Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS) - Review

Sep 24, 2026

Myelodysplastic neoplasms (MDS) are described as a heterogeneous set of clonal hematologic disorders marked by morphologic dysplasia, ineffective hematopoiesis, and cytopenia. Recent updates in 2022 advance classification through the WHO 5th edition and the International Consensus Classification (ICC), integrating morphologic, clinical, and genomic data. Improved risk stratification is enabled by IPSS-M, and lower-risk care prioritizes cytopenia control, particularly anemia, using newer agents such as luspatercept and decitabine/cedazuridine, alongside ongoing phase 3 trials.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages20

Reading Time50 min

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Introduction

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Review Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS) Josephine Lucero 1, Salman Al-Harbi 1 and Karen W. L. Yee 1,2, * Citation: Lucero, J.; Al-Harbi, S.; Yee, K.W.L. Management of Patients with Lower-Risk Myelodysplastic Neoplasms (MDS) . Curr. Oncol. 2023, 30, 6177–6196. [https://doi.org/](https://doi.org/)[ ](https://doi.org/)[10.3390/curroncol30070459](10.3390/curroncol30070459) Received: 11 May 2023 Revised: 15 June 2023 Accepted: 25 June 2023 Published: 27 June 2023 Copyright: © 2023 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license ([https://](https://)[ ](https://)[creativecommons.org/licenses/by/](creativecommons.org/licenses/by/)[ ](creativecommons.org/licenses/by/)[4.0/](4.0/)) . 1 Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University Health Network, 700 University Avenue, 6th Floor, Toronto, ON M5G 1Z5, Canada; [josephine.lucero@uhn.ca](josephine.lucero@uhn.ca) (J.L.); [salman.alharbi@uhn.ca](salman.alharbi@uhn.ca) (S.A.-H.) 2 Division of Hematology, University of Toronto, Toronto, ON M5S 3H2, Canada * Correspondence: [karen.yee@uhn.ca](karen.yee@uhn.ca); Tel.: +1-416-946-4495; Fax: +1-416-946-4563 Abstract: Myelodysplastic neoplasms (MDS) are a heterogenous group of clonal hematologic disorders characterized by morphologic dysplasia, ineffective hematopoiesis, and cytopenia. In the past year, the classification of MDS has been updated in the 5th edition of the World Health Organization (WHO) Classification of Haematolymphoid Tumours and the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemia with incorporation of morphologic, clinical, and genomic data. Furthermore, the more comprehensive International Prognostic Scoring System-Molecular (IPSS-M) allows for improved risk stratification and prognostication. These three developments allow for more tailored therapeutic decision-making in view of the expanding treatment options in MDS. For patients with lower risk MDS, treatment is aimed at improving cytopenias, usually anemia. The recent approval of luspatercept and decitabine/cedazuridine have added on to the current armamentarium of erythropoietic stimulating agents and lenalidomide (for MDS with isolated deletion 5q). Several newer agents are being evaluated in phase 3 clinical trials for this group of patients, such as imetelstat and oral azacitidine. This review provides a summary of the classification systems, the prognostic scores and clinical management of patients with lower risk MDS. Keywords: myelodysplastic neoplasm; prognostic scoring system; lenalidomide; luspatercept; antithymocyte globulin; hypomethylating agent; imetelstat 1. Introduction Myelodysplastic neoplasms (MDS) are a heterogenous group of clonal hematologic disorders characterized by morphologic dysplasia, ineffective hematopoiesis, and cytopenias. With recent updates to classiﬁcation and prognostic systems, alongside improvements in treatment, therapeutic strategies will become more personalized in the coming years. We review the recent changes to the classiﬁcation systems, prognostic scores, and discuss the expanding therapeutic armamentarium for treating adult patients with lower-risk (LR) MDS as deﬁned by IPSS low and intermediate-1-risk and revised IPSS (IPSS-R) very low, low and intermediate-risk (􀀔3.5) categories. 2. Classiﬁcations Two updated classiﬁcations of myeloid malignancies were introduced in 2022: (a) the 5th edition of the World Health Organization classiﬁcation (WHO 2022) and (b) the International Consensus Classiﬁcation (ICC 2022) . Both continue to expand the disease types that are genetically deﬁned (Table 1) [1–3] . The classiﬁcations attempt to further reﬁne and subtype this heterogeneous disease. WHO 2022 introduced the term myelodysplastic neoplasm (abbreviated

FAQs

What characterizes myelodysplastic neoplasms (MDS) in this review?

MDS are characterized by morphologic dysplasia, ineffective hematopoiesis, and cytopenia in a clonal hematologic context.

Which classification and prognostic tools are discussed for lower-risk MDS?

The review highlights updated WHO (2022) and ICC (2022) classification approaches and discusses IPSS-M for improved risk stratification and prognostication.

How is treatment generally approached for patients with lower-risk MDS?

For lower-risk MDS, treatment aims to improve cytopenias, commonly anemia, and incorporates agents such as luspatercept and decitabine/cedazuridine, with additional options including erythropoiesis-stimulating agents and lenalidomide for isolated del(5q).

What kinds of therapies are being evaluated for lower-risk MDS?

The review notes ongoing evaluation of newer agents in phase 3 trials, including imetelstat and oral azacitidine, alongside established therapies.

Sep 24, 2026

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