Treatment options for anemia in patients with low‑risk myelodysplastic syndrome

04/2020

MUDr. Petra Bělohlávková, Ph.D.

IV. interní hematologická klinika LF UK a FN, Hradec Králové

SUMMARY

The prognosis and treatment patients with myelodysplastic syndrome (MDS) depend on the conventional determination of IPSS (International Prognostic Scoring System) or its revised form IPSS-R, which divide patients into lower and higher risk groups. The risk of transformation into acute myeloid leukemia is a major problem in high-risk patients, while anemia represents the major issues for low-risk MDS patients. Improving erythropoiesis and eliminating the symptoms of anemia is a major therapeutic goal in this group of patients. Treatment by erythropoiesis-stimulating agents achieves a response rate in 50% of patients, but the median duration of response is between 15 to 18 months. Therapy by lenalidomide is indicated in patients with deletion 5 chromosome (MDS 5qsyndrome). The erythrocyte response is usually achieved in 70-80% of patients and the achievement of transfusion dependence occurs in 60-70% of patients. The newly approved drug this year is luspatercept, which is especially effective in forms of MDS with ring sideroblasts or SF3B1 mutation. Roxadustat and imetelstat are promising drugs for the treatment of anemia in low-risk MDS patients.

Key words

erythropoiesis-stimulating agents, myelodysplastic syndromes, lenalidomide, low-risk disease, luspatercept