The purpose of this study is to evaluate the efficacy and safety of avapritinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.
Gender unlimited;
Under 18 years;
Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).
Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;
Have received prior treatment with avapritinib;
Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;
Presence of active uncontrolled infection (including bacterial, fungal, or viral infection);
Present of significant underlying organ diseases: such as myocardial infarction, chronic heart failure, decompensated liver or kidney dysfunction；
Children's Hospital of Soochow University
Lead sponsor