复发/难治性急性髓系白血病（r/rAML）的预后很差，同种异体造血干细胞移植（allo-HSCT）是一种潜在的治愈方法。联合抗 PD-1、低甲基化剂 (HMA) 和 CAG（阿糖胞苷、阿克拉比星/伊达比星、粒细胞集落刺激因子）方案在 r/rAML 中显示出主要疗效。然而，移植前暴露于抗PD-1可能会导致严重的移植物抗宿主病（GVHD）。本初步研究旨在评估接受抗 PD-1 HMA CAG 方案的 r/rAML 患者中异基因 HSCT 的安全性和有效性。 15 名 r/rAML 患者（12 名相关单倍体供体 [HID]、2 名匹配的兄弟姐妹、1 名不相关的患者）供者）接受了该方案，并随后进行了外周血 HSCT。四名 HID 患者接受了 GVHD 预防方案，包括抗胸腺细胞球蛋白和减少剂量的移植后环磷酰胺。中位随访时间为 20.9 个月（范围：1.2-34.2）。 2-4级和3-4级急性GVHD的累积发生率分别为40%和13.3%。中重度慢性 GVHD、非复发死亡率和复发的 2 年发生率分别为 10%、22.3% 和 22.5%。 2 年总生存率和无 GVHD/无复发生存率分别为 54% 和 48.6%。 PTCy 组未观察到死亡或复发。抗 PD-1 HMA CAG 方案桥接异基因 HSCT 治疗 r/r AML 的耐受性良好，疗效良好。使用 PTCy 预防 HID-HSCT 的 GVHD 表现出初步的生存优势。版权所有 © 2024 Wang、Wang、Su、Wang、Li、Li、Jing、Xu、Wang、Zheng、Gao、Hu、Gao 和 Liu。

The prognosis of relapsed/refractory acute myeloid leukemia (r/rAML) is dismal, and allogeneic hematopoietic stem cell transplant (allo-HSCT) is a potential cure. Combining anti-PD-1, hypomethylating agent (HMA), and CAG (cytarabine, aclarubicin/idarubicin, granulocyte colony-stimulating factor) regimen has showed primary efficacy in r/rAML. However, pre-transplant exposure to anti-PD-1 may lead to severe graft-versus-host disease (GVHD). This preliminary study aimed to evaluate the safety and efficacy of allo-HSCT in r/rAML patients receiving the anti-PD-1+HMA+CAG regimen.Fifteen r/rAML patients (12 related haploidentical donors [HIDs], 2 matched siblings, 1 unrelated donor) received this regimen and subsequent peripheral blood HSCT.Four patients with HIDs received a GVHD prophylaxis regimen consisted of Anti-thymocyte globulin and a reduced-dose of post-transplant cyclophosphamide. The median follow-up was 20.9 months (range, 1.2-34.2). The cumulative incidences of acute GVHD grade 2-4 and grade 3-4 were 40% and 13.3%, respectively. The 2-year incidence of moderate-to-severe chronic GVHD, non-relapse mortality, and relapse were 10%, 22.3%, and 22.5%, respectively. The 2-year overall survival and GVHD-free/relapse-free survival rates were 54% and 48.6%, respectively. No death or relapse was observed in the PTCy group.The anti-PD-1+HMA+CAG regimen bridging to allo-HSCT for r/r AML was tolerable with promising efficacy. GVHD prophylaxis with PTCy for HID-HSCT showed preliminary survival advantage.Copyright © 2024 Wang, Wang, Su, Wang, Li, Li, Jing, Xu, Wang, Zheng, Gao, Hu, Gao and Liu.