Volpe Virginia O, Garcia-Manero Guillermo, Komrokji Rami S
Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, FL.
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.
Clin Lymphoma Myeloma Leuk. 2022 Jan;22(1):1-16. doi: 10.1016/j.clml.2021.07.031. Epub 2021 Aug 2.
Myelodysplastic syndromes (MDS) are a group of heterogeneous clonal hematopoietic stem cell disorders. The 2020 Surveillance, Epidemiology, and End Results data demonstrates the incidence rate of MDS increases with age especially in those greater than 70 years of age. Risk stratification that impact prognosis, survival, and rate of acute myeloid leukemia (AML) transformation in MDS is largely dependent on revised International Prognostic Scoring System along with molecular genetic testing as a supplement. Low risk MDS typically have a more indolent disease course in which treatment is only initiated to ameliorate symptoms of cytopenias. In many, anemia is the most common cytopenia requiring treatment and erythroid stimulating agents, are considered first line. In contrast, high risk MDS tend to behave more aggressively for which treatment should be initiated rapidly with Hypomethylating Agents (HMA) being in the frontline. In those with high risk MDS and eligible, evaluation for allogeneic stem cell transplant should be considered as this is the only potential curative option for MDS. With the use of molecular genetic testing, a personalized approach to therapy in MDS has ensued. As the treatment landscape in MDS continues to flourish with novel targeted agents, we ambitiously seek to improve survival rates especially among the relapsed/refractory and transplant ineligible.
骨髓增生异常综合征(MDS)是一组异质性克隆性造血干细胞疾病。2020年监测、流行病学和最终结果数据表明,MDS的发病率随年龄增长而增加,尤其是在70岁以上人群中。影响MDS预后、生存及急性髓系白血病(AML)转化发生率的风险分层很大程度上依赖于修订后的国际预后评分系统,并辅以分子遗传学检测。低危MDS通常病程较为惰性,仅在出现血细胞减少症状时才开始治疗。在许多患者中,贫血是最常见的需要治疗的血细胞减少症,促红细胞生成剂被视为一线治疗药物。相比之下,高危MDS往往进展更为迅速,应迅速启动治疗,一线治疗药物为去甲基化药物(HMA)。对于符合条件的高危MDS患者,应考虑进行异基因干细胞移植评估,因为这是MDS唯一潜在的治愈选择。随着分子遗传学检测的应用,MDS的个性化治疗应运而生。随着MDS治疗领域不断有新型靶向药物涌现,我们雄心勃勃地致力于提高生存率,尤其是在复发/难治性及不符合移植条件的患者中。