H. Lee Moffitt Cancer Center, Tampa, FL 33612, USA.
Curr Treat Options Oncol. 2011 Dec;12(4):354-68. doi: 10.1007/s11864-011-0165-1.
The 5q- syndrome is a myelodsyplastic syndrome (MDS) characterized by symptomatic anemia and an indolent natural history with low transformation potential. Our understanding of the molecular pathogenesis of this disease has advanced considerably, paralleled by the delineation of the relevant targets underlying selective lenalidomide sensitivity. The context in which one treats the 5q- syndrome, and all lower risk MDS, is critical. The focus of treatment should remain the amelioration of refractory cytopenias. In the 5q- syndrome, lenalidomide is the treatment of choice for patients with symptomatic anemia as it relieves the burden of transfusion dependence and iron overload in the majority of cases. We discuss herein the current understanding of the biology of the 5q- syndrome, actions of efficacy of lenalidomide and strategies for clinical management.
5q- 综合征是一种骨髓增生异常综合征（MDS），其特征为症状性贫血和惰性自然病史，转化潜能低。我们对该病的分子发病机制的理解有了很大的进展，同时也明确了选择性来那度胺敏感性的相关靶点。治疗 5q- 综合征和所有低危 MDS 的背景至关重要。治疗的重点应仍然是改善难治性血细胞减少症。在 5q- 综合征中，来那度胺是治疗有症状贫血患者的首选药物，因为它在大多数情况下可以减轻输血依赖和铁过载的负担。本文讨论了目前对 5q- 综合征生物学、来那度胺疗效作用和临床管理策略的理解。
