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[Study of clinical outcome and prognosis in pediatric core binding factor-acute myeloid leukemia]
1Department of Pediatrics, People's Hospital, Peking University, Beijing 100044, China.
Insights
Pediatric core binding factor-acute myeloid leukemia (CBF-AML) shows good chemotherapy response. Additional chromosomal abnormalities are the sole independent risk factor impacting overall survival in these young patients.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Research
Background:
- Core binding factor-acute myeloid leukemia (CBF-AML) is a distinct subtype of childhood leukemia.
- Understanding prognostic factors is crucial for optimizing treatment strategies in pediatric CBF-AML.
Purpose of the Study:
- To evaluate the clinical outcomes, including remission rates, relapse, and survival, in pediatric patients with CBF-AML.
- To identify independent prognostic factors influencing overall survival in this patient cohort.
Main Methods:
- Retrospective review of 121 newly diagnosed pediatric CBF-AML patients.
- Kaplan-Meier method for survival analysis (CIR, EFS, OS).
- Cox regression analysis for prognostic factor evaluation.
Main Results:
- High complete remission rates (83.3% after first, 99.2% after second chemotherapy course).
- 3-year overall survival rate of 82.8% and event-free survival of 77.5%.
- Additional chromosomal abnormalities identified as the only independent risk factor for overall survival (HR=4.289, P=0.040).
Conclusions:
- Pediatric CBF-AML presents unique prognostic subtypes with favorable responses to chemotherapy.
- Additional chromosomal abnormalities represent a significant independent risk factor for overall survival in pediatric CBF-AML.
Abstract:
Objective: To analyze the clinical outcome and the prognostic factor in pediatric patients with core binding factor-acute myeloid leukemia (CBF-AML). Methods: A total of 121 newly diagnosed pediatric CBF-AML patients enrolled from Aug. 2005 to Sep. 2017 were retrospectively reviewed. Cumulative incidence of relapse (CIR), event-free survival (EFS) and overall survival (OS) rates were estimated by Kaplan-Meier method and prognostic factors were evaluated by Cox regression with SPSS. Results: Of the 121 patients, 120 patients were assessed for bone marrow remission after induction chemotherapy. 100 cases (83.3%) achieved complete remission (CR) after the first course of chemotherapy. 119 cases (99.2%) achieved CR after the second course of chemotherapy. Of the 121 patients, 13 patients (10.7%) had recurrence with the median interval of recurrence as 13.8 months (3.7 to 58.8 months). 17 patients (14.0%) died. The CIR, EFS and OS at 3 years were 12.7%, 77.5% and 82.8%, respectively. The factors including age at diagnosis, sex, initial WBC count, presence of extramedullary leukemia, C-KIT expression, additional chromosomal abnormalities, and CR after the first course of chemotherapy were analyzed by multivariate regression analysis of Cox. Multivariate analysis identified that additional chromosomal abnormalities was the only independent risk factor affecting OS (HR=4.289, 95%CI 1.070-17.183, P=0.040). Conclusions: Pediatric CBF-AML was a unique setting of prognostic subtypes. Chemotherapy produced good responses. Additional chromosomal abnormalities was the only independent risk factor for OS in pediatric CBF-AML.
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