探讨CBA （克拉屈滨、白消安、阿糖胞苷）联合用药预处理方案的异基因造血干细胞移植（allo-HSCT）治疗高危急性髓系白血病（AML）和骨髓增生异常综合征（MDS）的疗效及安全性。

回顾性分析2018年1月至2024年5月于联勤保障部队第九四〇医院血液科行CBA强化预处理方案的allo-HSCT治疗的AML/MDS患者临床资料。总结植入情况、移植物抗宿主病（GVHD）发生率、感染、复发率、总生存（OS）和无进展生存（PFS）等。Kaplan-Meier法分析生存率，Fine-Gray竞争风险模型分析累积复发率及非复发死亡率，采用Log-rank检验比较组间差异。

24例患者纳入研究，中位年龄35 （6 ~ 52）岁，AML 15例，MDS 9例，其中13例为复发/难治性AML。所有患者粒细胞均成功植入，粒细胞中位植入时间18 （13 ~ 22）d。22例患者血小板成功植入，血小板中位植入时间17 （9 ~ 30）d，2例血小板植入功能不良经治疗后缓解。急慢性GVHD的发生率分别为45.8%、16.7%。24例患者移植后均获完全缓解（CR），流式MRD阴性。中位随访44.5 （1.5 ~ 84）月，预期移植后3年OS率为57.8% （95 %CI：35.7 % ~ 74.6 %），PFS率为48.9 % （95 %CI：27.8 % ~ 67.0 %）。AML和MDS患者3年OS率分别为52.5 % （95 %CI：25.2 % ~ 73.9 %）、66.7 % （95 %CI：28.2 % ~ 87.8 %），PFS率分别为45.0 % （95 %CI：19.4 % ~ 67.8 %）、55.6 % （95 % CI：20.4 % ~ 80.4 %），两者无统计学差异。移植后3年累积复发率为21.6 % （95 %CI：7.5 % ~ 40.4 %），非复发死亡率为29.4 % （95 %CI：12.6 % ~ 48.4 %）。

高危AML/MDS采用CBA预处理方案，移植后复发率较低，总生存期长，安全性良好。

To evaluate the efficacy and safety of the cladribine, busulfan, and cytarabine (CBA) -based intensified conditioning regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) .

Clinical data of AML/MDS patients who underwent CBA-based allo-HSCT at the Department of Hematology, 940th Hospital of the Joint Logistics Support Force from January 2018 to May 2024 were retrospectively analyzed. Engraftment kinetics, incidence of graft-versus-host disease (GVHD), infections, relapse rates, overall survival (OS), and progression-free survival (PFS) were summarized. Survival rates were analyzed using the Kaplan-Meier method, while cumulative incidence of relapse and non-relapse mortality were evaluated by the Fine-Gray competing risk model. Differences between groups were compared using the Log-rank test.

Twenty-four patients were included, with a median age of 35 (range: 6 ~ 52) years including 15 AML cases (including 13 relapsed/refractory AML) and 9 MDS cases. All patients achieved successful neutrophil with median time to engraftment of 18 (13 ~ 22) days. 22 patients achieved successful platelet engraftment with median time to engraftment of 17 (9 ~ 30) days, and 2 patients of poor platelet engraftment function were relieved after treatment. The incidences of acute and chronic GVHD were 45.8 % and 16.7 %, respectively. All 24 patients achieved complete remission (CR) after transplantation, with the flow cytometry-confirmed MRD was negative. After a median follow-up of 44.5 (1.5 ~ 84) months, the estimated 3-year OS and PFS rates were 57.8% (95%CI: 35.7%~74.6%) and 48.9% (95%CI: 27.8% ~ 67.0%), respectively. The 3-year OS rates of AML and MDS patients were 52.5% (95%CI: 25.2%~ 73.9%) and 66.7% (95%CI: 28.2%~ 87.8%), while PFS rates were 45.0% (95%CI: 19.4%~ 67.8%) and 55.6% (95%CI: 20.4%~ 80.4%), showing no statistically significant difference. The 3-year cumulative relapse rate and non-relapse mortality were 21.6% (95%CI: 7.5% ~ 40.4%) and 29.4% (95%CI: 12.6%~ 48.4%), respectively.

For high-risk AML/MDS patients, receiving the CBA conditioning regimen demonstrates low relapse rates, prolonged overall survival, and favorable safety profiles post-allo-HSCT.