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Research Article: Pre-transplant measurable residual disease by flow cytometry is an independent prognostic factor in pediatric acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation

Date Published: 2026-07-17

Abstract:
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a critical treatment for pediatric acute myeloid leukemia (AML); however, relapse after transplantation remains a major challenge. This study aimed to evaluate the prognostic value of pre-transplant measurable residual disease (MRD) detected by multiparameter flow cytometry (MFC) and high-risk (HR) fusion genes on survival after transplantation. This single-center retrospective study included 80 newly diagnosed pediatric AML patients who underwent allo-HSCT during their first complete remission between October 2019 and October 2025. All patients were treated according to the C-HUANAN-AML 15 protocol prior to transplantation, with risk stratification and treatment decisions based on European LeukemiaNet criteria and serial MFC-MRD assessments. Cox regression models were employed for statistical analysis. With a median follow-up of 34.5 months, the 3-year disease-free survival (DFS) and overall survival (OS) rates were 85.5% ± 4.2% and 86.8% ± 4.1%, respectively. Univariate analysis identified several risk factors for inferior survival, but multivariate analysis confirmed pre-transplant MFC-MRD positivity as an independent adverse prognostic factor for both 3-year DFS and OS (DFS: HR = 14.304, 95%CI: 1.892–108.155, P = 0.010; OS: HR = 15.847, 95%CI: 2.036–123.328, P = 0.008). Among relapsed patients, the majority harbored HR fusion genes. Given the limited number of cases with specific HR fusion genes (e.g., NUP98 rearrangements, n = 4; FUS :: ERG , n = 2), these subgroup findings are exploratory and require validation in larger cohorts. Pre-transplant MFC-MRD positivity is an independent adverse prognostic marker for survival in pediatric AML patients following allo-HSCT. Patients harboring HR fusion genes such as NUP98 rearrangements or FUS :: ERG fusions may remain at risk of relapse even after successful transplantation, although this observation is based on a limited number of cases. These findings emphasize the importance of achieving deep remission to reduce tumor burden before transplantation and provide a rationale for intensified post-transplant management strategies, including maintenance therapy, for this HR population.

Introduction:
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a critical treatment for pediatric acute myeloid leukemia (AML); however, relapse after transplantation remains a major challenge. This study aimed to evaluate the prognostic value of pre-transplant measurable residual disease (MRD) detected by multiparameter flow cytometry (MFC) and high-risk (HR) fusion genes on survival after transplantation.

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