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2952 Retrospective Comparison of Survival and Risk Stratification By Quantitative RT-PCR Monitoring in Newly Diagnosed Pediatric Acute Myeloid Leukemia with RUNX1::RUNX1T1 Fusion Treated with AML-CAMS-2009 and AML-CAMS-2016

Program: Oral and Poster Abstracts
Session: 619. Acute Myeloid Leukemias: Disease Burden and Minimal Residual Disease in Prognosis and Treatment: Poster II
Hematology Disease Topics & Pathways:
Research, Acute Myeloid Malignancies, AML, Clinical Practice (Health Services and Quality), Clinical Research, Diseases, Real-world evidence, Myeloid Malignancies, Measurable Residual Disease
Sunday, December 8, 2024, 6:00 PM-8:00 PM

Luyang Zhang1,2*, Ye Guo3,4*, Wenyu Yang4,5*, Xiaojuan Chen, MD3,4*, Yumei Chen3,4*, Li Zhang4,5*, Yao Zou4,6*, Xiaofan Zhu, MD4,6 and Min Ruan, MD2,7*

1State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences&Peking Union Medical College, Tianjin, China
2Chinese Academy of Medical Sciences Tianjin Institutes of Health Science, Tianjin, China
3Department of Pediatric Hematology and Oncology, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology& Blood Diseases Hospital, Chinese Academy of Medical Sciences& Peking Union Medical College, Tianjin, China
4Tianjin Institutes of Health Science, Tianjin, China
5State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology& Blood Diseases Hospital, Chinese Academy of Medical Sciences& Peking Union Medical College, Tianjin, China
6State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin, China
7State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China

Objective: The study is intended to compare the clinical outcomes of pediatric patients with AML with RUNX1::RUNX1T1 fusion treated with the AML-CAMS-2009 and AML-CAMS-2016 regimens, as well as to explore early molecular thresholds with prognostic advice. Methods: The study retrospectively analyzed the clinical data of 146 pediatric patients newly diagnosed with AML with RUNX1::RUNX1T1 fusion who received treatment with AML-CAMS-2009 (n = 74) or AML-CAMS-2016 (n = 72) regimens in a single center. Patients on protocol 2009 were followed until December 31, 2020, with a median follow-up of 66 (1-135) months; patients on protocol 2016 were followed until November 30, 2022, with a median follow-up of 43.5 (1-82) months. Results: There was a trend toward improved overall survival with the 2016 regimen in comparison to the 2009 regimen (87.3±4.5% vs. 73.9±5.5%, P = 0.060). At remission following one course of induction chemotherapy, a ≥3 log reduction in RUNX1::RUNX1T1 transcripts in BM had a significant influence on RFS (93.3±6.4% vs. 63.4±8.9%, P = 0.013) and OS (100.0% vs. 69.3±8.6%, P = 0.013) in patients treated with regimen 2009. Compared to the 2009 regimen, the 2016 regimen improved the prognosis of patients with a <3 log reduction in RUNX1::RUNX1T1 transcripts, although the difference was not statistically significant (P = 0.162). Nevertheless, with the 2016 regimen, the worse survival of patients with a < 3 log reduction in fusion gene by induction chemotherapy was not statistically significant (<3 log vs. ≥3 log reduction: RFS 67.7±8.4% vs.91.3±5.9%, P=0.051; OS 83.8±7.4% vs. 100%, P=0.059). Fusion genes detected by quantitative real-time PCR of less than 0.4%, 0.2%, and 0.01% after induction chemotherapy, consolidation 1, and consolidation 2, respectively, significantly affected RFS in patients, both in 2009 and 2016 protocols (P<0.050). Significant improvements in RFS and OS were seen in patients who achieved two or more detections of the above targeted quantification of fusion genes (P<0.005). Conclusion: 1. The 2016 regimen did not result in a decrease in the overall prognosis, despite the fact that it reduced the intensity of therapy. 2. The extent of molecular remission following one course of induction chemotherapy has a substantial impact on the long-term prognosis. 3. Regardless of the regimen, integrating the response to the first three courses of chemotherapy provides prognostic significance.

Disclosures: No relevant conflicts of interest to declare.

*signifies non-member of ASH