The recently approved Bcl-2 inhibitor venetoclax (VEN) has achieved promising outcomes in new and relapsed/refractory (R/R) acute myeloid leukemia (AML). Although its use is not well-established in R/R AML with extramedullary disease (EMD), some reports have shown promising outcomes. We retrospectively analyzed 11 patients of R/R AML with EMD [with (n=4) or without (n=7) concurrent marrow involvement], who were treated with VEN plus decitabine (n=9) or low-dose cytarabine (n=2) between May 2020 and October 2020 in Seoul St. Mary's Hospital. The median number of prior treatment lines was 3 (1-6) and most (n=9, 81.8%) had multiple sites of EMD. Nine patients (81.8%) received concurrent therapy for EM involvement sites with radiotherapy (RT) (n=4), surgery (n=1) and both of them (n=4). Among 11 patients, four patients (36.4%) had either marrow or EM responses to VEN-combination; EM response was seen in one patient (9.1%, partial response) who had received concurrent radiotherapy (25Gy, 10fx) during 1st cycle of VEN-combination, and other three patients showed marrow response without EM response. After median follow-up of 27.0 months, the median overall survival was estimated to be 5.4 months. To conclude, VEN-combination regimens have shown only modest efficacy in EM recurrence of AML with little impact on eliciting EM response.

中文翻译：

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Venetoclax 联合方案治疗急性髓系白血病伴髓外复发的疗效有限。

最近批准的 Bcl-2 抑制剂 venetoclax (VEN) 在治疗新发和复发/难治性 (R/R) 急性髓系白血病 (AML) 方面取得了可喜的成果。尽管其在伴有髓外疾病 (EMD) 的 R/R AML 中的应用尚未得到充分证实，但一些报告显示了有希望的结果。我们回顾性分析了 11 例患有 EMD 的 R/R AML 患者[有 (n=4) 或没有 (n=7) 并发骨髓受累]，他们接受了 VEN 加地西他滨 (n=9) 或低剂量阿糖胞苷 (n=9) 治疗。 =2) 2020 年 5 月至 2020 年 10 月期间在首尔圣母医院。既往治疗线的中位数为 3 (1-6)，大多数（n=9，81.8%）有多个 EMD 部位。9 名患者 (81.8%) 接受了放射治疗 (RT) (n=4)、手术 (n=1) 和两者同时治疗 (n=4) 的 EM 受累部位同步治疗。在 11 名患者中，4 名患者 (36.4%) 对 VEN 组合有骨髓或 EM 反应；在第一个 VEN 联合治疗周期期间接受同步放疗（25Gy，10fx）的 1 名患者（9.1%，部分缓解）中观察到 EM 反应，其他 3 名患者显示骨髓反应，但没有 EM 反应。中位随访 27.0 个月后，中位总生存期估计为 5.4 个月。总而言之，VEN 联合方案对 AML 的 EM 复发仅显示有限的疗效，对引发 EM 反应几乎没有影响。