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摘要: 目的 探讨维奈克拉联合去甲基化药物治疗髓系肉瘤的有效性及安全性。方法 纳入5例髓系肉瘤患者,其中初诊患者2例,复发难治患者2例,造血干细胞移植后1例。3例给予维奈克拉联合地西他滨,2例给予维奈克拉联合阿扎胞苷,分析临床疗效及安全性。结果 5例患者治疗后病灶超声影像学消失,4例伴有骨髓侵犯均获得完全缓解,不良反应主要为骨髓抑制。截至随访终止日期,4例患者存活,1例患者后因本病复发死亡。结论 维奈克拉联合去甲基化药物治疗髓系肉瘤可取得较好的临床疗效,且无明显不良反应。Abstract: Objective To evaluate the efficacy and safety of venetoclax and hypomethylating agents in the treatment of myeloid sarcoma.Methods The clinical data of 5 patients with myeloid sarcoma treated with venetoclax and hypomethylating agents were retrospectively analyzed, among them one patient was isolated myeloid sarcoma after allogeneic hematopoietic cell transplantation, the others were accompanied by bone marrow involvement.Results All the patients got lesions disappeared, 4 cases with bone marrow invasion achieved complete remission, and the main adverse events were bone marrow suppression. By the end of follow-up, four patients were alive and one patient died of subsequent recurrence.Conclusion The combination of venetoclax and hypomethylating agents can achieve good clinical efficacy in the treatment of myeloid sarcoma, without significant adverse events.
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表 1 5例髓系肉瘤患者的基本临床特征
序号 性别 年龄/岁 AML分层(ELN) 突变或融合基因 WBC/(×109/L) 治疗方案 复发难治AML 是否移植 侵犯部位 骨髓侵犯 免疫组织化学结果 1 女 39 高危(M4) 复杂核型 6.83 IA、HAG 是 否 颈部淋巴结 原幼47% MPO+、CD34+、C117+、BCL2+、CD3-、CD20-、Ki-67(60%) 2 男 43 低危(M2) CEBPA双突变 104.00 IAC、中剂量Ara-c、中剂量Ara-c 是 否 肺部 原幼10% MPO+、CD34+、CD117+、CD3-、CD20-、Ki-67(40%~60%) 3 女 40 高危(M5) FLT3-ITD (AR=0.67) 19.57 IA、索拉非尼+HAA、FA、HSCT 否 是 眼眶内直肌 未累及 MPO+、CD34+、CD1a-、CD3-、CD19-、CD15dim、Ki-67(53%) 4 男 45 中危(M5) 阴性 2.20 初诊 否 否 颈部淋巴结 原幼93% CD45+、MPO+、CD117+、CD34+、CD3-、Ki-67(>70%) 5 女 52 中危(M4) DNMT3A、IDH2 3.94 初诊 否 否 皮肤 原幼58% MPO+、CD34+、BCL2+、CD3-、CD22-、Ki-67(70%) IA:去甲氧柔红霉素+阿糖胞苷;HAG:高三尖杉酯碱+阿糖胞苷+粒细胞集落刺激因子;IAC:去甲氧柔红霉素+阿糖胞苷+克拉屈滨;Ara-c:阿糖胞苷;HAA:高三尖杉酯碱+阿克拉霉素+阿糖胞苷;FA:氟达拉滨+阿糖胞苷。 表 2 5例髓系肉瘤患者的治疗及转归
序号 治疗方案 骨髓评估 髓外表现 后续治疗 随访时间/月 转归(死亡原因) 1 VEN+AZA MRD阴性 CR 中剂量Ara-c、Haplo-HSCT 10 死亡(复发、感染) 2 VEN+AZA MRD阴性 CR VEN+AZA、Haplo-HSCT 12 存活 3 VEN+DAC 未评估 PR DLI、VEN+DAC、放疗、VEN+DAC、DLI 15 存活 4 VEN+DAC MRD阴性 CR DAC+中剂量Ara-c、中剂量Ara-c、DAC 15 存活 5 VEN+DAC MRD阴性 CR DAC+中剂量Ara-c、中剂量Ara-c+ 去甲氧柔红霉素、Haplo-HSCT 18 存活 -
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