Treatment sequencing with gilteritinib and allo-HSCT in R/R FLT3m AML: A real-world study

Results from a multicenter, retrospective, real-world study evaluating treatment sequences with gilteritinib and allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with relapsed/refractory (R/R) FLT3-internal tandem duplication (ITD) and/or FLT3-tyrosine kinase domain (TKD)-mutated acute myeloid leukemia (AML) were published in the British Journal of Haematology by Kunadt et al. Gilteritinib was administered in three treatment sequences: gilteritinib monotherapy (n = 116), gilteritinib followed by allo-HSCT (n = 40), and gilteritinib followed by allo-HSCT and subsequent gilteritinib maintenance (n = 17).

Key data: The overall complete remission/incomplete remission (CR/CRi) rate was 45.3%, and, at a median follow-up of 13.1 months, median overall survival (OS) was 10.0 months. OS at 1-year was 34% (95% confidence interval [CI], 25–47) with gilteritinib monotherapy, compared with 76% (95% CI, 60–97) with allo-HSCT and 92% (95% CI, 79–100) from the start of maintenance therapy (p < 0.001). Frequent adverse events (AEs) associated with gilteritinib were hepatic toxicity (6.4%) and cardiac events (4.4%).

Key learning: This real-world study confirmed the efficacy and safety of gilteritinib monotherapy in patients with R/R FLT3-ITD and/or FLT3-TKD-mutated AML, particularly when used as a bridge to allo-HSCT followed by gilteritinib maintenance.