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Trials recruiting now, the landmark trials, the key papers and what they mean, the latest literature, and the milestones year by year.
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IMerge validated telomerase as a drug target in cancer, decades after its discovery, and gave a second-line option for MDS patients whose anaemia no longer responds to erythropoietin or luspatercept. The hint of clonal reduction is what makes the drug interesting beyond transfusion counts. Cytopenias require close monitoring in the first cycles.
COMMANDS moved luspatercept from second line (after ESA failure, MEDALIST trial) to first line, offering transfusion-dependent lower-risk MDS patients a better chance of transfusion freedom from the start. It changed guidelines and labels in 2023. Erythropoietin remains a reasonable and cheaper option for patients without ring sideroblasts or with low transfusion burden.
Allogeneic transplant should be included in the management plan of fit older adults with higher-risk myelodysplastic syndrome; donor search should start at diagnosis.
Query for this cancer: (TITLE:"Myelodysplastic syndromes / neoplasms" OR ABSTRACT:"Myelodysplastic syndromes / neoplasms" OR TITLE:"MDS" OR ABSTRACT:"MDS") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Myelodysplastic syndromes / neoplasms (MDS), not a curated reading list.
Bennett and colleagues define refractory anaemia, RARS, RAEB, RAEB-T and CMML.
Greenberg's International Prognostic Scoring System combines blasts, cytogenetics and cytopenias.
First drug approved for MDS (US); AZA-001 (2009) shows survival benefit in higher-risk disease.
Transfusion independence in two-thirds of del(5q) patients (List et al.).
Revised score with five cytogenetic groups and refined thresholds.
MEDALIST in ring-sideroblast MDS after ESA failure; COMMANDS (2023) moves it to first line.
Molecular risk model with 31 genes; classification renamed myelodysplastic neoplasms with genetically defined entities.
Magrolimab (ENHANCE) and sabatolimab (STIMULUS-MDS2) add nothing to azacitidine; VERONA (venetoclax) negative in 2024.
IMerge: telomerase inhibitor achieves durable transfusion independence after ESA failure.