Skill Checkup: A 72-Year-Old Woman With Myelodysplastic Syndrome Presents With Persistent Anemia and Fatigue

Emmanuel C. Besa, MD

Disclosures

April 08, 2022

In 2020, the US Food and Drug Administration approved the recombinant fusion protein luspatercept for the treatment of anemia in patients with lower-risk MDS with ring sideroblasts who have failed treatment with ESAs.

In this case, hypomethylating agent (HMA) therapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT) may be considered if disease does not respond to ESA. These treatment approaches are generally indicated for patients with MDS, symptomatic anemia, and EPO level > 500 who have not responded to therapy with luspatercept or other agents, including lenalidomide, imetelstat, or immunosuppressive therapy.

The upfront use of HMAs such as azacitidine and decitabine in patients with MDS-RARS is not a common practice, unless they have failed ESA, developed additional cytopenias, or shown evidence of disease progression. In addition, pivotal studies have not demonstrated a clear survival advantage for first-line allo-HSCT for lower-risk MDS patients, regardless of myeloablative or reduced intensity conditioning strategy.

Assessing for symptoms is critical in guiding therapy in this setting, as asymptomatic low-risk patients without significant cytopenias are candidates for active surveillance. Furthermore, when treating patients with MDS and symptomatic anemia, underlying causes of anemia must be treated, such as gastrointestinal bleeding, hemolysis, renal disease, and nutritional deficiency, before further treatment is pursued.

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