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。
