Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation

Prior treatment

• Have received prior treatment with avapritinib;
• Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;

Other illness

• 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；
• With other malignancies requiring treatment；

Other

• Already enrolled in another interventional clinical study；
• The researchers determined that the individual is not suitable to participate in this trial.

• 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;
• KIT mutation;
• Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;
• Relapsed AML: patients who achieved remission after consolidation therapy or transplantation, FISH confirmed that the fusion gene turned positive, or extramedullary leukemia infiltration;
• No active infections;
• Liver function: Tbil ≤2×ULN, ALT/AST ≤3×ULN, creatinine clearance ≥50ml/min;
• ECOG score \<2;
• Expected survival time \>12 weeks;
• Participants must have the ability to understand and be willing to participate in this study and must sign an informed consent form.