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Adding the kinase inhibitor sorafenib to chemotherapy improved event-free survival in children with high allelic ratio FLT3-ITD acute myeloid leukaemia compared with matched historical controls, the first FLT3-targeted result in paediatric leukaemia.
Non-randomised cohort within the AAML1031 trial of 92 children with high allelic ratio FLT3-ITD AML treated with sorafenib during induction, consolidation and maintenance, compared with 76 concurrent and historical controls treated without sorafenib.
Three-year event-free survival was 55.9 versus 31.9 percent and relapse risk lower with sorafenib; benefit was concentrated in patients who received it from the first induction course, and toxicity was mostly rash and hand-foot syndrome.
FLT3 inhibitors are now incorporated into frontline paediatric AML therapy for FLT3-ITD, with gilteritinib being studied in the successor trial.
Shares Acute myeloid leukaemia in children, Journal of Clinical Oncology.