Introduction :

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment for relapsed or refractory acute myeloid leukemia (R/R-AML) patients. However, it is not clear about appropriate salvage therapy bridged to allo-HSCT for R/R-AML patients. In this study, we compared the outcomes of R/R-AML patients treated with venetoclax plus hypomethylating agents (VEN-HMA) regimen or venetoclax plus azacitidine and homoharringtonine (VAH) regimen as salvage therapies bridged to allo-HSCT.

Methods :

A total of 105 R/R-AML patients treated with VEN-HMA regimen or VAH regimen bridged to allo-HSCT between September 2018 and February 2023 were enrolled. The outcomes including survival, relapse and NRM after allo-HSCT were analyzed in two groups.

Results :

Forty-four patients were treated with VEN-HMA regimen, and 61 with VAH regimen. Seventy-four patients had primary refractory AML and 31 relapsed AML, including 19 relapsed after chemotherapy and 12 relapsed after allo-HSCT. Thirty-one (70.5%) patients achieved composite complete remission (CRc) in VEN-HMA group including 20 (45.5%) with minimal residual disease (MRD) negative, as well as 52 (85.2%) with CRc including 40 (65.6%) with MRD negative in VAH group (P=0.09). Twenty-five (56.8%) patients were stratified with adverse risk in VEN-HMA group and 45 (73.8%) in VAH group according to the 2022 European Leukemia Net (ELN) classification (P=0.07). The 2-year overall survival (OS) and event-free survival (EFS) in VAH group were higher (78.4% vs 58.7%, P=0.03; 68.3% vs 38.7%, P=0.003), as well as 2-year cumulative incidence of relapse (CIR) was lower than VEN-HMA group (23.2% vs 42.4%, P=0.04). Among patients with CRc, the 2-year OS, EFS and CIR was 67.5%, 48.9% and 37.0% in VEN-HMA group, as well as 82.5%, 72.5% and 23.6% in VAH group (P=0.15, 0.05 and 0.24). Among patients with MRD negative, the 2-year OS, EFS and CIR was 75.5%, 55.5% and 31.6% in VEN-HMA group, as well as 90.8%, 80.0% and 17.5% in VAH group (P=0.15, 0.10 and 0.28). Among patients with adverse risk, the 2-year OS and EFS in VAH group were higher (79.3% vs 47.7%, P=0.01; 70.9% vs 24.1%, P=0.0002), as well as 2-year CIR was lower than VEN-HMA group (17.4% vs 45.8%, P=0.01). Non-relapse mortality (NRM) was similar between VEN-HMA group and VAH group (18.9% vs 8.5%, P=0.12).

Conclusion :

VAH regimen is an effective salvage therapy bridged to allo-HSCT for R/R-AML patients compared with VEN-HMA regimen.

Keywords :

Venetoclax, Azacitidine, Homoharringtonine, Relapsed/Refractory AML, Allogeneic Hematopoietic Cell Transplantation.

No relevant conflicts of interest to declare.

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