Allogeneic hematopoietic cell transplantation (allo‐HCT) is recommended for core‐binding factor mutated (CBF) AML patients achieving second complete remission (CR2). However, approximately 20% of patients may relapse after transplant and donor preference remains unclear. We compared in this EBMT global multicenter registry‐based analysis the allo‐HCT outcomes using either haploidentical (Haplo), matched siblings donors (MSD), or 10/10 matched unrelated donors (MUD). Data from 865 de novo adult CBF AML patients in CR2 receiving allo‐HCT in 227 EBMT centers from 2010 to 2022 were analyzed, in which 329 MSD, 374 MUD, and 162 Haplo‐HCTs were included. For the entire cohort, 503 (58%) patients were inv(16)/CBFB‐MYH11 and 362 patients (42%) were t(8;21)/RUNX1‐RUNX1T1 AML. On multivariate analysis, Haplo‐HCT was associated with a lower Relapse Incidence (RI) compared to either MSD (hazard ratio [HR] = 0.56, 95% CI 0.32–0.97; p < .05) or MUD (HR = 0.57, 95% CI: 0.33–0.99, p < .05). No significant difference was observed among the 3 types of donors on LFS, OS and GRFS. CBF‐AML with t(8;21) was associated with both higher RI (HR = 1.79, 95% CI 1.3–2.47; p < .01) and higher NRM (HR = 1.58, 95% CI 1.1–2.27; p < .01) than CBF‐AML with inv(16), which led to worse LFS, OS and GRFS. To conclude, for CBF‐AML patients in CR2, Haplo‐HCTs were associated with a lower RI compared to MSD and MUD allo‐HCTs. There was no difference on LFS, OS or GRFS. CBF AML patients with inv(16) had a better progonosis than those with t(8;21) after allo‐HCT in CR2.

中文翻译：

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CR2 期核心结合因子 AML 患者的半相合造血细胞移植与匹配的兄弟姐妹或无关供体造血细胞移植相比，复发率较低：来自欧洲血液和骨髓移植学会全球委员会和急性白血病工作组的一项研究

对于核心结合因子突变 (CBF) AML 患者，建议实现第二次完全缓解 (CR2) 时进行同种异体造血细胞移植 (allo-HCT)。然而，大约 20% 的患者可能在移植后复发，且供体偏好仍不清楚。我们在这项基于 EBMT 全球多中心注册的分析中，使用半相合 (Haplo)、匹配的兄弟姐妹供体 (MSD) 或 10/10 匹配的无关供体 (MUD) 比较了异基因 HCT 的结果。分析了 2010 年至 2022 年在 227 个 EBMT 中心接受 allo-HCT 的 865 例 CR2 新生 CBF AML 成人患者的数据，其中包括 329 例 MSD、374 例 MUD 和 162 例 Haplo-HCT。对于整个队列，503 名患者 (58%) 接受了 inv(16)/CBFB-MYH11 治疗，362 名患者 (42%) 接受了 inv(16)/CBFB-MYH11 治疗。t(8;21)/RUNX1-RUNX1T1 AML。在多变量分析中，与任一 MSD 相比，Haplo-HCT 的复发率 (RI) 较低（风险比 [HR] = 0.56，95% CI 0.32–0.97；p< .05) 或 MUD (HR = 0.57, 95% CI: 0.33–0.99,p< .05)。 3类供者的LFS、OS和GRFS没有观察到显着差异。 CBF-AML 与t(8;21) 与较高的 RI 相关（HR = 1.79，95% CI 1.3–2.47；p< .01）和更高的 NRM（HR = 1.58，95% CI 1.1–2.27；p< .01) 比使用 inv(16) 的 CBF-AML 更差，这导致 LFS、OS 和 GRFS 更差。总之，对于 CR2 期的 CBF-AML 患者，与 MSD 和 MUD allo-HCT 相比，Haplo-HCT 的 RI 较低。 LFS、OS 或 GRFS 没有差异。患有 inv(16) 的 CBF AML 患者比患有 inv(16) 的 CBF AML 患者具有更好的预后t(8;21) CR2 中异基因 HCT 后。