Related Experiment Video
Updated: Dec 20, 2025
Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies
Published on: September 6, 2017
Impact of detectable measurable residual disease on umbilical cord blood transplantation
Frédéric Baron1, Myriam Labopin2,3,4,5, Annalisa Ruggeri6,7
1Laboratory of Hematology, GIGA-I3, University of Liege and CHU of Liège, Liege, Belgium.
Abstract:
The impact of measurable residual disease (MRD) on cord blood transplantation (CBT) outcomes has remained debated. To address this issue, we assessed the impact of measurable MRD at CBT on outcomes in large cohort of patients with acute leukemia. Inclusion criteria included adult patients with acute myeloid (AML) or acute lymphoblastic leukemia (ALL), CBT as first allo-HCT in first or second complete remission (CR) at transplantation, and known MRD status at the time of CBT. Data from 506 patients were included in the analysis. Among them, 317 patients had AML and 189 had ALL. Positive MRD was reported in 169 (33%) patients while the remaining 337 patients were MRD negative at CBT. At 2 years, relapse incidence was 18% in patients with MRD negativity vs 33% in those with MRD positivity at transplantation (P < .001). Two-year leukemia-free survival (LFS) and overall survival (OS) were 57% and 60%, respectively, in MRD negative patients, vs 38% (P < .001) and 48% (P = .004), respectively, in those with MRD positivity. There was no interaction between the impact of MRD on OS and LFS and diagnosis (ie, ALL vs AML), single or double CBT, and reduced-intensity or myeloablative conditioning. On multivariate analysis, MRD positivity was associated with a higher risk of relapse (HR = 1.8, P = .003), comparable non-relapse mortality (P = .44), worse LFS (HR = 1.4, P = .008) and a trend towards worse OS (HR = 1.3, P = .065). In conclusion, these data suggest that novel strategies that are aiming to achieve MRD negativity at CBT are needed for leukemic patients with positive MRD pre-CBT.
Insights
Measurable residual disease (MRD) impacts cord blood transplantation (CBT) outcomes in acute leukemia patients. Achieving MRD negativity before CBT is crucial for improving leukemia-free survival and overall survival, indicating a need for new treatment strategies.
Area of Science:
- Hematology
- Oncology
- Transplantation Immunology
Background:
- Measurable residual disease (MRD) status is a critical factor in hematopoietic stem cell transplantation outcomes.
- The impact of MRD on outcomes following cord blood transplantation (CBT) in acute leukemia remains debated.
- Assessing MRD's influence on CBT outcomes is essential for refining treatment strategies.
Purpose of the Study:
- To evaluate the impact of measurable residual disease (MRD) at the time of cord blood transplantation (CBT) on outcomes in adult patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL).
- To determine the association between pre-CBT MRD status and key clinical endpoints, including relapse incidence, leukemia-free survival (LFS), and overall survival (OS).
Main Methods:
- Retrospective analysis of 506 adult patients with AML or ALL undergoing CBT as first allo-HCT in complete remission (CR).
- Patients were categorized based on MRD status (positive or negative) at the time of CBT.
- Outcomes including relapse incidence, LFS, and OS were compared between MRD-positive and MRD-negative groups using univariate and multivariate analyses.
Main Results:
- Patients with MRD positivity at CBT had a significantly higher relapse incidence (33% vs 18%) and worse 2-year LFS (38% vs 57%) and OS (48% vs 60%) compared to MRD-negative patients.
- MRD positivity was independently associated with a higher risk of relapse (HR=1.8) and worse LFS (HR=1.4) on multivariate analysis.
- No significant interaction was observed between MRD status and leukemia type (AML vs ALL), CBT strategy, or conditioning regimen intensity.
Conclusions:
- Measurable residual disease positivity at the time of cord blood transplantation is a significant predictor of adverse outcomes in acute leukemia.
- These findings underscore the critical need for novel strategies to achieve MRD negativity prior to CBT in patients with leukemia.
- Optimizing MRD clearance before CBT is essential for improving survival rates in this patient population.
Related Concept Videos
01:22Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
01:24Tissue Transplantation
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...