EBMT Paris study office / CEREST-TC, Paris, France, Department of Hematology, Saint Antoine Hospital, Paris, France, INSERM UMR 938, Sorbonne University, Paris, France.
Department of Internal Medicine, Jacobi Medical Center/Einstein College of Medicine, Bronx, NY, USA.
Bone Marrow Transplant. 2024 Oct;59(10):1458-1465. doi: 10.1038/s41409-024-02373-5. Epub 2024 Aug 2.
Core-binding factor acute myeloid leukemia (CBF-AML) represents 12-15% of all AML cases. Although CBF positivity infers a survival advantage, overall survival (OS) remains dismal. Treatment is with cytarabine/anthracycline-based chemotherapy induction followed by high-dose cytarabine (HiDAC) consolidation. Allogeneic hematopoietic stem cell transplantation (allo-SCT) is reserved for relapse or for patients having not achieved MRD-negativity at high risk for relapse. The role of SCT in first complete remission (CR1) remains controversial and is considered in high risk conditions. In this retrospective, multi-national, European Society for Blood and Marrow Transplantation (EBMT)-based study, we identified 1901 patients with de novo CBF-AML who received an allo-SCT or autologous transplantation (ASCT) in CR1. 65.5% harbored t(8;21) and 34.4% inv(16). In this group, the majority (77%) were treated with allo-SCT in CR1. In multivariate analysis, treatment with allo-SCT was an independent and significant, negative predictor of NRM and OS (HR 4.26, p < 0.0001 and HR 1.67, p = 0.003) and among patients treated with allo-SCT, those treated with MSD had the best outcomes, comparable to those treated with ASCT. There was no interaction between the type of transplant and MRD status at time of SCT. In both, MRD-negative and MRD-positive groups, NRM was worse in the allo-SCT group (MRD-: 12.9% vs 5.2%, p = 0.007; MRD+: 10.6% vs 0%, p = 0.004). We therefore demonstrated that consolidation in CR1 with allo-SCT results in worse outcomes than ASCT. Whether consolidation with ASCT yields better outcomes than chemotherapy alone or chemotherapy in combination with Gemtuzumab Ozogamicin is yet to be investigated.
核心结合因子急性髓系白血病（CBF-AML）占所有 AML 病例的 12-15%。尽管 CBF 阳性提示生存优势，但总体生存率（OS）仍然很差。治疗方法是使用阿糖胞苷/蒽环类药物为基础的化疗诱导，然后进行高剂量阿糖胞苷（HiDAC）巩固。异基因造血干细胞移植（allo-SCT）仅用于复发或高复发风险的患者未能达到微小残留病（MRD）阴性。SCT 在首次完全缓解（CR1）中的作用仍存在争议，并在高危情况下考虑。在这项回顾性、多国家、欧洲血液和骨髓移植学会（EBMT）为基础的研究中，我们确定了 1901 例新诊断的 CBF-AML 患者，他们在 CR1 中接受了 allo-SCT 或自体移植（ASCT）。65.5%的患者存在 t(8;21)，34.4%的患者存在 inv(16)。在这组患者中，大多数（77%）在 CR1 中接受 allo-SCT 治疗。多变量分析显示，allo-SCT 治疗是 NRM 和 OS 的独立且显著的负预测因素（HR 4.26，p<0.0001 和 HR 1.67，p=0.003），在接受 allo-SCT 治疗的患者中，接受 MSD 治疗的患者结局最好，与接受 ASCT 治疗的患者相当。在移植类型和 SCT 时的 MRD 状态之间没有相互作用。在 MRD 阴性和 MRD 阳性组中，allo-SCT 组的 NRM 更差（MRD-：12.9% vs 5.2%，p=0.007；MRD+：10.6% vs 0%，p=0.004）。因此，我们证明了在 CR1 中用 allo-SCT 巩固治疗比 ASCT 更差。用 ASCT 巩固治疗是否比单独化疗或化疗联合吉妥珠单抗奥佐米星的疗效更好，仍有待研究。
