Clinical Characteristics and Prognostic Analysis of Pediatric Acute Myeloid Leukemia With CBFB::MYH11: A 10-Year Single-Center Cohort Study.
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INTRODUCTION: CBFB::MYH11-positive AML is classified as favorable-risk, yet outcomes vary. The prognostic roles of KIT mutations, additional chromosomal abnormalities (ACAs), and minimal residual disease (MRD) kinetics remain debated, particularly in Chinese pediatric cohorts. PATIENTS AND METHODS: We retrospectively analyzed 66 consecutive pediatric patients with CBFB::MYH11-positive AML. Cytogenetic and molecular data were collected, and MRD was monitored by real-time quantitative PCR. Overall survival (OS), event-free survival (EFS), and cumulative incidence of relapse (CIR) were estimated using Kaplan-Meier and competing risk methods. RESULTS: At a median follow-up of 65 months, 5-year OS, EFS, and CIR were 97.0%, 88.7%, and 8.6%. KIT mutations were present in 56.1% and were not associated with EFS (hazard ratios 0.58, 95% confidence interval 0.13-2.59). ACAs correlated with lower first-course remission rates and higher central nervous system leukemia incidence but not survival. Postinduction MRD positivity was almost universal (94.9%), and no MRD cut-off at assessed time points was associated with EFS. Of 13 transcript re-emergences, 3 progressed to relapse, and 8 (peak ≤0.14%) did not, 4 without therapy modification. Five-year EFS was 90.4% with chemotherapy in first complete remission (CR1; n = 58) and 100% after CR1 transplantation (n = 6). CONCLUSION: This study confirms excellent prognosis of pediatric CBFB::MYH11-positive AML with modern therapy. Owing to the few events, it could neither establish nor exclude an effect of traditional risk factors. Low-level molecular re-emergence frequently resolved without relapse, and the data are consistent with, but do not independently establish, current recommendations against routine CR1 transplantation. Larger studies are needed to identify patients for treatment intensification.