Translational Research - Observational Study for Identification of New Possible Prognostic Factors and Future Therapeutic Targets in Children With Acute Lymphoblastic Leukemia (ALL)
- Collect clinical data in parallel with biological data and samples from children with
newly diagnosed acute lymphoblastic leukemia for biobanking, and use part of the
biobanked material to perform specific translational projects to achieve objectives
- To identify new prognostic factors (e.g., minimal-residual disease [MRD] significance
in small subgroups, miRNAs expression profile, PAX5 mutation, genetic abnormalities in
T-cell acute lymphoblastic leukemia [T-ALL], and RAS pathway activation) and future
therapeutic targets in children with newly diagnosed acute lymphoblastic leukemia.
- To identify leukemia cell genetic alterations (e.g., mutations in T-ALL and miRNA
expression in B-cell acute lymphoblastic leukemia [B-ALL]) and related molecular
pathways (e.g., RAS pathway) underlying leukemogenesis.
- To identify patient pharmacogenetic polymorphisms impacting individual response to
corticosteroids as part of standard therapy and investigate their prognostic
OUTLINE: This is a prospective observational biobanking study.
Patients undergo clinical evaluation, laboratory tests, and imaging periodically. Data are
collected before, during, and after first-line standard therapy. Clinical data are collected
from all patients in parallel with the biological data and samples. Biological samples are
partly used to perform specific translational research (TR) projects. Remaining biological
materials are stored for future research.
The following TR projects are performed on the biological samples for this study. Biological
samples are analyzed for allele-specific amplification of Ig/TCR clonal rearrangements to
quantify minimal-residual disease (MRD) via real-time PCR (TR1 Project); miRNA expression
via qPCR (TR 2 Project); the detection of main point mutations via high-resolution melting
PCR (TR 3 Project); genetic polymorphisms via real-time TaqMan allelic-discrimination method
(TR 4 Project); clinical significance of genetic abnormalities via quantitative real-time
RT-PCR, direct sequencing, and fluorescence in situ hybridization (TR 5 Project); and RAS
pathway activation via single-nucleotide polymorphism (SNP) analysis and gene-expression
analysis (TR 6 Project).
CHU - Hopital Robert Debre