Myelodysplastic Syndromes Clinical Practice Guidelines (ESMO, 2021)

European Society of Medical Oncology

This is a quick summary of the guidelines without analysis or commentary. For more information, go directly to the guidelines by clicking the link in the reference.

February 03, 2021

Guidelines on the diagnosis and treatment of myelodysplastic syndromes were published in November 2020 by the European Society of Medical Oncology.[1]

The diagnosis of myelodysplastic syndromes (MDS) is based on the blood count, the marrow aspirate, and the marrow karyotype.

A bone marrow biopsy is recommended at the time of diagnosis.

Molecular biology is useful for the diagnosis of MDS if the blood and marrow tests are not conclusive.

Flow cytometry of blood and marrow cells is useful for the diagnosis of MDS in those who have experience with this testing.

The international prognostic scoring system, revised version( IPSS-R) is required for prognostic evaluation of MDS.

Molecular analysis may add prognostic value, especially for TP53 [in del(5q) MDS] and SF3B1 mutations in patients who have <5% blasts.

Azacitidine is recommended for patients who have higher-risk MDS without major comorbidities who are not immediately eligible for allogeneic stem cell transplantation (allo-SCT).

Acute myeloid leukemia-like chemotherapy (AML-like ChT) is recommended for patients who are considered fit (generally <70 yr) with favorable cytogenetics (according to IPSS) and marrow blasts ≥10%, preferably as a bridge to allo-SCT.

Allo-SCT should be proposed to all higher-risk MDS patients <70 yr who do not have major comorbidities and have a donor.

Reducing the marrow blast count before allo-SCT with AML-like ChT or hypomethylating agents (HMAs) is generally considered when marrow blasts are ≥10%, especially for non-myeloablative allo-SCT.

Erythropoiesis-stimulating agents (ESAs) (especially erythropoietin [EPO] alpha) are recommended as first-line treatment of anemia in lower-risk MDS in patients without del(5q).

The most effective drug for transfusion-dependent anemia of lower-risk MDS with del(5q), is lenalidomide (LEN).

After ESA failure, anti-thymocyte globulin (ATG) (± cyclosporine) has efficacy in specific younger patient cohorts of lower-risk MDS.

Luspatercept is recommended after ESA failure in red blood cell transfusion-dependent MDS-RS (ring sideroblasts).

Other second-line treatments for anemia after ESA failure include LEN with or without ESA and HMAs (not approved in Europe for this indication).

Thrombopoietin receptor agonists (TPO-RAs) (romiplostim, eltrombopag) have some efficacy in severe thrombocytopenia, but they are not approved for MDS and should only be used in patients with marrow blasts <5%.

In patients who have transfusion iron overload, iron chelation is strongly recommended in patients who are candidates for allo-SCT.

In non-transplant candidates with lower-risk MDS, iron chelation is strongly recommended in patients who have major iron overload (eg, significantly reduced cardiac T2∗); however, its use is more controversial in patients who do not have major iron overload.

For more information, please go to Myelodysplastic Syndrome.

For more Clinical Practice Guidelines, please go to Guidelines.

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