Sanz-Solas Antonio, Saiz-Rodríguez Miriam, Simal Sara Calvo, Rodríguez-Veiga Rebeca, Solana-Altabella Antonio, Montesinos Pau, Labrador Jorge
Research Unit, Fundación Burgos por la Investigación de la Salud (FBIS), Hospital Universitario de Burgos, Burgos, 09006, Spain.
Universidad Autónoma de Madrid | UAM, Madrid, Spain.
Ann Hematol. 2025 Aug 30. doi: 10.1007/s00277-025-06543-3.
This meta-analysis, comprising 24 studies, evaluated the efficacy of venetoclax (VEN) in combination with hypomethylating agents (HMAs), including azacitidine (AZA) and decitabine (DEC), in untreated patients with acute myeloid leukemia (AML), comparing outcomes from clinical trials and real-world practice. No significant difference in composite complete response (CRc) rates was observed between clinical trials (52%, 95% CI: 39-65%) and real-world studies (67%, 95% CI: 47-87%). However, overall survival (OS) was significantly longer in clinical trials (13.98 months, 95% CI: 11.89-16.07) compared to real-world cohorts (9.35 months, 95% CI: 8.46-10.23; p < 0.005). In real-world studies, the VEN + HMA combination was associated with a significantly higher CRc rate (67%, 95% CI: 48-85%) compared to HMA monotherapy (17%, 95% CI: 13-21%; p < 0.005), although no significant difference in OS was observed between these groups (9.35 vs. 9.38 months; p = 0.964). These findings highlight the need to optimize the implementation of VEN + HMA regimens in clinical practice, as real-world outcomes remain inferior to those reported in clinical trials.
这项荟萃分析纳入了24项研究，评估了维奈克拉（VEN）联合包括阿扎胞苷（AZA）和地西他滨（DEC）在内的低甲基化药物（HMA），用于治疗初治急性髓系白血病（AML）患者的疗效，并比较了临床试验和真实世界实践中的结果。临床试验（52%，95%置信区间：39 - 65%）和真实世界研究（67%，95%置信区间：47 - 87%）的复合完全缓解（CRc）率无显著差异。然而，与真实世界队列（9.35个月，95%置信区间：8.46 - 10.23；p < 0.005）相比，临床试验中的总生存期（OS）显著更长（13.98个月，95%置信区间：11.89 - 16.07）。在真实世界研究中，与HMA单药治疗（17%，95%置信区间：13 - 21%；p < 0.005）相比，VEN + HMA联合治疗的CRc率显著更高（67%，95%置信区间：48 - 85%），尽管这些组之间的OS无显著差异（9.35对9.38个月；p = 0.964）。这些发现凸显了在临床实践中优化VEN + HMA方案实施的必要性，因为真实世界的结果仍低于临床试验报告的结果。
