Abstract
Myelodysplastic syndrome is a clonal disease of the hemopoiesis. In patients with a low risk of disease progression or transition to acute myeloid leukemia, cytopenia, especially anemia, is the dominant problem.
It occurs in up to 90% of patients. Deeper anemia, especially in transfusion-dependent patients, significantly reduces quality of life, increases morbidity and mortality.
So far, we could use as a treatment only transfusions or erythropoietins for most patients (except for patients with 5q-syndrome, who are excellent responders to lenalidomide therapy). However, erythropoietins are not very effective in patients with already developed transfusion dependence.
Therefore, the new drug luspatercept, an inhibitor of the TGF-β pathway, is of great benefit in the treatment of these patients, resulting in the elimination of the need for transfusions in up to 50% of patients, a significant improvement in quality of life and, as recently demonstrated, prolonged survival. We present a case report of a patient where this agent was successfully used as a fourth line therapy.