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骨髓增生异常综合征治疗的最新进展。

An update on the treatment of myelodysplastic syndromes.

作者信息

Kurtin Sandra E, Demakos Erin P

机构信息

Arizona Cancer Center and College of Medicine, University of Arizona, Tucson, AZ, USA.

出版信息

Clin J Oncol Nurs. 2010 Jun;14(3):E29-44. doi: 10.1188/10.CJON.E24-E39.

Abstract

Myelodysplastic syndromes (MDS) are a group of heterogeneous clonal disorders of myeloid hematopoietic stem cells affecting about 300,000 people worldwide. Ineffective hematopoiesis and clonal proliferation result in significant cytopenias in affected individuals. Patients are categorized into risk groups (i.e., low, intermediate [1 and 2], and high) based on severity of cytopenias, cytogenetic abnormalities, and the presence of bone marrow blasts. The only potentially curative treatment for MDS is hematopoietic stem cell transplantation, which often is not an option because of advanced age at diagnosis (median age = 76 years). Several alternative treatments to hematopoietic stem cell transplantation show great promise. For low- and intermediate-1-risk MDS, the novel antitumor immunomodulatory agent lenalidomide is approved for patients with del(5q), and two different hypomethylating agents, azacitidine and decitabine, are approved for intermediate-2- and high-risk MDS. Trial results have increased the understanding of these treatments, alone or in combination with other therapies. Effective treatment often requires at least three to six months to achieve a clinical response. In the meantime, or in addition to active therapy, supportive care has a positive effect on quality of life. Greater understanding of the factors affecting MDS treatment options will assist oncology nurses in facilitating the optimal combination of treatment, supportive care, and management of adverse events.

摘要

骨髓增生异常综合征(MDS)是一组异质性的髓系造血干细胞克隆性疾病,全球约有30万人受其影响。无效造血和克隆性增殖导致患病个体出现明显的血细胞减少。根据血细胞减少的严重程度、细胞遗传学异常以及骨髓原始细胞的存在情况,患者被分为不同的风险组(即低危、中危[1和2]、高危)。MDS唯一可能治愈的治疗方法是造血干细胞移植,但由于诊断时患者年龄较大(中位年龄 = 76岁),这通常不是一个可行的选择。造血干细胞移植的几种替代治疗方法显示出巨大的前景。对于低危和中危1的MDS,新型抗肿瘤免疫调节剂来那度胺被批准用于伴有del(5q)的患者,两种不同的去甲基化药物阿扎胞苷和地西他滨被批准用于中危2和高危的MDS。试验结果增进了对这些治疗方法单独使用或与其他疗法联合使用的了解。有效的治疗通常需要至少三到六个月才能产生临床反应。与此同时,或者除了积极治疗之外,支持性护理对生活质量有积极影响。对影响MDS治疗选择的因素有更深入的了解,将有助于肿瘤学护士促进治疗、支持性护理和不良事件管理的最佳组合。

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