Jiang Zhonghui, Zhou Ya, Bai Yuxin, Dai Fang, Fan Menglin, Zhang Tian, Nie Danian, Zeng Yunxin, Jiang Yirong, Zhu Ping, Fan Zhiping, Xu Na, Huang Fen, Lin Ren, Dai Min, Xu Xiaojun, Li Zhangkun, Jin Hua, Sun Jing, Liu Qifa, Xuan Li
Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, 510515, China.
Clinical Medical Research Center of Hematology Diseases of Guangdong Province, Guangzhou, China.
Biomark Res. 2025 Aug 20;13(1):106. doi: 10.1186/s40364-025-00816-9.
There is no established standard treatment for acute myeloid leukemia (AML) patients with FLT3 wild-type relapsing after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Multi-kinase inhibitor sorafenib has been widely explored in the treatment of AML patients with FLT3 internal tandem duplication (FLT3-ITD) mutations. Some studies have revealed that the addition of sorafenib to standard chemotherapy could improve outcomes in newly diagnosed AML regardless of FLT3 status. However, the application of sorafenib in FLT3 wild-type AML patients experiencing relapse after allo-HSCT remains minimally investigated.
We retrospectively compared the effects of conventional treatment combined with or without sorafenib on the outcomes of these patients. The study mainly focused on the treatment response of salvage therapy and survival.
Sixty-two AML patients with FLT3 wild-type who relapsed after allo-HSCT were enrolled in this study, including 38 with sorafenib and 24 without sorafenib. Fifty patients received 68 doses of donor lymphocyte infusion (DLI). The rate of composite complete remission was 65.8% in the sorafenib group, compared with 29.2% in the non-sorafenib group (P = 0.005). With a median follow-up of 13.2 months (IQR, 3.2-43.5) after relapse, the 2-year overall survival (OS) was 47.4% (95%CI, 33.9%-66.2%) and 16.7% (6.8%-40.8%) in the sorafenib and non-sorafenib groups (P = 0.006). The 2-year event-free survival (EFS) was 44.7% (31.4%-63.7%) and 16.7% (6.8%-40.8%) in the two groups (P = 0.012). Multivariable analysis revealed that salvage therapy including sorafenib was the protective factor for longer OS and EFS (HR = 0.395, 95% CI: 0.209-0.746, P = 0.004; HR = 0.406, 95% CI: 0.218-0.754, P = 0.004). The incidences of acute graft-versus-host disease (GVHD) and chronic GVHD were similar between the two groups (P = 0.806, P = 0.908).
Our results suggest that salvage therapy including sorafenib and DLI is associated with improved outcomes for AML patients with FLT3 wild-type relapsing after allo-HSCT.
对于异基因造血干细胞移植（allo-HSCT）后复发的FLT3野生型急性髓系白血病（AML）患者，尚无既定的标准治疗方案。多激酶抑制剂索拉非尼已被广泛用于治疗伴有FLT3内部串联重复（FLT3-ITD）突变的AML患者。一些研究表明，无论FLT3状态如何，在标准化疗中加入索拉非尼均可改善新诊断AML患者的预后。然而，索拉非尼在allo-HSCT后复发的FLT3野生型AML患者中的应用仍研究较少。
我们回顾性比较了常规治疗联合或不联合索拉非尼对这些患者预后的影响。该研究主要关注挽救治疗的反应和生存情况。
本研究纳入了62例allo-HSCT后复发的FLT3野生型AML患者，其中38例接受索拉非尼治疗，24例未接受索拉非尼治疗。50例患者接受了68次供者淋巴细胞输注（DLI）。索拉非尼组的完全缓解率为65.8%
