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[Clinical features and prognosis of pediatric acute megakaryocytic leukemia]
Tie-Mei Luo1, Jie Yu1, Xi-Zhou An1
1Department of Hematology and Oncology, Children's Hospital of Chongqing Medical University/National Clinical Research Center for Child Health and Disorders/Ministry of Education Key Laboratory of Child Development and Disorders/Chongqing Key Laboratory of Pediatrics, Chongqing 401122, China.
Insights
Pediatric acute megakaryocytic leukemia (AMKL) shows a poor prognosis. Key factors influencing outcomes include CD56 expression, early treatment response, and minimal residual disease (MRD) levels.
Area of Science:
- Pediatric Hematology Oncology
- Leukemia Research
- Clinical Trial Analysis
Background:
- Acute megakaryocytic leukemia (AMKL) is a challenging subtype of childhood leukemia.
- Understanding clinical features and treatment efficacy is crucial for improving outcomes in pediatric AMKL.
Purpose of the Study:
- To investigate the clinical characteristics and prognosis of childhood AMKL.
- To evaluate the effectiveness of the acute myeloid leukemia 03 (AML03) regimen in treating pediatric AMKL.
Main Methods:
- Retrospective analysis of clinical data from 47 children diagnosed with AMKL between May 2011 and December 2019.
- Survival analysis using the Kaplan-Meier method and log-rank test to identify prognostic factors.
- Evaluation of treatment outcomes, including remission rates and minimal residual disease (MRD) status.
Main Results:
- The AML03 regimen was used in 22 non-Down syndrome-AMKL patients, achieving an 85% bone marrow remission rate and 79% MRD-negative rate post-induction II.
- Two-year overall survival (OS) and event-free survival (EFS) rates were 50%±13% and 40%±12%, respectively.
- Positive CD56 expression, lack of early remission, and positive MRD were associated with significantly poorer OS and EFS (P < 0.05).
Conclusions:
- Pediatric AMKL is characterized by a low remission rate and poor prognosis.
- CD56 expression, early treatment response, and MRD are significant prognostic indicators.
- Allogeneic hematopoietic stem cell transplantation did not demonstrate a significant impact on AMKL prognosis in this cohort.
Objective:
To study the clinical features and prognosis of children with acute megakaryocytic leukemia (AMKL) and the clinical effect of acute myeloid leukemia 03 (AML03) regimen for the treatment of pediatric AMKL.
Methods:
The clinical data were collected from 47 children with AMKL who were diagnosed from May 2011 to December 2019. The treatment outcomes and prognostic factors were analyzed. The Kaplan-Meier method and the log-rank test were used for survival analysis.
Results:
Among the 47 children with AMKL, 22 with non-Down syndrome-AMKL were treated by the AML03 regimen, with a median follow-up time of 11.4 months. For the 22 non-Down syndrome-AMKL patients, the remission rate of bone marrow cytology was 85% and the negative rate of minimal residual disease (MRD) was 79% after induction Ⅱ, with a 2-year overall survival (OS) rate of (50±13)% and a 2-year event-free survival (EFS) rate of (40±12)%. The group with positive immunophenotypic marker CD56 had significantly lower 2-year EFS and OS rates than the group with negative CD56 (P < 0.05). The group without remission of bone marrow cytology after induction Ⅱ had significantly lower 2-year EFS and OS rates than the group with remission (P < 0.05). The group with positive MRD after induction Ⅱ had a significantly lower 2-year EFS rate than the group with negative MRD (P < 0.05). There was no significant difference in 2-year OS and EFS rates between the patients with transplantation and those without transplantation (P > 0.05).
Conclusions:
Children with AMKL tend to have a low remission rate and a poor prognosis. Positive immunophenotypic marker CD56, bone marrow cytology during early treatment response, and MRD results are important factors influencing the prognosis. Allogeneic hematopoietic stem cell transplantation has no significant effect on the prognosis of AMKL.
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