Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2024 Jun;25(6):752-768.
doi: 10.1007/s11864-024-01217-0. Epub 2024 May 30.

Treatment of Anemia in Lower-Risk Myelodysplastic Syndrome

Muriel R Battaglia #  1 , Joseph Cannova #  2 , Rafael Madero-Marroquin #  2 , Anand A Patel  3
Affiliations
Review

Treatment of Anemia in Lower-Risk Myelodysplastic Syndrome

Muriel R Battaglia et al. Curr Treat Options Oncol. 2024 Jun.

Erratum in

Abstract

A majority of patients with lower-risk myelodysplastic syndrome (MDS) will present with or develop anemia. Anemia in MDS is associated with decreased quality of life and may correlate with decreased progression-free survival and overall survival. In this state of the art review we summarize current risk stratification approaches to identify lower-risk MDS (LR-MDS), the natural history of the disease, and meaningful clinical endpoints. The treatment landscape of LR-MDS with anemia is also rapidly evolving; we review the role of supportive care, erythropoietin stimulating agents, lenalidomide, luspatercept, hypomethylating agents (HMAs), and immunosuppressive therapy (IST) in the management of LR-MDS with anemia. In patients with deletion 5q (del5q) syndrome lenalidomide has both efficacy and durability of response. For patients without del5q who need treatment, the management approach is impacted by serum erythropoietin (EPO) level, SF3B1 mutation status, and ring sideroblast status. Given the data from the Phase III COMMANDS trial, we utilize luspatercept in those with SF3B1 mutation or ring sideroblasts that have an EPO level < 500 U/L; in patients without an SF3B1 mutation or ring sideroblasts there is equipoise between luspatercept and use of an erythropoietin stimulating agent (ESA). For patients who have an EPO level ≥ 500 U/L or have been previously treated there is not a clear standard of care. For those without previous luspatercept exposure it can be considered particularly if there is an SF3B1 mutation or the presence of ring sideroblasts. Other options include HMAs or IST; the Phase III IMERGE trial supports the efficacy of the telomerase inhibitor imetelstat in this setting and this may become a standard option in the future as well.

Keywords: Anemia; Myelodysplastic neoplasm; Myelodysplastic syndrome.

PubMed Disclaimer

Similar articles

See all similar articles

References

References and Recommended Reading

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
    1. Cazzola M. Myelodysplastic Syndromes. N Engl J Med. 2020;383:1358–74. - PubMed - DOI
    1. Sébert M. Next-generation therapy for lower-risk MDS. Hematology Am Soc Hematol Educ Program. 2023;2023:59–64. - PubMed - DOI
    1. Arber DA, Orazi A, Hasserjian RP, Borowitz MJ, Calvo KR, Kvasnicka H-M, et al. International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140:1200–28. - PubMed - PMC - DOI
    1. Loghavi S, Kanagal-Shamanna R, Khoury JD, Medeiros LJ, Naresh KN, Nejati R, et al. Fifth Edition of the World Health Classification of Tumors of the Hematopoietic and Lymphoid Tissue: Myeloid Neoplasms. Mod Pathol. 2024;37: 100397. - PubMed - DOI
    1. Jiang M, Chen M, Liu Q, Jin Z, Yang X, Zhang W. SF3B1 mutations in myelodysplastic syndromes: A potential therapeutic target for modulating the entire disease process. Front Oncol. 2023;13:1116438. - PubMed - PMC - DOI

LinkOut - more resources