Treatment options for lower-risk myelodysplastic syndromes. Where are we now?

Myelodysplastic syndromes Myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (MDS) are a spectrum of clonal stem-cell disorders characterized clinically by bone-marrow failure. Resultant cytopenias are responsible for significant mortality and decreased quality of life in patients with MDS. In patients with low-risk MDS (LR-MDS), anemia anemia: (uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness. is the most common cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. and erythropoiesis-stimulating agents (ESA) are usually used as first-line therapy. Those patients who become refractory refractory: Not responsive to treatment or cure. For example, refractory anemia is a low red blood cell count that doesn't respond to standard treatments. to ESA have a poor survival. Available treatment options such as lenalidomide lenalidomide: Lenalidomide is a capsule that is taken by mouth. It is approved for treating low-risk, transfusion-dependent myelodysplastic syndrome (MDS) patients with an abnormality of chromosome 5q. It is currently in clinical trials to test its efficacy with a broader range of MDS patients. For some MDS… , hypomethylating agents, and immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… can provide some hematologic response among selected subsets of patients, however durable responses are limited, and these agents can carry significant adverse effects. Chronic transfusions help to alleviate symptoms of anemia but still carry risks associated with transfusion and iron overload iron overload: A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated. . Luspatercept, recently approved for those LR-MDS with ring sideroblasts refractory to ESA, was found to have an improvement in transfusion independence transfusion independence: No longer needing any type of blood transfusion. with a well-tolerated safety profile. While anemia is the most common cytopenia, thrombocytopenia thrombocytopenia: (throm-buh-sie-tuh-PEE-nee-uh) A condition in which there is a shortage of platelets in the bloodstream. This results in a low platelet count. Bleeding and bruising often occur with thrombocytopenia. and neutropenia neutropenia: (noo-truh-PEE-nee-uh) A condition in which there is a shortage of neutrophils in the bloodstream. Neutrophils are a type of white blood cell. This results in a low white blood cell count. management is challenging and the co-occurrence of these cytopenias with anemia may dictate the choice of therapy. In this article, we review LR-MDS and discuss the optimal use of current treatment options and explore new therapeutic options on the horizon.