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Updated: Sep 22, 2025
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Pediatric acute myeloid leukemia patients with KMT2A rearrangements: a single-center retrospective study
Wei Yang1, Maoquan Qin1, Chenguang Jia1
1Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, People's Republic of China.
Insights
Pediatric acute myeloid leukemia with KMT2A rearrangements shows poor outcomes. Hematopoietic stem cell transplantation outcomes were similar between matched and haploidentical donors, but matched donors had less severe graft-versus-host disease.
Area of Science:
- Pediatric Hematology Oncology
- Cancer Genetics
- Stem Cell Transplantation
Background:
- Pediatric acute myeloid leukemia (AML) with KMT2A rearrangements presents a challenging prognosis.
- Outcomes remain poor despite advancements like hematopoietic stem cell transplantation (HSCT).
Purpose of the Study:
- To retrospectively analyze the prognosis and efficacy of treatments for pediatric AML with KMT2A rearrangements.
- To compare outcomes between different types of HSCT and chemotherapy.
Main Methods:
- Retrospective analysis of 32 children with KMT2A-rearranged AML treated between January 2015 and February 2021.
- Comparison of overall survival (OS), event-free survival (EFS), and cumulative incidence of relapse (CIR) between haploidentical HSCT and matched HSCT.
- Assessment of acute graft-versus-host disease (aGVHD) severity.
Main Results:
- Patients in the medium-risk group achieved 100% OS and EFS with chemotherapy alone.
- No significant differences in OS, EFS, or CIR were observed between haploidentical HSCT and matched HSCT.
- Matched HSCT demonstrated significantly lower rates of severe acute graft-versus-host disease (aGVHD) compared to haploidentical HSCT.
Conclusions:
- For pediatric AML with KMT2A rearrangements, chemotherapy alone can achieve excellent outcomes in the medium-risk group.
- Both matched and haploidentical HSCT offer comparable survival benefits, but matched HSCT is associated with less severe aGVHD.
- HLA-matched sibling or unrelated donors are preferred, with haploidentical donors as a secondary option.
Purpose:
Pediatric acute myeloid leukemia (AML) with KMT2A rearrangements has a very different prognosis. Poor outcomes cannot be avoided even after hematopoietic stem cell transplantation. In order to investigate the prognosis and efficacy, we conducted a retrospective analysis.
Patients And Methods:
We retrospectively analyzed a total of 32 children with KMT2A rearrangements AML treated in our hospital between January 2015 and February 2021.
Results:
The proportion of patients with KMT2A-rearranged in the medium-risk group of overall survival (OS) and event-free survival (EFS) was 100%. No differences in OS, EFS and cumulative incidence of relapse (CIR) were detected between the haploidentical hematopoietic stem cell transplantation (haplo-HSCT) and full matched HSCT (P = 0.289, P = 0.303, P = 0.303). Acute graft-versus-host disease (aGVHD) was often detected in the haplo-HSCT cohort, while full matched HSCT had no obvious aGVHD, assessed as≤1 grade (P < 0.05). Patients in the medium-risk pediatric group could acquire 100% OS and EFS only after chemotherapy. There was no significant difference in OS, EFS and CIR between full matched HSCT and haploidentical transplantation in pediatric AML with KMT2A rearrangements, but full matched HSCT seemed to have a lower death rate. The severity of aGVHD in the full matched HSCT was less than that in the haploidentical transplantation group.
Conclusion:
The primary choice of donor can be HLA-matched sibling donors or matched unrelated donors for children with AML with KMT2A rearrangements, and the secondary choice can be haploid donors.
