Aplastic Anemia & MDS International Foundation (AA&MDSIF): Bone Marrow Failure Disease Scientific Symposium 2018. (May 2019)

• Record Type: Journal Article
• Authors: Becktell, Kerri; Berlyne, Deborah; Pagliuca, Simona; Pommert, Lauren; Prata, Pedro H.; Margolis, David; de Latour, Régis Peffault; Dufour, Carlo; Pierri, Filomena
• Is Part Of: Leukemia research. Volume 80(2019)
• Page Start: 19
• Page End: 25
• Publication Date: 2019-05
• Subjects: BMF bone marrow failure -- DC dyskeratosis congenita -- AML acute myelogenous leukemia -- MDS myelodysplastic syndromes -- HSC hematopoietic stem cell -- HSCT hematopoietic stem cell transplantation -- PNH paroxysmal nocturnal hemoglobinuria -- OS overall survival -- GVHD graft-versus-host disease -- RBC red blood cell -- IST immunosuppressive therapy -- ATG antithymocyte globulin -- SAA severe aplastic anemia -- MUD matched unrelated donor -- EFS event-free survival -- iPSC induced pluripotent stem cell -- CMML chronic myelomonocytic leukemia -- HMA hypomethylating agent -- PD-1 programmed cell death protein 1 -- MPN myeloproliferative neoplasm -- FDA U.S. Food and Drug Administration; Bone marrow failure -- Myelodysplastic syndromes -- Aplastic anemia -- Paroxysmal nocturnal hemoglobinuria -- Hematopoietic stem cell transplantation -- Novel therapies; Leukemia -- Periodicals; Leukemia -- Periodicals; Leucémie -- Périodiques; Leukemia; Periodicals; Electronic journals; Electronic journals; 616.9941905
• DOI: 10.1016/j.leukres.2019.03.003
• Languages: English
• ISSNs: 0145-2126

Abstract

Abstract: The bone marrow failure (BMF) syndromes are a group of rare disorders characterized by ineffective hematopoiesis resulting from deficiencies in the hematopoietic stem cell compartment. Although these diseases are typically acquired, some forms (e.g., Fanconi anemia, dyskeratosis congenita, Diamond Blackfan anemia, and Shwachman Diamond syndrome) are inherited. Patients with BMF syndromes can develop peripheral blood cytopenias and pancytopenia, and their disease can ultimately progress to acute myelogenous leukemia (AML). Research around the world is shedding light on the biology of the BMF syndromes, their clinical effects, and novel treatments. The Aplastic Anemia and MDS International Foundation (AAMDSIF) is an independent nonprofit organization whose mission is to help patients and family members cope with BMF syndromes. This report summarizes presentations on the latest scientific discoveries in BMF syndromes from the Sixth International Bone Marrow Failure Disease Scientific Symposium sponsored by AAMDSIF on March 22–23, 2018, in Rockville, Maryland.