Sorafenib therapy is associated with improved outcomes for FLT3-ITD AML relapsing after allo-HSCT

Abstract The optimal therapy for acute myeloid leukemia (AML) with FMS-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) who relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains unclear. In this study, we retrospectively evaluated the efficacy of sorafenib combined with other therapeutic strategies as salvage therapy for these patients. A total of 83 AML with FLT3-ITD relapsing after allo-HSCT were enrolled in this study. Fifty-three patients received salvage therapy containing sorafenib, and 30 patients did not. Salvage therapy containing sorafenib was superior to that without sorafenib with respect to complete remission rates, overall survival (OS) and progression-free survival (PFS) (66.0% vs 30.0%, 46.8% vs 20.0%, 44.9% vs 16.7%; P =0.002, P =0.003, P =0.001). Further subgroup analysis revealed that the OS and PFS of patients who received sorafenib combined with chemotherapy followed by donor lymphocyte infusion (DLI) were superior to those receiving other therapeutic regimens, including sorafenib combined with chemotherapy, chemotherapy followed by DLI, and monochemotherapy ( P =0.003, P P =0.035, hazard ratio (HR)=0.526); salvage therapy including sorafenib and DLI were the protective factors for longer PFS ( P =0.011, HR=0.423; P =0.019, HR=0.508). Our data suggest that sorafenib therapy is associated with improved outcomes for FLT3-ITD AML relapsing after allo-HSCT, and whether sorafenib combined with chemotherapy followed by DLI reveals an optimal efficacy merits further study.