Background-Objectives:Although considered standard of care for patients with low-/intermediate-1 risk MDS and isolated del(5q), lenalidomide is not widely used in patients exhibiting additional cytogenetic abnormalities, on top of del(5)q. The aim of this study was to provide real-world evidence for the efficacy of lenalidomide in patients with del(5q), with or without additional cytogenetic abnormalities.Methods:Patients with MDS exhibiting del(5q) in the Greek National Myelodysplastic Syndromes Registry were analyzed if they had received at least one lenalidomide dose and detailed response assessment/follow-up was available.Results:Among 238 patients analyzed, 153 (64.3%) had del(5q) syndrome (Group-I), 34 (14.3%) had an isolated del(5q) abnormality but were not 5q− syndrome (Group-II), 26 (10.9%) had del(5q) plus only one additional cytogenetic abnormality (Group-III), and 25 (10.5%) had del(5q) plus >1 additional abnormality (Group-IV). Among 218 (91.6%) evaluable patients, a major response was achieved by 146 (67.0%) patients, 114/146 (78.1%) in Group-I, 18/31 (58.1%) in Group-II, 10/20 (50.0%) in Group-III, and 4/21 (19.0%) in Group-IV. Overall, hematological response was seen in 177/218 (81.2%) patients, even among those with an excess of bone marrow blasts/frank acute myeloid leukemia. Duration of response was comparable between the four patient groups. A complete cytogenetic response was achieved by 38.0% overall, more commonly in Group-I (42.3%) and -III (35.7%). Transfusion-independent patients and those with a higher MCV or lower marrow blast cells at baseline had a higher probability of achieving a major response. With multivariate analysis, factors associated with overall survival were age, performance status, transfusion dependence, and marrow blast cell percentage at treatment start, as well as time from initial diagnosis to lenalidomide start.Conclusions:Lenalidomide was highly effective in patients with the del(5)q syndrome and also in those with isolated del(5)q, other than del(5)q syndrome, or those exhibiting del(5)q plus only one additional cytogenetic abnormality, not affecting chromosome 7.
背景与目的：来那度胺虽被视为低危/中危-1级骨髓增生异常综合征（MDS）伴单纯5号染色体长臂缺失[del(5q)]患者的标准治疗方案，但对于在del(5q)基础上存在额外细胞遗传学异常的患者，其应用尚未普及。本研究旨在为伴或不伴额外细胞遗传学异常的del(5q)患者使用来那度胺的疗效提供真实世界证据。 方法：对希腊国家骨髓增生异常综合征登记系统中存在del(5q)的MDS患者进行分析，纳入标准为至少接受过一剂来那度胺治疗且具有详细疗效评估及随访记录。 结果：在分析的238例患者中，153例（64.3%）为del(5q)综合征（第一组），34例（14.3%）为单纯del(5q)异常但不满足5q-综合征诊断标准（第二组），26例（10.9%）为del(5q)合并仅一种额外细胞遗传学异常（第三组），25例（10.5%）为del(5q)合并超过一种额外异常（第四组）。在218例（91.6%）可评估患者中，146例（67.0%）获得主要治疗反应，其中第一组114/146例（78.1%）、第二组18/31例（58.1%）、第三组10/20例（50.0%）、第四组4/21例（19.0%）。总体血液学反应率达177/218例（81.2%），即使在骨髓原始细胞比例过高或已进展为急性髓系白血病的患者中仍观察到反应。四组患者的反应持续时间具有可比性。完全细胞遗传学缓解率为38.0%，在第一组（42.3%）和第三组（35.7%）中更为常见。基线时无需输血、平均红细胞体积较高或骨髓原始细胞比例较低的患者获得主要治疗反应的概率更高。多变量分析显示，与总生存期相关的因素包括年龄、体能状态、输血依赖情况、治疗起始时骨髓原始细胞百分比以及从初诊到来那度胺治疗开始的时间间隔。 结论：来那度胺对del(5q)综合征患者疗效显著，对于不符合del(5q)综合征诊断标准的单纯del(5q)患者，以及del(5q)合并仅一种额外细胞遗传学异常（非7号染色体异常）的患者同样具有良好疗效。
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