H. Lee Moffitt Cancer Center & Research Institute, University of South Florida, 12902 Magnolia Drive, Tampa, FL 33612-9497, USA.
Curr Hematol Malig Rep. 2006 Mar;1(1):16-9. doi: 10.1007/s11899-006-0012-9.
The myelodysplastic syndromes (MDS) can be divided into "early" and "advanced" disease by evaluation of prognostic variables such as the number of cytopenias, karyotype, and percentage of myeloblasts. Patients with an isolated interstitial deletion of chromosome 5q31 represent a distinct subset who may derive particular benefit from immunomodulatory drugs. Goals of therapy for early MDS focus on hematologic improvement and maximizing quality of life. Thalidomide, the prototype of the immunomodulatory drugs, yields major erythroid responses in some patients with early MDS, but doselimiting neurologic toxicities limit its potential clinical benefit. Lenalidomide, a more potent and non-neurotoxic derivative, has shown promising results in early MDS, yielding hematologic improvement in almost half of patients and transfusion independence with cytogenetic remissions in approximately two thirds of patients harboring the chromosome 5q31 deletion.
骨髓增生异常综合征（MDS）可以通过评估细胞减少症、核型和原始细胞百分比等预后变量来分为“早期”和“晚期”疾病。孤立性 5q31 染色体间缺失的患者代表一个独特的亚组，他们可能特别受益于免疫调节药物。早期 MDS 的治疗目标侧重于血液学改善和最大程度提高生活质量。沙利度胺是免疫调节药物的原型，可使一些早期 MDS 患者产生主要的红细胞反应，但剂量限制的神经毒性限制了其潜在的临床获益。来那度胺是一种更有效且非神经毒性的衍生物，在早期 MDS 中显示出有前景的结果，几乎一半的患者出现血液学改善，大约三分之二携带 5q31 染色体缺失的患者出现输血独立性和细胞遗传学缓解。
