générale, CHU Bicêtre, Le Kremlin-Bicêtre, France
²Pediatrics, SCD Referral Bicetre Center, Kremlin-Bicetre, France
³Service de chirurgie pédiatrique, CHU Bicêtre, Le Kremlin-Bicêtre, France
⁴Service de chirurgie pédiatrique, CHU Bicêtre, le Kremlin-Bicêtre, France
⁵Laboratoire d'Hématologie, CHU Bicêtre, Le Kremlin Bicetre, France
⁶Laboratoire de Biochimie, CHU Bicêtre, Le Kremlin-Bicêtre, France
⁷New York Blood Center, New York, NY
⁸CRLD, Paris, France
⁹Laboratoire d'Hématologie, CHU Bicêtre, Le Kremlin Bicêtre, France
¹⁰Service d'Hématologie Biologique, CHU Amiens, Amiens, France

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^*signifies non-member of ASH

101.O2.6 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Heme and Anemia