Department of Medical Oncology, Dana Farber Cancer Institute, Boston, MA.
Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX.
Clin Lymphoma Myeloma Leuk. 2023 Mar;23(3):168-177. doi: 10.1016/j.clml.2023.01.001. Epub 2023 Jan 13.
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.
骨髓增生异常综合征(MDS)是一种克隆性干细胞肿瘤,具有从低危疾病到危及生命的短期高危疾病的谱。疾病风险由临床和分子特征决定。大多数 MDS 患者,包括低危疾病患者,不幸因疾病相关并发症(即细胞减少症)而死亡。虽然细胞减少症在诊断早期可能较轻,可以进行监测,但最终仍需要治疗。贫血是疾病的标志,也是低危 MDS 中最常见的治疗指征。红细胞刺激剂用于一线治疗。治疗可以采用个体化方法,例如在某些特定患者中,如存在 del(5q)或环形铁幼粒细胞的患者,来那度胺和芦可替尼分别在改善细胞减少症方面非常有效。年轻患者和低增生性 MDS 也表现出对免疫抑制治疗的反应改善。对于具有高危特征或血小板减少/中性粒细胞减少的患者,可选择低甲基化药物。难治性细胞减少症仍然令人沮丧,因为选择有限,需要在我们的治疗方案中增加更多的治疗方法。