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

Kerri Becktell, Deborah Berlyne, Simona Pagliuca, Lauren Pommert, Pedro H Prata, David Margolis, Régis Peffault de Latour, Carlo Dufour, Filomena Pierri

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)19-25
Number of pages7
JournalLeukemia Research
Volume80
DOIs
Publication statusPublished - May 2019

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