Study of Molecular Markers to Predict Tyrosine Kinase Inhibitor Response in Children and Adolescents with Chronic Myeloid Leukemia

What this Trial Is About

Researchers are investigating chronic myeloid leukemia CML in children, a rare condition that differs in disease features and treatment response compared to adults. The study focuses on understanding molecular factors that might predict how children respond to tyrosine kinase inhibitors TKIs, a revolutionary treatment for CML. Since resistance to TKIs can occur and is not well understood in children, this research aims to identify genetic signatures linked to treatment outcomes. The study uses targeted next generation sequencing of DNA and RNA from blood samples taken at diagnosis. It retrospectively analyzes mutations in 64 genes and genetic expression profiles to find associations with response to TKI treatment. Children diagnosed with chronic phase CML and treated with TKIs are studied, including a subgroup with detailed RNA analysis. The goal is to compare molecular profiles of those who do and do not achieve complete cytogenetic response CCR at 12 months. Participants provide blood samples at diagnosis for genetic testing. Researchers track treatment response at 3, 12, 18, and 24 months using molecular and cytogenetic measures, as well as monitoring disease progression and survival. The main outcome is CCR at 12 months after starting TKI treatment. This observational study provides insights into genetic markers that might predict TKI response in pediatric CML, with total participation spanning at least two years of follow-up.

CML Pediatric ITK Response According to Molecular Identification at Diagnosis