SOHO State of the Art Update and Next Questions: Biology and Treatment of MDS

Publication date: Available online 25 September 2017 Source:Clinical Lymphoma Myeloma and Leukemia Author(s): David A. Sallman, Tiffany N. Tanaka, Alan List, Rafael Bejar Myelodysplastic Syndromes (MDS) are a heterogeneous group of myeloid neoplasms characterized by clonal hematopoiesis leading to bone marrow dysplasia and cytopenias. Recently, significant advancements have been made in understanding the pathogenic mechanisms of this disease. In particular, how a wide array of somatic mutations can induce a common clinical phenotype has been investigated. Specifically, activation of innate immune signaling (i.e. myeloid derived suppressor cells) and the NLRP3 inflammasome in hematopoietic stem/progenitor cells play a central role in the biology of MDS leading to pyroptotic cell death and clonal expansion. Additionally, deciphering the molecular drivers of MDS via next generation sequencing has also rapidly expanded our understanding of MDS with profound implications in prognosis, treatment decisions and future clinical investigations. Together, unraveling of the role of innate immunity/pyroptosis in the clinical phenotype of MDS patients and comprehensive molecular characterization has identified novel therapeutic strategies that offer significant promise.
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research