Prognostic value of pre-HSCT MRD in pediatric and AYA patients with de novo AML

A retrospective, multicenter study evaluated the prognostic value of measurable residual disease (MRD) by multiparametric flow cytometry (MFC) before hematopoietic stem cell transplantation (HSCT) in 218 pediatric and adolescent and young adult (AYA) patients (aged 0–23 years at diagnosis) with de novo acute myeloid leukemia (AML). Outcomes assessed were 8-year overall survival (OS), event-free survival (EFS), cumulative incidence of relapse (CIR), and non-relapse mortality (NRM). Results were published in Haematologica by Buldini et al.

Key data: At a median follow-up of 57 months, pre-HSCT MRD negativity was associated with higher 8-year OS (77.8% vs 54.0%; p < 0.0001) and EFS (74.4% vs 42.9%; p < 0.0001), and lower 8-year CIR (18.5% vs 49.1%; p < 0.0001) vs MRD positivity. In multivariable analysis, MRD positivity was independently associated with poorer OS (hazard ratio [HR], 3.77; p < 0.001), EFS (HR, 3.39; p < 0.001), and CIR (HR, 3.66; p < 0.001) vs MRD negativity.

Key learning: In this study, pre-HSCT MFC-MRD negativity was associated with improved relapse and survival outcomes vs MRD positivity, supporting its role as a prognostic marker in pediatric and AYA patients with de novo AML.