Objective: To investigate and verify a novel acute graft versus host disease (aGVHD) prevention protocol in the context of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) . Methods: Patients who underwent haplo-HSCT in our center between January 2022 and December 2022 were included. All patients received reduced doses of cyclophosphamide, Rabbit anti-human tymoglobulin, ruxolitinib, methotrexate, cyclosporine, and MMF to prevent aGVHD. The transplantation outcomes, complications, and survival rate of all patients were investigated. Results: A total of 52 patients with haplo-HSCT were enrolled, 29 (55.8%) male and 23 (44.2%) female, with a median age of 28 (5-59) years. There were 25 cases of acute myeloid leukemia, 17 cases of acute lymphocyte leukemia, 6 cases of myelodysplastic syndrome, 2 cases of chronic myeloid leukemia and 2 cases of myeloproliferative neoplasms. 98.1% of patients had successful engraftment. The incidence of Ⅱ-Ⅳ aGVHD and Ⅲ-Ⅳ aGVHD was 19.2% (95% CI 8.2% -30.3% ) and 7.7% (95% CI 0.2% -15.2% ), respectively. No patients experienced severe gastrointestinal mucositis. The Epstein-Barr virus and CMV reactivation rates were 40.4% and 21.3%, respectively. 9.6% of patients relapsed during followup, with 1-year overall survival, progression-free survival, and non-relapse mortality rates of 86.5% (95% CI 76.9% -96.1% ), 78.8% (95% CI 67.4% -90.3% ) and 11.5% (95% CI 2.6% -20.5% ), respectively. Conclusion: Ruxolitinib combined with a low dose of PTCY is a safe and effective first-line aGVHD prevention strategy.
目的： 评估芦可替尼联合低剂量后置环磷酰胺为基础的急性移植物抗宿主病（GVHD）预防方案在恶性血液病单倍体造血干细胞移植（haplo-HSCT）中的临床疗效和安全性。 方法： 收集2022年1月至12月在联勤保障部队第九二〇医院行haplo-HSCT患者，所有患者在预处理后予以减低剂量后置环磷酰胺、兔抗人胸腺细胞免疫球蛋白、芦可替尼、甲氨蝶呤及环孢素A、霉酚酸酯等预防急性GVHD，分析所有患者移植结局、并发症及生存情况。 结果： 共纳入52例haplo-HSCT患者，男29例（55.8%），女23例（44.2%），中位年龄为28（5～59）岁。急性髓系白血病25例，急性淋巴细胞白血病17例、骨髓增生异常综合征6例、慢性髓性白血病2例，骨髓增殖性肿瘤2例。98.1%的患者实现正常植入，其中Ⅱ～Ⅳ、Ⅲ/Ⅳ度急性GVHD发生率分别为19.2%（95% CI 8.2%～30.3%）、7.7%（95% CI 0.2%～15.2%）。未发生严重消化道黏膜炎。EB病毒、巨细胞病毒再激活率分别为40.4%、21.3%。9.6%的患者在随访期间复发，移植后1年总生存率、无进展生存率、非复发死亡率分别为86.5%（95% CI 76.9%～96.1%）、78.8%（95% CI 67.4%～90.3%）、11.5%（95% CI 2.6%～20.5%）。 结论： 在恶性血液病患者haplo-HSCT中，芦可替尼联合减低剂量后置环磷酰胺为基础的急性GVHD预防方案可有效控制急性GVHD且安全性良好。.
Keywords: Cyclophosphamide; Graft versus host disease; Haploidentical hematopoietic stem cell transplantation; Ruxolitinib.