Journal of Clinical OncologyVenetoclax Plus Gilteritinib for FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia

Gilteritinib is standard therapy for relapsed/refractory FLT3-mutated (FLT3mut) AML but seldom reduces FLT3mut burden or induces sustained efficacy. This phase Ib open-label, dose-escalation/dose-expansion study enrolled 61 patients (63% having received prior FLT3 therapy) to receive 400 mg oral venetoclax once daily and 80 mg or 120 mg oral gilteritinib once daily. The combination of venetoclax and gilteritinib was associated with high mCRc and FLT3 molecular response rates regardless of prior FLT3 inhibitor exposure, but did require dose interruptions for myelosuppression.