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A decade of progress in myelodysplastic syndrome with chromosome 5q deletion.
Leukemia ( IF 11.4 ) Pub Date : 2018-Jul-01 , DOI: 10.1038/s41375-018-0029-9
Alan List , Benjamin L. Ebert , Pierre Fenaux

There are few instances in oncology where reciprocal clinical and laboratory translation studies have accelerated the understanding of disease biology and treatment more so than the decade following the Food and Drug Administration (FDA) approval of lenalidomide (RevlimidTM; Celgene Corporation, Summit, NJ, USA) for the treatment of patients with myelodysplastic syndrome (MDS) and chromosome 5q deletion (del(5q)). Lenalidomide was approved by the FDA in December 2005 on the merits of a multicenter phase 2 study, which demonstrated sustained and prolonged transfusion independence in the majority of participants. Since then, del(5q) MDS has emerged as one of the best characterized bone marrow malignancies and, in particular, has raised our understanding as to how allelic haplodeficiency underlies both its hematological phenotype and the selective sensitivity to lenalidomide by virtue of synthetic lethality. Herein, we review the clinical and biological discoveries that have advanced our understanding of del(5q) MDS and its treatment since its approval by United States and European regulatory agencies.

中文翻译:

染色体5q缺失在骨髓增生异常综合症中的十年进展。

在食品和药物管理局(FDA)批准来那度胺(Revlimid TM; Celgene Corporation,Summit,NJ,美国)用于治疗患有骨髓增生异常综合症(MDS)和5q号染色体缺失(del(5q))的患者。来那度胺在2005年12月通过了一项多中心2期研究的优点而被FDA批准,该研究在大多数参与者中证明了持续和长期的输血独立性。从那时起,del(5q)MDS成为最典型的骨髓恶性肿瘤之一,尤其是提高了我们对等位基因单倍体缺乏症是其血液学表型和来那度胺选择性致敏性的基础的认识,而这种致敏性是由于合成杀伤力而产生的。在此,我们回顾了自美国和欧洲监管机构批准以来,对del(5q)MDS及其治疗方法加深了了解的临床和生物学发现。
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