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Durable Hematological and Major Cytogenetic Response in a Patient with Isolated 20q Deletion Myelodysplastic Syndrome Treated with Lenalidomide

机译:来那度胺治疗孤立的20q缺失骨髓增生异常综合征患者的持久血液学和主要细胞遗传学反应

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摘要

Myelodysplastic syndrome (MDS) is a clonal bone marrow disorder characterized by ineffective hematopoiesis. It is characterized by peripheral blood cytopenia and significant risk of progression to acute myeloid leukemia result. Deletion of the long arm of chromosome 20 (20q deletion) is present in 3–7% of patients with MDS. Lenalidomide is an immunomodulatory agent with antiangiogenic activity. It is FDA approved for the treatment of anemia in patients with low or int-1 risk MDS with chromosome 5q deletion with or without additional cytogenetic abnormalities. Study of lenalidomide in patients with MDS without 5q deletion but other karyotypic abnormalities demonstrated meaningful activity in transfusion dependent patients; however, response of patients with isolated 20q deletion to lenalidomide is not known. We are reporting a patient with 20q deletion MDS treated with lenalidomide after he failed to respond to azacytidine; to our knowledge this is the first report of a patient with isolated 20q deletion treated with lenalidomide.
机译:骨髓增生异常综合症(MDS)是一种以造血无效为特征的克隆性骨髓疾病。它的特征是外周血细胞减少和导致发展为急性髓细胞性白血病的重大风险。 MDS患者中有3–7%存在20号染色体长臂的缺失(20q缺失)。来那度胺是一种具有抗血管生成活性的免疫调节剂。它已被FDA批准用于患有5q染色体缺失,伴有或不伴有其他细胞遗传学异常的低或int-1风险MDS患者的贫血治疗。对来那度胺治疗无5q缺失但其他核型异常的MDS患者的研究表明,在依赖输血的患者中有意义的活性;然而,尚不知道有单独20q缺失的患者对来那度胺的反应。我们报道了一名患者对来氮胞苷无反应后用来那度胺治疗的20q缺失MDS患者。据我们所知,这是接受来那度胺治疗的孤立的20q缺失患者的首次报道。

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