Prognostic stratification of molecularly and clinically distinct subgroup in children with acute monocytic leukemia
Li-Peng Liu, Ao-Li Zhang, Min Ruan, Li-Xian Chang, Fang Liu, Xia Chen, Ben-Quan Qi, Li Zhang, Yao Zou, Yu-Mei Chen, Xiao-Juan Chen, Wen-Yu Yang, Ye Guo, Xiao-Fan Zhu, Li-Peng Liu, Ao-Li Zhang, Min Ruan, Li-Xian Chang, Fang Liu, Xia Chen, Ben-Quan Qi, Li Zhang, Yao Zou, Yu-Mei Chen, Xiao-Juan Chen, Wen-Yu Yang, Ye Guo, Xiao-Fan Zhu
Abstract
Background: The prognosis of children with acute monocytic leukemia (AML-M5) remains unsatisfactory and the risk profile is still controversial. We aim to investigate the prognostic value of clinical and cytogenetic features and propose a new risk stratification in AML-M5 children.
Methods: We included 132 children with AML-M5. Overall survival (OS) and progression-free survival (PFS) were documented. Cox regression was performed to evaluate the potential risk factors of prognosis.
Results: The 5-year-OS was 46.0% (95% confidence intervals, 41.6%-50.4%) in all patients. There was significantly lower OS in the age ≤ 3 years old (P = .009) and hyperleukocytosis (P < .001). The FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) and MLL-rearrangement carriers were associated with fewer survivors in all patients (37.1% and 36.7%) and chemotherapy-only group (19.0% and 35.0%). Notably, the number of survivor with MLL-rearrangement did not increase in hematopoietic stem cell transplant (HSCT) group. According to the Cox regression analysis, HSCT was a significantly favorable factor (P = .001), while hyperleukocytosis, age ≤ 3 years old, and BM blast ≥ 70% adversely affected the OS in all patients (all P < .05). Additionally, FLT3-ITD was a risk factor for OS in the chemotherapy-only group (P = .023), while hyperleukocytosis and age ≤ 3 years independently contributed to poor PFS (both P < .05). In comparison to the standard-risk group, significant poorer outcome was found in the high-risk group (both P < .005).
Conclusions: We propose that AML-M5 children with any of MLL-rearrangement, FLT3-ITD, hyperleukocytosis, BM blast ≥ 70%, or age ≤ 3 years old are classified into the high-risk group, and HSCT is beneficial especially in patients with FLT3-ITD mutation, hyperleukocytosis, and age ≤ 3 years old. Importantly, the choice of HSCT should be made more carefully in children with MLL-rearrangement for its suboptimal performance.
Keywords: acute monocytic leukemia; children; clinical characteristics; gene mutation; prognostic factors.
Conflict of interest statement
None declared.
© 2020 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.
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Source: PubMed