医学
细胞减少
来那度胺
骨髓增生异常综合症
骨髓增生性肿瘤
中性粒细胞减少症
疾病
贫血
重症监护医学
内科学
儿科
多发性骨髓瘤
骨髓
化疗
骨髓纤维化
作者
Virginia O. Volpe,Guillermo Garcia‐Manero,Rami S. Komrokji
标识
DOI:10.1016/j.clml.2023.01.001
摘要
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.
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