Venetoclax plus azacitidine with or without homoharringtonine followed by allogeneic haematopoietic cell transplantation in patients with relapsed/refractory acute myeloid leukaemia: A multicentre cohort study

The appropriate salvage regimen followed by allogeneic haematopoietic stem cell transplantation (allo-HSCT) for relapsed/refractory (R/R) acute myeloid leukaemia (AML) patients remains unclear. Three hundred and fifty R/R AML patients receiving venetoclax and azacitidine (VA) regimen or VA plus homoharringtonine (VAH) regimen as salvage therapy were enrolled in this study, with a higher composite complete remission rate in the VAH group (69.9%) than in the VA group (46.1%). A total of 105 patients underwent allo-HSCT, with a median follow-up post-transplantation of 37.2 months. The 3-year cumulative incidence of transplant-related mortality was 18.2% in the VA group and 9.8% in the VAH group. The 3-year cumulative incidence of relapse was lower in the VAH group (19.7%) than in the VA group (43.2%). The 3-year overall survival and event-free survival (EFS) were 82.0% and 70.5%, respectively, in the VAH group, which were higher than 59.1% and 38.6%, respectively, in the VA group. Multivariate analysis revealed the VAH regimen and MRD-negative at transplantation were protective factors for relapse (HR = 0.427 and HR = 0.368) and EFS (HR = 0.469 and HR = 0.384). In conclusion, the VAH regimen is an effective and safe salvage therapy bridge to allo-HSCT for R/R AML patients compared with the VA regimen.