Myelodysplastic Syndromes (MDS)

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…

ABSTRACT Background Elevated blood counts in polycythemia vera (PV) are associated with increased thrombotic risk, which contributes to morbidity and mortality. Research design and methods This retrospective study describes treatment patterns and blood count control in patients…

Amer Zeidan, MBBS, MHS, is professor of internal medicine at Yale School of Medicine in New Haven, Connecticut. He is also the chief of the Division of Hematologic Malignancies at Yale Cancer Center, New Haven. Dr. Zeidan spoke with Blood Cancers Today about the phase 1/2 BEXMAB…

VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly identified monogenic disorder with symptoms including recurrent fever, skin involvement, pulmonary infiltrates, systemic vasculitis, and chondritis. 1 Notably, 25-50% of patients also have…