AAML1031: sorafenib combined with chemotherapy for children with high allelic ratio FLT3-ITD acute myeloid leukaemia
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.
Overview
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.
- Three-year event-free survival 55.9 percent vs 31.9 percent.
- Three-year relapse risk 22.8 percent vs 55.5 percent.
FLT3 inhibitors are now incorporated into frontline paediatric AML therapy for FLT3-ITD, with gilteritinib being studied in the successor trial.
- Non-randomised comparison; sorafenib patients more often had haematopoietic stem cell transplant.