Myelodysplastic Syndromes (MDS)

Clonal Hematopoiesis Hematopoiesis: (hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow. is defined as the presence of mutations in peripheral blo

Hypomethylating agent (HMA) failure myelodysplastic syndrome (MDS) patients have poor outcomes and urgent need for novel therapies. Hedgehog pathway signaling upregulation plays a central role in myeloid neoplasm pathogenesis and leukemia stem cell survival. We evaluated the efficacy and safety of the smoothened inhibitor glasdegib in HMA-failure MDS (n = 35, median age 73 years).

Risk stratification is critical in the care of patients with myelodysplastic syndromes (MDS). Approximately 10% have a complex karyotype (CK), defined as more than two cytogenetic abnormalities, which is a highly adverse prognostic marker. However, CK-MDS can carry a wide range of chromosomal abnormalities and somatic mutations. To refine risk stratification of CK-MDS patients, we examined data from 359 CK-MDS patients shared by the International Working Group for MDS. Mutations were underrepresented with the exception of TP53 mutations, identified in 55% of patients.

Lenalidomide and hypomethylating agents (HMAs) azacitidine and decitabine are approved for treating myelodysplastic syndromes (MDS), but optimal sequencing is unclear. Adults with MDS were identified from a US payer claims database (Inovalon MORE2 Registry) to compare outcomes with lenalidomide followed by HMA (LEN-HMA) or HMA followed by lenalidomide (HMA-LEN). There were 96 patients who received LEN-HMA and 89 who received HMA-LEN. LEN-HMA-treated patients had a longer time to second treatment discontinuation (29.0 vs.

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Next generation sequencing (NGS) has become an important tool to inform disease risk for myeloid malignancies, however data remains conflicting regarding the significance of individual mutations. We performed targeted NGS on 112 patients with AML, and 80 with MDS, who underwent allogeneic hematopoietic cell transplantation. The most common mutations in AML were TET2 (14.7%), FLT3 (12.9%), DNMT3A (12.1%), and RUNX1 (7.8%). Complex cytogenetics (HR 2.82, P = .017) and disease status (<CR) (HR 2.58, P < .001) was significantly associated with worse RFS.