Study of Molecular Subtyping and MRD-Guided Induction Therapy in Children and Adolescents With Acute Myeloid Leukemia

What this Trial Is About

Researchers are studying children and adolescents with acute myeloid leukemia AML to estimate the chance of achieving complete remission, including remission with incomplete blood count recovery, and to evaluate event-free survival and relapse rates. The study aims to see if combining molecular subtyping with measurable residual disease MRD-guided remission induction improves outcomes compared to the previous AML2018 treatment protocol. This is a Phase II multicenter clinical trial focusing on personalized treatment approaches based on genetic markers and MRD status. Treatment involves two main induction regimens assigned based on specific genetic fusion genes or mutations. Patients with certain fusion genes or KIT mutation receive a standard dose chemotherapy SDC regimen including homoharringtonine, cytarabine, and etoposide. Those without these genetic markers receive a low-dose chemotherapy LDC regimen combined with venetoclax. Targeted drugs such as venetoclax, avapritinib, gilteritinib, or sorafenib may be added based on individual molecular profiles. Post-induction, patients undergo 3 to 4 cycles of standard-dose chemotherapy, with high-risk patients considered for bone marrow transplantation. Participants will be closely monitored through scheduled assessments including blood counts and MRD evaluations at specific time points Day 26 or 32 after inductions. Researchers will track remission status, event-free survival up to 72 months, and relapse incidence. Safety and treatment responses are evaluated continuously, with informed consent obtained from patients or guardians. The study involves detailed follow-up to assess treatment effects and long-term outcomes in this pediatric AML population.

Clinical Study of Induction Therapy Options Based on Molecular Subtyping and MRD in Children and Adolescents With AML