Evaluation of the prognostic value of the leukemic stem cell (LSC) and pre-leukemic hematopoietic stem cell (pre-L HSC) for minimal residual disease monitoring and clinical management of pediatric acute myeloid leukemia patients

01 January 2015 → 31 December 2018
Funding by bilateral agreement (private and foundations)
Research disciplines
  • Medical and health sciences
    • Pediatrics and neonatology
    • Pediatrics and neonatology
    • Nursing
    • Pediatrics and neonatology
leukemic stem cell
Project description

Pediatric Acute Myeloid Leukemia (AML) patients exhibit a high risk of relapse (30-40%), resulting in a 5 year overall survival rate of 65-70%. Flow Cytometry (FCM)-based Minimal Residual Disease (MRD) monitoring holds strong prognostic power, and can assist in risk group stratification and treatment modification, and can assist in risk group stratification and treatment modification. However, relapse is generally believed to occur through the presence and outgrowth of a therapy resistant cell fraction denominated Leukemic Stem Cells (LSCs) or the persistence of pre-leukemic hematopoietic stem cells (pre-L HSC). It thus would be of great interest to have a toolbox for careful monitoring of these cell fractions and identify specific markers for development of highly sensitive MRD assays and targeted drugs.
The two major goals:
(1) Evaluate current and novel FCM markers for LSC and pre-L HSC characterization and determine their prognostic value.
(2) Identify molecular characteristics and antigens of LSCs and pre-L HSCs, useful in targeted therapy and MRD detection