The multi-kinase inhibitor sorafenib was initially evaluated in the COG Phase 1 trial ADVL0413 in children with advanced solid tumors and leukemias; two children with relapsed/refractory FLT3-ITD AML achieved ≥CR2 with sorafenib monotherapy on this trial and subsequently underwent HSCT (74). This evidence concerns the gene FLT3 and leukemia.