Efficacy and safety of venetoclax combined with hypomethylating agents for relapse of acute myeloid leukemia and myelodysplastic syndrome post-transplantation: a systematic review and meta-analysis

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Background Currently, there is no standard treatment for managing relapse after allogeneic hematopoietic cell transplantation in patients with acute myeloid leukemia and myelodysplastic syndrome (AML/MDS). Venetoclax (Ven)-based therapies have been increasingly used for treating post-transplant relapse. The aim of this systematic review and meta-analysis was to evaluate the efficacy and adverse events of Ven combined with hypomethylating agents (HMAs) for AML/MDS relapse post-transplantation. Methods 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 literature. The inverse variance method calculated the pooled proportion and 95% confidence interval (CI). Results This meta-analysis included 9 studies involving a total of 202 patients. The pooled complete response and complete response with incomplete blood count recovery rate of Ven combined with HMAs for post-transplant relapse in AML/MDS was 32% (95% CI, 25%-40%, I 2 = 0%), with an overall response rate of 50% (95% CI, 40%-59%, I 2 = 38%). The 6-month survival rate was 43% (95% CI, 28%-59%, I 2 = 68%) and the 1-year survival rate was 27% (95% CI, 13%-42%, I 2 = 72%). Conclusion This study demonstrated a moderate benefit of Ven in combination with HMAs for AML/MDS patients with relapsed post-transplantation (including those who have received prior HMAs therapy), and may become one of the treatment options in the future. Large-scale prospective studies are needed to confirm this study in future.