Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Aplastic Anemia & MDS International Foundation (AA&MDSIF): Bone Marrow Failure Disease Scientific Symposium 2018. Leuk Res 2019 05;80:19-25

Date

03/26/2019

Pubmed ID

30908982

DOI

10.1016/j.leukres.2019.03.003

Scopus ID

2-s2.0-85063209621   1 Citation

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.

Author List

Becktell K, Berlyne D, Pagliuca S, Pommert L, Prata PH, Margolis D, de Latour RP, Dufour C, Pierri F

Authors

Kerri Becktell MD Assistant Professor in the Pediatrics department at Medical College of Wisconsin
David A. Margolis MD Interim Chair, Professor in the Pediatrics department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Anemia, Aplastic
Congresses as Topic
Humans
Myelodysplastic Syndromes
Prognosis