• J. Intern. Med. · Feb 2024

    Multicenter Study

    Sorafenib plus triplet therapy with venetoclax, azacitidine and homoharringtonine for refractory/relapsed acute myeloid leukemia with FLT3-ITD: A multicenter phase 2 study.

    • Sijian Yu, Yu Zhang, Guopan Yu, Yu Wang, Ruoyang Shao, Xin Du, Na Xu, Dongjun Lin, Weihua Zhao, Xiong Zhang, Jie Xiao, Zhiqiang Sun, Lan Deng, Xinquan Liang, Hongyu Zhang, Ziwen Guo, Min Dai, Pengcheng Shi, Fen Huang, Zhiping Fan, Qiong Liu, Ren Lin, Xuejie Jiang, Li Xuan, Qifa Liu, and Hua Jin.
    • Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
    • J. Intern. Med. 2024 Feb 1; 295 (2): 216228216-228.

    BackgroundPatients with relapsed or refractory acute myeloid leukemia (R/R AML) and FLT3-internal tandem duplication (FLT3-ITD) respond infrequently to salvage chemotherapy.ObjectiveTo investigate the efficacy of sorafenib plus triplet therapy with venetoclax, azacitidine, and homoharringtonine (VAH) as a salvage therapy in this population.MethodsThis multicenter, single-arm, phase 2 study was conducted at 12 hospitals across China. Eligible patients had R/R AML with FLT3-ITD (aged 18-65 years) who were treated with VAH. The primary endpoint was composite complete remission (CRc) after two cycles. Secondary outcomes included the overall response rate (ORR), safety, and survival.ResultsBetween July 9, 2020, and March 19, 2022, 58 patients were assessed for eligibility, 51 of whom were enrolled. The median patient age was 47 years (interquartile range [IQR] 31-57). CRc was 76.5% with ORR of 82.4%. At a median follow-up of 17.7 months (IQR, 8.7-24.7), the median duration of CRc was not reached (NR), overall survival was 18.1 months (95% confidence interval [CI], 11.8-NR) and event-free survival was 11.4 months (95% CI, 5.6-NR). Grade 3 or 4 adverse events occurring in ≥10% of patients included neutropenia in 47 (92.2%), thrombocytopenia in 41 (80.4%), anemia in 35 (68.6%), febrile neutropenia in 29 (56.9%), pneumonia in 13 (25.5%), and sepsis in 6 (11.8%) patients. Treatment-related death occurred in two (3.9%) patients.ConclusionsThe sorafenib plus VAH regimen was well tolerated and highly active against R/R AML with FLT3-ITD. This regimen may be a suitable therapeutic option for this population, but larger population trials are needed to be explored.Trial RegistrationClinical Trials Registry: NCT04424147.© 2023 The Association for the Publication of the Journal of Internal Medicine.

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