Impact of genetic patterns on sorafenib efficacy in patients with FLT3-ITD acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation: a multi-center, cohort study

Sorafenib therapy improves overall survival (OS) in patients with FLT3 internal tandem duplication (ITD) acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation. We explored the efficacy of sorafenib therapy in this population with different concomitant genetic pat...

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Published in:Signal transduction and targeted therapy 2023-09, Vol.8 (1), p.348-11, Article 348
Main Authors: Shao, Ruoyang, Zhang, Yu, He, Jinping, Huang, Fen, Fan, Zhiping, Yang, Kaibo, Xu, Yajing, Xu, Na, Luo, Yi, Deng, Lan, Zhang, Xi, Chen, Jia, Han, Mingzhe, Li, Xudong, Yu, Sijian, Liu, Hui, Liang, Xinquan, Luo, Xiaodan, Shi, Pengcheng, Wang, Zhixiang, Jiang, Ling, Zhou, Xuan, Lin, Ren, Chen, Yan, Tu, Sanfang, Sun, Jing, Wang, Yu, Liu, Qifa, Xuan, Li
Format: Article
Language:English
Subjects:
692/4028/67/1990
692/699/1541/1990
Acute myeloid leukemia
Allografts
Cancer Research
Cell Biology
Cohort analysis
DNA methylation
Hematopoietic stem cells
Inhibitor drugs
Internal Medicine
Leukemia
Medicine
Medicine & Public Health
Oncology
Pathology
Population genetics
Risk groups
Stem cell transplantation
Targeted cancer therapy
Transcription factors
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Summary:Sorafenib therapy improves overall survival (OS) in patients with FLT3 internal tandem duplication (ITD) acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation. We explored the efficacy of sorafenib therapy in this population with different concomitant genetic patterns. In this multi-center, cohort study, we enrolled patients with FLT3-ITD AML undergoing allogenic hematopoietic cell transplantation. Patients with sorafenib maintenance post-transplantation for at least four weeks were allocated to the sorafenib group, and otherwise to the control group. Endpoints were OS, disease-free survival, and relapse for the whole cohort and OS for genetic pattern subgroups. Among 613 patients enrolled, 275 were in the sorafenib and 338 the control group. Median follow-up was 36.5 (interquartile range (IQR), 25.2–44.7) months post-transplantation. The 3-year OS post-transplantation was 79.6% (95% confidential interval (CI) 74.8%–84.6%) and 65.2% (95% CI 60.3%–70.6%) (Hazard ratio (HR) 0.50, 95% CI 0.37–0.69; P  
ISSN:2059-3635
2095-9907
2059-3635
DOI:10.1038/s41392-023-01614-1