How we manage adults with myelodysplastic syndrome. (30th September 2019)

• Record Type: Journal Article
• Authors: Fenaux, Pierre; Platzbecker, Uwe; Ades, Lionel
• Is Part Of: British journal of haematology. Volume 189:Number 6(2020)
• Page Start: 1016
• Page End: 1027
• Publication Date: 2019-09-30
• Subjects: myelodysplastic syndromes -- erythropoietic stimulating factors -- hypomethylating agents; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.16206
• Languages: English
• ISSNs: 0007-1048

Abstract

Summary: The prognosis in Myelodysplastic syndromes (MDS), although recently refined by molecular studies, remains largely based on conventional prognostic scores [International Prognostic Scoring System (IPSS), revised IPSS], classifying patients into "lower risk" MDS (LR‐MDS) and "higher risk" MDS (HR‐MDS). In LR‐MDS, treatment mainly aims at improving cytopenias, principally anaemia, while in HR‐MDS it aims at delaying disease progression and prolonging survival. In LR‐MDS without deletion 5q, anaemia is generally treated first by erythropoietic stimulating factors, while second line treatments are currently not approved [lenalidomide, hypomethylating agents (HMA), luspatercept] or rarely indicated (antithymocyte globulin). Lenalidomide has major efficacy in LR‐MDS with deletion 5q. Allogeneic stem cell transplantation (allo‐SCT) is sometimes considered in LR‐MDS, and iron chelation can be considered when multiple red blood cell transfusions are required. Allo‐SCT is the only potentially curative treatment for HR‐MDS; however, it is rarely applicable. It is generally preceded by intensive chemotherapy (IC) or HMA in patients with excess of marrow blasts (especially if >10%). In other patients, HMA can improve survival. The role of new drugs, including venetoclax or, in case of specific mutations, IDH1 or IDH2 inhibitors, is investigated. IC is mainly indicated as a bridge to allo‐SCT, in the absence of unfavourable karyotype.