National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Department of Clinical Hematology and Cellular Therapy, Saint-Antoine Hospital, AP-HP, Sorbonne University, EBMT Paris office, Paris, France.
Bone Marrow Transplant. 2023 Dec;58(12):1322-1330. doi: 10.1038/s41409-023-02070-9. Epub 2023 Aug 28.
In patients with acute myeloid leukemia (AML) of intermediate-risk (IR) in first remission (CR1) with no measurable residual disease (MRD negative), the choice of the best consolidation is questionable. 1122 adult patients from 196 centers, transplanted in 2010-21 were analyzed: 547 received an autologous stem cell transplantation (ASCT) and 575 a Haploidentical donor transplant. Because of a significant interaction, comparisons were done separately for patients with wild-type FLT3 (FLT3-wt) and FLT3-ITD mutation (FLT3-ITD). In FLT3-wt patients, haploidentical transplants had two year lower relapse incidence (RI) (16.9% versus 32.6%; HR = 0.40, p < 0.001), higher NRM higher (17.2% vs 3.5%; HR = 7.02, p < 0.001), similar LFS (65.9% vs 63.8%; p = 0.37) and lower OS (73.2% vs 80.6%; HR = 1.69, p = 0.018). In FLT3-ITD patients, haploidentical transplants had two year lower RI (8.2% vs 47.8%; HR = 0.14, p < 0.001) higher NRM (20.2% vs 5.6%; HR = 3.43, p = 0.002), better LFS (71.5% vs 46.6%; HR = 0.53, p = 0.007) and similar OS (73.5% vs 61.9%; p = 0.44). In IR AML patients with FLT3-wt in MRD negative CR1, autologous stem cell transplantation is a valid option, while in patients with FLT3-ITD, haploidentical transplant is better. Whether autologous transplantation is superior to chemotherapy in FLT3-wt patients and the role of maintenance therapy with FLT3 inhibitors remain to be studied.
在无微小残留病(MRD 阴性)的中危(IR)急性髓系白血病(AML)首次缓解(CR1)患者中,最佳巩固治疗方案的选择仍存在争议。对 2010-2021 年 196 个中心的 1122 例成人患者进行了分析:547 例接受了自体干细胞移植(ASCT),575 例接受了单倍体相合供者移植。由于存在显著的交互作用,因此分别对野生型 FLT3(FLT3-wt)和 FLT3 内部串联重复突变(FLT3-ITD)患者进行了比较。在 FLT3-wt 患者中,单倍体相合移植的 2 年复发率(RI)较低(16.9% vs 32.6%;HR=0.40,p<0.001),非复发死亡率(NRM)较高(17.2% vs 3.5%;HR=7.02,p<0.001),无事件生存率(EFS)相似(65.9% vs 63.8%;p=0.37),总生存率(OS)较低(73.2% vs 80.6%;HR=1.69,p=0.018)。在 FLT3-ITD 患者中,单倍体相合移植的 2 年 RI 较低(8.2% vs 47.8%;HR=0.14,p<0.001),NRM 较高(20.2% vs 5.6%;HR=3.43,p=0.002),EFS 较好(71.5% vs 46.6%;HR=0.53,p=0.007),OS 相似(73.5% vs 61.9%;p=0.44)。在 MRD 阴性 CR1 的中危 AML 患者中,FLT3-wt 患者自体干细胞移植是一种有效的选择,而在 FLT3-ITD 患者中,单倍体相合移植则更好。FLT3-wt 患者中自体移植是否优于化疗,以及 FLT3 抑制剂维持治疗的作用仍有待研究。