Treatment of Anemia in Transfusion‐Dependent and Non‐Transfusion‐Dependent Lower‐Risk MDS: Current and Emerging Strategies

Myelodysplastic syndromes (MDS) are a heterogeneous group of bone marrow disorders with a highly diverse clinical course. For lower‐risk MDS patients, therapeutic objectives aim to correct chronic anemia and improve/maintain health‐related quality of life (HRQoL). However, disease burden is often insufficiently recognized, and although some patients do not respond/lose response to standard treatment, many are treated late. This is the case for non‐transfusion‐dependent patients with symptomatic anemia, in whom delayed treatment initiation may lead to unnecessary morbidity. Current active treatment options for lower‐risk MDS are limited. Standard care for lower‐risk 5q deletion [del(5q)] MDS patients with anemia remains supportive, consisting of red blood cell (RBC) transfusions, iron chelation therapy, and treatment with erythropoiesis‐stimulating agents (ESAs) in the case of low serum erythropoietin levels. Response rates to ESAs range from 15% to 63%, whereas 56% to 67% of patients with del(5q) MDS achieve RBC transfusion independence with lenalidomide. Treatment options for patients’ refractory to ESAs and/or lenalidomide, however, are limited. Frequent transfusions are associated with profound clinical, HRQoL, and economic consequences for transfusion‐dependent patients. This review focuses on the multiple unmet clinical needs that exist in the treatment of anemia associated with lower‐risk MDS and the current and future treatment options that may improve disease management and patient outcomes.