What changed: Myelodysplastic syndromes / neoplasms (MDS)

When this page itself was last checked or edited.

Lower-risk MDS transfusion-dependent anaemia

Preparative regimen for allogeneic HSCT in AML/MDS with fludarabine

Venetoclax added to azacitidine did not significantly improve overall survival in untreated higher-risk myelodysplastic syndromes.

Low- to intermediate-1-risk MDS with transfusion-dependent anaemia after ESA failure

IMerge: telomerase inhibitor achieves durable transfusion independence after ESA failure.

First-line anaemia in ESA-naive lower-risk MDS

More patients achieved transfusion independence with a haemoglobin rise on luspatercept than on epoetin alfa; first-line approval in August 2023.

Imetelstat produced eight-week transfusion independence in significantly more patients than placebo; approved in June 2024.

Magrolimab (ENHANCE) and sabatolimab (STIMULUS-MDS2) add nothing to azacitidine; VERONA (venetoclax) negative in 2024.

Molecular risk model with 31 genes; classification renamed myelodysplastic neoplasms with genetically defined entities.

Three-year overall survival 47.

MDS/CMML

MDS and CMML

MDS-RS and beta-thalassaemia

Very low- to intermediate-risk MDS with ring sideroblasts after ESA failure

Luspatercept produced transfusion independence for eight weeks or longer in significantly more patients than placebo; approved in April 2020.

Median event-free survival 1,440 days with deferasirox against 1,091 days with placebo (hazard ratio 0.

MEDALIST in ring-sideroblast MDS after ESA failure; COMMANDS (2023) moves it to first line.

Paroxysmal nocturnal haemoglobinuria

Revised score with five cytogenetic groups and refined thresholds.

Azacitidine improved overall survival over conventional care regimens in higher-risk myelodysplastic syndromes.

MDS del(5q)

Transfusion-dependent anaemia in low-risk MDS with del(5q)

Transfusion independence in two-thirds of del(5q) patients (List et al.).

First hypomethylating agent approved (MDS)

First drug approved for MDS (US); AZA-001 (2009) shows survival benefit in higher-risk disease.

Greenberg's International Prognostic Scoring System combines blasts, cytogenetics and cytopenias.

Bennett and colleagues define refractory anaemia, RARS, RAEB, RAEB-T and CMML.