Myelodysplastic Syndromes (MDS)

Hypomethylating agents (HMAs) are the standard of care for patients with myelodysplastic syndrome (MDS). However, only around 50% of patients respond to these agents, and responses tend to be transient, with loss of response frequently happening within 2 years and being associated with very poor prognosis and limited therapeutic options. Identification of patients who will respond to HMAs is challenging. Mechanisms underlying resistance to HMAs are not clear yet. Recently, absence of response has been associated with increased cell-cycle quiescence among the hematopoietic progenitor cells.

This PDQ cancer information summary has current information about the treatment of myelodysplastic syndromes myelodysplastic syndromes:

Commonly used scoring systems rely on blood counts, histological and cytological examination of bone marrow bone marrow: The soft, spongy tissue inside most bones.

Immunosuppressive therapy Immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response.

Myeloid neoplasms, including myelodysplastic syndromes myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A grou

A patient with a diagnosis of myelodysplastic syndrome (MDS) with isolated 5q deletion underwent repeat bone marrow biopsy bone marrow biopsy: A medical procedur

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).