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在中低收入国家,使用低强度预处理方案评估门诊外周血造血干细胞移植方案的疗效。

Assessing the efficacy of an ambulatory peripheral blood hematopoietic stem cell transplant program using reduced intensity conditioning in a low-middle-income country.

机构信息

Universidad Autónoma de Nuevo León, Facultad de Medicina y Hospital Universitario Dr. José E. González, Department of Hematology, Internal Medicine Division, Monterrey, Mexico.

出版信息

Bone Marrow Transplant. 2019 Jun;54(6):828-838. doi: 10.1038/s41409-018-0338-7. Epub 2018 Sep 21.

Abstract

Efficacy of an ambulatory hematopoietic stem cell transplant (HSCT) program with a reduced intensity conditioning regimen (RIC) in malignant hematological diseases was assessed. We analyzed 217 patients who underwent HSCT from August 2013 to July 2017. There were 78 (35.9%) HLA-identical, 56 (25.8%) haploidentical, and 83 (38.2%) autologous transplants. Two-year transplant-related mortality (TRM) for HLA-identical, haploidentical, and auto grafts were 20%, 25%, and 2.5%; relapse/progression was 44%, 60%, and 55%; overall survival (OS) was 61%, 44.8%, and 78.0%; and disease-free survival (DFS) was 36.8%, 26.5%, and 43.5%, respectively. Factors associated with a high risk of TRM were male sex (HR = 2.62, P = 0.031), fever and neutropenia (HR = 3.30, P = 0.023), and cell dose < 5 × 10 CD34 +/kg (HR = 4.24, P = 0.001); cGVHD was a protective factor for TRM (HR = 0.29, P = 0.022). Transfusion was associated with increased risk of relapse/progression in univariate and multivariate analysis (HR = 3.10, P = 0.001 and HR = 3.30, P = 0.004); cGVHD was a protective factor (HR = 0.18, P = 0.001 and HR = 0.17, P = 0.002). In a multivariate analysis for allo-HSCT, infections were associated with high risk of mortality (HR = 3.90, P = 0.016) and transfusion with reduced DFS (HR = 2.76, P = 0.029); for haplo-HSCT, CD34 + < 5 × 10/kg was a risk factor for mortality and lower DFS (HR = 5.41, P = 0.001 and HR = 3.93, P = 0.001). Outcomes of our RIC-based outpatient transplant program are comparable to excellence centers in high-income countries.

摘要

评估了采用低强度预处理方案(RIC)的门诊造血干细胞移植(HSCT)方案在恶性血液病中的疗效。我们分析了 2013 年 8 月至 2017 年 7 月期间接受 HSCT 的 217 名患者。其中 78 例(35.9%)为 HLA 同型,56 例(25.8%)为半相合,83 例(38.2%)为自体移植。HLA 同型、半相合和自体移植的 2 年移植相关死亡率(TRM)分别为 20%、25%和 2.5%;复发/进展分别为 44%、60%和 55%;总生存率(OS)分别为 61%、44.8%和 78.0%;无病生存率(DFS)分别为 36.8%、26.5%和 43.5%。TRM 风险高的相关因素包括男性(HR=2.62,P=0.031)、发热和中性粒细胞减少(HR=3.30,P=0.023)以及细胞剂量<5×10 CD34+/kg(HR=4.24,P=0.001);cGVHD 是 TRM 的保护因素(HR=0.29,P=0.022)。输血与单变量和多变量分析中的复发/进展风险增加相关(HR=3.10,P=0.001 和 HR=3.30,P=0.004);cGVHD 是一个保护因素(HR=0.18,P=0.001 和 HR=0.17,P=0.002)。在异基因 HSCT 的多变量分析中,感染与高死亡率相关(HR=3.90,P=0.016),输血与较低的 DFS 相关(HR=2.76,P=0.029);在半相合 HSCT 中,CD34+<5×10/kg 是死亡率和较低 DFS 的危险因素(HR=5.41,P=0.001 和 HR=3.93,P=0.001)。我们基于 RIC 的门诊移植方案的结果与高收入国家的卓越中心相当。

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