Abstract
Purpose: To evaluate the outcome and prognostic significance of FLT3/ITD allelic ratios (AR) among pediatric acute myeloid leukemia (AML) from TARGET dataset. Methods: A total of 1,857 pediatric patients who were diagnosed with AML were enrolled in this study by using a retrospective cohort study method from September 1996 to December 2016. Results: The prevalence of FLT3/ITD mutation in the whole cohort was 18.4%. Generalized additive models and threshold effect analysis using piece-wise linear regression were applied to identify the cut-off value of 0.5 on FLT3/ITD AR. High FLT3/ITD AR were significantly associated with higher WBC ( P <0.001), higher proportion of peripheral blood blast and bone marrow blast (both of P <0.001), high risk group ( P <0.001), more common to have FAB M1 ( P <0.001) and lower complete remission induction rates ( P =0.048). Overall, high FLT3/ITD AR patients had a significantly inferior 5-year EFS ( P =0.036) and OS ( P =0.04) compared to low AR patients. Stem cell transplantation (SCT) had no significant impact on the survival of patients with low FLT3/ITD AR patients, while SCT may improve the outcome patients with high AR. Multivariate analysis further confirmed that high FLT3/ITD AR were an independent adverse indicators of inferior outcome in terms of EFS ( P =0.017) and OS ( P =0.032). Conclusions: The current study demonstrates that high FLT3/ITD AR, but not the presence of FLT3/ITD, are independent poor prognostic factors in pediatric AML. Furthermore, the optimal cut-off value for FLT3/ITD AR is 0.5, which could serve as an useful marker for risk-stratified therapy and prognostic evaluation. Funding Statement: This work was supported by the Guangdong Basic and Applied Basic Research Foundation (NO.2021A1515011809), Guangzhou Science and Technology Program key projects (No.201803010032), and Bethune Medical Scientific Research Fund Project (No.SCE111DS). Declaration of Interests: The authors declare that they have no competing interests. Ethics Approval Statement: The study protocol and informed consent were approved by the Ethics Committee of COG.
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