Clinical profiling of AML1::ETO and KIT exon 17 mutation in pediatric AML by high-throughput drug sensitivity.

ABSTRACT:

Background

The favorable prognosis of pediatric AML1::ETO acute myeloid leukemia (AML) is well-established, yet the impact of co-occurring KIT mutations-particularly in exon 17-on clinical outcomes and chemosensitivity remains incompletely defined.

Methods

We analyzed the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) database comprising 957 pediatric AML patients to assess the clinical characteristics and prognosis of pediatric patients harboring AML1::ETO and KIT mutation using Kaplan-Meier survival analysis and Cox proportional hazards models. Subsequently, we performed experimental validation using bone marrow samples from 16 pediatric AML patients at Shenzhen Children's Hospital by high-throughput drug sensitivity (HDS) screening against t

SUBMITTER: Hu Z 

PROVIDER: S-EPMC13628913 | biostudies-literature | 2026 Jun

REPOSITORIES: biostudies-literature