SOHO State of the Art Updates and Next Questions: Treatment of Lower Risk Myelodysplastic Syndromes

MDS is a clonal stem cell neoplasm with a spectrum from lower risk disease to short term life threatening higher risk disease. The disease risk is dictated by clinical and molecular features. Majority of MDS patients including lower risk disease unfortunately succumb from disease related complications namely cytopenia. While cytopenias may be mild early upon diagnosis and can be surveilled, ultimately treatment is required. Anemia is the hall mark of disease and most common indication to treat in lower risk MDS. Erythroid stimulating agents are used in the first line setting. Treatment can be a personalized approach as in select patient such as patients with del(5q) and those with ringed sideroblasts, lenalidomide, and luspatercept can be extremely effective respectively at improving cytopenias. Younger patients and hypoplastic MDS have also shown and improved response to immunosuppressive therapy. Hypomethylating agents can be option for patients with higher risk features or thrombocytopenia/neutropenia. Refractory cytopenias still poses frustration as options are limited and there is need to add more treatments to our armamentarium.

Automated summary

MDS is a stem cell neoplasm with varying risk levels, and most patients suffer from complications related to cytopenia. Anemia is a hallmark of the disease and commonly requires treatment. Different approaches, such as erythroid stimulating agents and targeted drugs, can effectively improve cytopenias in specific MDS patient populations. However, refractory cytopenias remain a challenge, and more treatment options are needed.