Clinical decision‐making and treatment of myelodysplastic syndromes

The 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) constitute a profoundly heterogeneous myeloid malignancy with a common origin in the hemopoietic stem cell compartment. Consequently, patient management and treatment are as heterogeneous. Decision-making includes identifying risk, symptoms, and options for the individual patient and to make a risk-benefit analysis. The only potential cure is allogeneic stem cell transplantation and albeit the fraction of transplanted MDS patients increase over time due to a better management and increased donor availability a majority are not eligible for this intervention. Current challenges encompass to decrease the relapse risk, the main cause of HSCT failure. Hypomethylating agents (HMA) constitute first-line treatment for higher-risk MDS. Combinations with other drugs as first-line treatment has to date not proven more efficacious than monotherapy, although combinations approved for acute myeloid leukemia acute myeloid leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… , including venetoclax venetoclax: Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines to treat these conditions. currently are under evaluation and often used as rescue treatment. The treatment goal for lower-risk MDS is to improve cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. , mainly 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. , quality-of-life, and possibly overall survival. Erythropoiesis-stimulating agents (ESAs) constitute first-line treatment for anemia and have better and more durable responses if initiated before the onset of a permanent transfusion need. Treatment in case of ESA failure or ineligibility should be tailored to the main disease mechanism; immunosuppression for hypoplastic MDS without high-risk genetics, 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… for low-risk del(5q) MDS, and luspatercept for MDS with ring sideroblasts. Approved therapeutic options are still more scarce for MDS than for most other hematological malignancies. Better tools to match disease biology with treatment, i.e. applied precision medicine is needed to improve patient outcome.