Department of Hematology, Dalian Key Laboratory of hematology, Liaoning Medical Center for Hematopoietic Stem Cell Transplantation, the Second Hospital of Dalian Medical University, Dalian, 116027, China.
Blood Stem Cell Transplantation Institute, Liaoning Key Laboratory of Hematopoietic Stem Cell Transplantation and Translational Medicine, Dalian Medical University, Dalian, 116044, China.
BMC Cancer. 2023 Aug 17;23(1):764. doi: 10.1186/s12885-023-11259-6.
Currently, there is no standard treatment for managing relapse in patients with acute myeloid leukemia and myelodysplastic syndrome (AML/MDS) after allogeneic hematopoietic cell transplantation. Venetoclax-based therapies have been increasingly used for treating post-transplantation relapse of AML. The aim of this systematic review and meta-analysis was to evaluate the efficacy and adverse events of Venetoclax combined with hypomethylating agents (HMAs) for AML/MDS relapse post-transplantation.
We searched PubMed, Web of Science, Excerpta Medica Database, Cochrane Library, and Clinical. gov for eligible studies from the inception to February 2022. The Methodological Index for Non-Randomized Studies was used to evaluate the quality of the included literatures. The inverse variance method calculated the pooled proportion and 95% confidence interval (CI).
This meta-analysis included 10 studies involving a total of 243 patients. The pooled complete response and complete response with incomplete blood count recovery rate of Venetoclax combined with HMAs for post-transplantation relapse in AML/MDS was 32% (95% CI, 26-39%, I = 0%), with an overall response rate of 48% (95% CI, 39-56%, I = 37%). The 6-month survival rate was 42% (95% CI, 29-55%, I = 62%) and the 1-year survival rate was 23% (95% CI, 11-38%, I = 78%).
This study demonstrated a moderate benefit of Venetoclax in combination with HMAs for patients with relapsed AML/MDS post-transplantation (including those who have received prior HMAs therapy), and may become one of treatment options in the future. Large-scale prospective studies are needed to confirm the potential benefit from venetoclax combined with HMAs.
目前，对于异基因造血细胞移植后急性髓系白血病和骨髓增生异常综合征（AML/MDS）患者的复发，尚无标准的治疗方法。基于 Venetoclax 的治疗方法已越来越多地用于治疗移植后 AML 的复发。本系统评价和荟萃分析旨在评估 Venetoclax 联合低甲基化剂（HMAs）治疗 AML/MDS 移植后复发的疗效和不良事件。
我们从建库到 2022 年 2 月，在 PubMed、Web of Science、Excerpta Medica Database、Cochrane Library 和 Clinical. gov 中检索了符合条件的研究。采用非随机研究方法学指数评估纳入文献的质量。采用逆方差法计算合并后的比例及其 95%置信区间（CI）。
本荟萃分析纳入了 10 项研究，共 243 例患者。Venetoclax 联合 HMAs 治疗 AML/MDS 移植后复发的完全缓解率和不完全血细胞计数恢复的完全缓解率为 32%（95%CI，26-39%，I=0%），总缓解率为 48%（95%CI，39-56%，I=37%）。6 个月生存率为 42%（95%CI，29-55%，I=62%），1 年生存率为 23%（95%CI，11-38%，I=78%）。
本研究表明，Venetoclax 联合 HMAs 对移植后复发的 AML/MDS 患者（包括既往接受 HMAs 治疗的患者）具有中等获益，可能成为未来的治疗选择之一。需要进行大规模的前瞻性研究来证实 Venetoclax 联合 HMAs 的潜在获益。
