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Treatment Algorithms for Lower-Risk Myelodysplastic Syndrome

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Diagnosis and Management of Myelodysplastic Syndromes

Abstract

Lower-risk myelodysplastic syndromes (MDS) include patients with very low, low, and part of intermediate revised IPSS (i.e., a score below 4), characterized by a low risk of progression to AML, but often prominent anemia. Prognosis of those patients can however be worsened by the presence of myelofibrosis, somatic mutations (except SF3B1 and TET2), resistance to first- and second-line treatments, and comorbidities.

Anemia is generally the predominant cytopenia in lower-risk MDS, and its first-line treatment generally includes erythropoietic-stimulating agents (ESA), with about 50% responses and a median response duration of 2 years. Lower-risk MDS with isolated del 5q however responds better to lenalidomide, capable of achieving a cytogenetic response in 40% of the patients. About 20% of patients with this MDS subset however have TP53 mutation, with poor response to lenalidomide and a high risk of AML.

After ESA failure, second-line treatment with lenalidomide (+/− ESA), hypomethylating agents, antithymocyte globulin, and more recently luspatercept (especially in MDS with ring sideroblasts) can lead to RBC independence in a substantial proportion of lower-risk MDS patients. Allogeneic stem cell transplantation may be indicated in lower-risk MDS patients with poor risk features, including life-threatening cytopenias, myelofibrosis, unfavorable karyotype, unfavorable mutations like TP53 mutation, resistance to treatment. Most lower-risk MDS eventually require regular RBC transfusions, leading to iron overload. Iron chelators are often indicated in such patients, but serum ferritin levels which should trigger iron chelation are disputed, except in potential transplant candidates, where early chelation appears important. In a minority of lower-risk MDS, thrombocytopenia is the predominating cytopenia, which is often difficult to treat, although TPO agonist receptors can significantly improve platelets in about 50% of those patients.

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  1. Service D’hématologie Séniors, Hôpital St Louis, Assistance Publique – Hôpitaux de Paris (APHP) and Université de Paris, Paris, France

    Pierre Fenaux & Lionel Adès

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  1. Pierre Fenaux
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  2. Lionel Adès
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Correspondence to Pierre Fenaux .

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  1. Department of Hematology and Medical Oncology, Cleveland Clinic, Cleveland, OH, USA

    Aziz Nazha

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Fenaux, P., Adès, L. (2020). Treatment Algorithms for Lower-Risk Myelodysplastic Syndrome. In: Nazha, A. (eds) Diagnosis and Management of Myelodysplastic Syndromes. Springer, Cham. https://doi.org/10.1007/978-3-030-51878-3_8

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  • DOI: https://doi.org/10.1007/978-3-030-51878-3_8

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