Stanford Comprehensive Cancer Center , USA.
J Natl Compr Canc Netw. 2011 Jan;9(1):30-56. doi: 10.6004/jnccn.2011.0005.
These suggested practice guidelines are based on extensive evaluation of the reviewed risk-based data and indicate useful current approaches for managing patients with MDS. Four drugs have recently been approved by the FDA for treating specific subtypes of MDS: lenalidomide for MDS patients with del(5q) cytogenetic abnormalities; azacytidine and decitabine for treating patients with higher-risk or nonresponsive MDS; and deferasirox for iron chelation of iron overloaded patients with MDS. However, because a substantial proportion of patient subsets with MDS lack effective treatment for their cytopenias or for altering disease natural history, clinical trials with these and other novel therapeutic agents along with supportive care remain the hallmark of management for this disease. The role of thrombopoietic cytokines for management of thrombocytopenia in MDS needs further evaluation. In addition, further determination of the effects of these therapeutic interventions on the patient's quality of life is important.(116,119,120,128,129) Progress toward improving management of MDS has occurred over the past few years, and more advances are anticipated using these guidelines as a framework for coordination of comparative clinical trials.
这些建议实践指南是基于对已审查的基于风险的数据进行广泛评估而制定的,这些数据表明了目前管理 MDS 患者的有用方法。最近,有四种药物已获得美国食品药品监督管理局(FDA)批准,用于治疗特定类型的 MDS:来那度胺用于治疗具有 del(5q)细胞遗传学异常的 MDS 患者;阿扎胞苷和地西他滨用于治疗高危或无反应性 MDS 患者;去铁酮用于治疗 MDS 伴铁过载的患者进行铁螯合治疗。然而,由于相当一部分 MDS 患者亚群缺乏针对其血细胞减少症或改变疾病自然史的有效治疗方法,因此,这些药物和其他新型治疗药物的临床试验以及支持性护理仍然是治疗这种疾病的主要方法。血小板生成素在 MDS 血小板减少症管理中的作用仍需进一步评估。此外,进一步确定这些治疗干预措施对患者生活质量的影响也很重要。(116,119,120,128,129) 过去几年中,在改善 MDS 管理方面已经取得了进展,并且预计将根据这些指南作为协调比较临床试验的框架,取得更多进展。