Sekeres Mikkael A, List Alan
H Lee Moffitt Cancer Center & Research Institute, University of South Florida, 12902 Magnolia Drive, Tampa, FL 33612-9497, USA.
Curr Hematol Rep. 2005 May;4(3):182-5.
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 dose-limiting 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）可通过评估诸如血细胞减少数量、核型和成髓细胞百分比等预后变量分为“早期”和“晚期”疾病。孤立性5号染色体长臂31区间质缺失的患者代表了一个独特的亚组，他们可能从免疫调节药物中获得特别的益处。早期MDS的治疗目标集中在血液学改善和最大限度地提高生活质量。沙利度胺作为免疫调节药物的原型，在一些早期MDS患者中产生主要的红系反应，但剂量限制性神经毒性限制了其潜在的临床益处。来那度胺是一种更有效且无神经毒性的衍生物，在早期MDS中已显示出有前景的结果，几乎一半的患者实现了血液学改善，在大约三分之二携带5号染色体长臂31区缺失的患者中实现了脱离输血并伴有细胞遗传学缓解。
