Myelodysplastic Syndromes (MDS)

Key Points NK cells phenotyping reveals impaired cytotoxicity, chronic activation and exhaustion in VEXAS syndrome. Decreased circulating NK cells were independently associated with an increased risk of severe infections in VEXAS syndrome. VEXAS (vacuoles, E1 enzyme, X-linked…

Their research led to the 2022 approval of olutasidenib for certain patients with IDH1-mutant AML, which occurs in about 10% of AML. After that success, Dr. Watts and his team began to focus closely on testing the drug in MDS, a related condition that often progresses to AML. IDH…

A phase 2 study found that eprenetapopt plus azacitidine induced responses in patients with TP53-mutated myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). This combination may be an opportune bridge to transplantation, according to David A. Sallman, MD, of the…

A phase 2 study found that eprenetapopt plus azacitidine induced responses in patients with TP53-mutated myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). This combination may be an opportune bridge to transplantation, according to David A. Sallman, MD, of the…

In this issue of Blood, Takashima et al show that reduced STAT1 abundance in SRSF2-mutant myelodysplastic syndrome (MDS) cells confers protection against interferon (IFN)-driven cell suppression relative to normal wild-type (WT) cells.1 Importantly, the authors demonstrate that…

Abstract With age, clonal expansions occur pervasively across normal tissues yet only in rare instances lead to cancer, despite being driven by well-established cancer drivers. Characterization of the factors that influence clonal progression is needed to inform interventional…

Abstract GATA2 deficiency is an autosomal dominant transcriptopathy disorder with high risk for myelodysplastic syndrome (MDS). To elucidate genotype-phenotype associations and identify new genetic risk factors for MDS, we analyzed 218 individuals with germline heterozygous GATA2…

Abstract Background: RNA splicing factor (SF) mutations are associated with adverse outcomes in patients with acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes/neoplasms (HR-MDS). Preclinical data suggest that aberrant RNA splicing can lead to the generation…

A post-hoc analysis of pooled data from the IMerge trial found that patients with low-risk myelodysplastic syndromes (MDS) who were ineligible for erythropoiesis-stimulating agents (ESAs) due to elevated baseline serum erythropoietin derived clinical benefit from frontline…