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美国食品药品监督管理局(FDA)批准的治疗类固醇难治性慢性移植物抗宿主病新型药物对治疗模式和结局的影响——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.

作者信息

Fridberg Gil, Amit Odelia, Karni Chen, Tshernichovsky Dina, Shasha David, Rouach Vanessa, Varssano David, Bar-Shai Amir, Goldberg Ilan, Wasserman Gilad, Avivi Irit, Ram Ron

机构信息

Bone Marrow Transplantation Unit, Tel Aviv Sourasky Medical Center, Tel Aviv 6423906, Israel.

Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv 6997801, Israel.

出版信息

Cancers (Basel). 2024 Oct 17;16(20):3521. doi: 10.3390/cancers16203521.

Abstract

-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. -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). -mean cumulative steroid dose and dose/total cGVHD-treatment days was lower in groups 2-3 compared to 1 ( = 0.008 and = 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, = 0.1). Groups 2-3 compared to 1 had better glycemic control ( < 0.01), higher bone density ( = 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 ( = 0.36). Employment reintegration was higher in groups 2-3 compared with 1 ( = 0.05) and so was earlier return to work ( = 0.01). There were no differences in survival outcomes. -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

-慢性移植物抗宿主病(cGVHD)与较高的发病率和死亡率相关。我们旨在分析过去十年中,由于在治疗手段中加入新型免疫抑制疗法(IST)药物,治疗策略和治疗结果取得的进展。

-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18岁以上患者(n = 91),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)。

-与第1组相比,第2 - 3组的平均累积类固醇剂量和剂量/总cGVHD治疗天数较低(分别为P = 0.008和P = 0.042)。无IST生存期的中位数为79(95%CI 54 - 94)个月,第3组中有更多患者(47%(95%CI 25 - 54%))在3年时停用IST(P = 0.1)。与第1组相比,第2 - 3组血糖控制更好(P < 0.01),骨密度更高(P = 0.06),心血管事件更少。每位患者的住院次数从第1组的每年0.7次降至第2 - 3组的每年0.24次和0.36次(P = 0.36)。与第1组相比,第2 - 3组的就业再融入情况更好(P = 0.05),重返工作岗位也更早(P = 0.01)。生存结果无差异。

-新型药物的加入似乎与总体类固醇负担减轻、cGVHD控制改善以及长期副作用减少有关。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d832/11506359/ef500b7e2c86/cancers-16-03521-g001.jpg

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