复发难治性急性髓系白血病（R-R AML）患者，特别是非缓解（NR）患者，在异基因造血干细胞移植（allo-HSCT）后预后差。为了优化R-R AML患者的整个allo-HSCT过程，并确定影响HSCT后临床结果的潜在因素，我们回顾性分析了2013年至2022年在华中科技大学同济医学院协和医院血液病研究所进行救治性allo-HSCT的44名成年R-R AML患者，这些患者在NR状态下或合并自髓外白血病。 44名患者的1年和2年总生存率（OS）分别为55.3％（95％置信区间[CI]，41.1％-74.3％）和44.4％（95％CI，30.2％-65.4％）。1年和2年累积复发率（CIR）分别为39.4％（95％CI，38.0％-40.7％）和53.0％（95％CI，51.0％-55.1％），1年和2年无白血病生存率（LFS）分别为37.8％（95％CI，24.8％-57.7％）和20.3％（95％CI，9.1％-45.3％）。100天、1年和2年的治疗相关性死亡率（TRM）分别为13.8％（95％CI，13.3％-14.4％）、22.8％（95％CI，21.9％-23.7％）和26.7％（95％CI，25.5％-27.8％）。多因素分析显示，移植后出现慢性移植物抗宿主病（cGVHD）的患者复发率较低。我们的分析还表明，骨髓中的生物计数<20％和≥20％的患者在allo-HSCT后具有可比的临床结果。总之，我们的研究表明，在移植时无论白血病负担如何，NR状态或合并自髓外白血病的R-R AML患者都可以从allo-HSCT中受益。接受allo-HSCT后出现cGVHD的患者可能由于增强的移植物抗白血病（GVL）效应而具有较低的复发率，但应控制cGVHD在轻度至中度水平，以避免危及生命的并发症。© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.

Patients with relapsed and refractory acute myeloid leukemia (R-R AML), especially those in non-remission (NR) have a poor prognosis after allogeneic hematopoietic stem cell transplantation (allo-HSCT). In order to optimize the entire allo-HSCT process for R-R AML patients and identify potential factors affecting clinical outcomes after HSCT, we retrospectively analyzed 44 adult patients with R-R AML who underwent salvage allo-HSCT while in NR or with concomitant extramedullary leukemia at the Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology from 2013 to 2022. The 1-year and 2-year overall survival (OS) of the 44 patients were 55.3% (95% confidence interval [CI], 41.1%-74.3%) and 44.4% (95%CI, 30.2%-65.4%), respectively. The 1-year and 2-year cumulative incidence of relapse (CIR) were 39.4% (95%CI, 38.0%-40.7%) and 53.0% (95%CI, 51.0%-55.1%), respectively, and the 1-year and 2-year leukemia-free survival (LFS) were 37.8% (95%CI, 24.8%-57.7%) and 20.3% (95%CI, 9.1%-45.3%), respectively. The 100-day, 1-year and 2-year treatment-related mortality (TRM) was 13.8% (95%CI, 13.3%-14.4%), 22.8% (95%CI, 21.9%-23.7%) and 26.7% (95%CI, 25.5%-27.8%), respectively. Multivariate analysis revealed that patients who developed chronic graft-versus-host disease (cGVHD) after transplantation had lower relapse rate. Our analysis also indicated that patients with blast counts in bone marrow (BM) <20% and those with ≥20% had comparable clinical outcomes after allo-HSCT. In conclusion, our study demonstrated that R-R AML patients in NR or with concomitant extramedullary leukemia can benefit from allo-HSCT, regardless of leukemia burden at the time of transplantation. Patients who experience cGVHD after allo-HSCT may have lower relapse rate due to enhanced graft-versus-leukemia (GVL) effects, but cGVHD should be controlled at mild to moderate level to avoid life-threatening complications.© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.