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文章:

美国食品药品监督管理局批准的用于治疗类固醇难治性慢性移植物抗宿主病的新型药物对治疗模式及预后的影响——一项单中心纵向队列分析

The Impact of FDA-Approved Novel Agents for Steroid-Refractory Chronic Graft vs. Host Disease on Treatment Patterns and Outcomes—A Single-Center Longitudinal Cohort Analysis

原文发布日期:17 October 2024

DOI: 10.3390/cancers16203521

类型: Article

开放获取: 是

 

英文摘要:

Objectives—chronic graft vs. host disease (cGVHD) is associated with substantial morbidity and mortality. We aimed to analyze advances in treatment strategy and outcomes during the last decade due to the incorporation of novel immunosuppressive therapy (IST) drugs in the armamentarium.Methods—we retrospectively analyzed all patients > 18 years with cGVHD after their first hematopoietic cell transplantation (HCT) between 2012 and 2020 (n = 91), divided into three treatment periods: 2012–2014, 2015–2017, and 2018–2020 (groups 1, 2, and 3, respectively).Results—mean cumulative steroid dose and dose/total cGVHD-treatment days was lower in groups 2–3 compared to 1 (p= 0.008 andp= 0.042, respectively). The median IST-free survival was 79 (95%CI54–94) months, with more patients in group 3 (47% (95%CI 25–54%) discontinuing IST at 3 years,p= 0.1). Groups 2–3 compared to 1 had better glycemic control (p< 0.01), higher bone density (p= 0.06), and fewer cardiovascular events. The number of admissions/patient dropped from 0.7/year in group 1 to 0.24/year and 0.36/year in groups 2–3, respectively (p= 0.36). Employment reintegration was higher in groups 2–3 compared with 1 (p= 0.05) and so was earlier return to work (p= 0.01). There were no differences in survival outcomes.Conclusions—the incorporation of novel agents appears to be associated with reduced overall steroid burden, improved cGVHD control, and fewer long-term side effects.

 

摘要翻译: 

目的——慢性移植物抗宿主病(cGVHD)与显著的发病率和死亡率相关。本研究旨在分析过去十年间,由于新型免疫抑制治疗(IST)药物纳入治疗方案后,治疗策略及临床结局的进展。方法——我们回顾性分析了2012年至2020年间首次接受造血细胞移植(HCT)后发生cGVHD的所有成年(>18岁)患者(n=91),并将其分为三个治疗时期:2012–2014年、2015–2017年和2018–2020年(分别为第1、2、3组)。结果——与第1组相比,第2–3组的平均累积类固醇剂量及剂量/总cGVHD治疗天数均较低(p值分别为0.008和0.042)。中位无IST生存期为79个月(95%CI 54–94),其中第3组有更多患者在3年内停用IST(47%,95%CI 25–54%,p=0.1)。与第1组相比,第2–3组的血糖控制更佳(p<0.01)、骨密度更高(p=0.06),且心血管事件更少。患者年均住院次数从第1组的0.7次/年分别降至第2–3组的0.24次/年和0.36次/年(p=0.36)。与第1组相比,第2–3组的职业重返率更高(p=0.05),且重返工作时间更早(p=0.01)。生存结局无显著差异。结论——新型药物的应用似乎与总体类固醇负荷降低、cGVHD控制改善以及长期副作用减少相关。

 

原文链接:

The Impact of FDA-Approved Novel Agents for Steroid-Refractory Chronic Graft vs. Host Disease on Treatment Patterns and Outcomes—A Single-Center Longitudinal Cohort Analysis

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