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实际世界托西利祖马布在儿科住院患者中的使用
Gideon A Stitt1, Mark W Hall2, Mary Ann Diliberto3
1Division of Clinical Pharmacology (GAS), Department of Pediatrics, University of Utah, Salt Lake City, UT.
概括
托西利祖马布 (TCZ) 用于治疗各种儿科疾病,常见的是透氨炎. 身体外支持和瘤诊断与死亡率有关,但TCZ使用期间的活跃感染并非如此.
科学领域:
- 儿科风湿病学 儿科风湿病学
- 儿科血液瘤学
- 儿科重症监护中心儿童重症监护中心
背景情况:
- 托西利祖马布 (TCZ) 是一种用于各种儿科炎症疾病的干白素-6受体抑制剂.
- 有限的数据存在于TCZ利用模式,安全性和住院儿科患者的结果.
研究的目的:
- 量化儿童住院患者中TCZ的使用情况.
- 在这个人群中探索与TCZ管理相关的安全性和临床结果.
主要方法:
- 从2016年1月至2021年5月接受静脉TCZ的儿科住院患者的回顾性队列研究.
- 数据收集包括人口统计,适应症,剂量,安全事件 (TCZ后0-7天),体外支持 (ES),并发感染和生存.
- 探索性分析确定了与死亡率相关的因素.
主要成果:
- 分析了103个TCZ疗程 (87名患者);中位数年龄为14岁.
- 常见的征兆:细胞因子释放综合征 (CRS; 56%),自身免疫性疾病 (27%),移植与宿主疾病 (GVHD; 5%),COVID-19 (4%).
- 膜炎 (ALT/AST>3x ULN) 在53%-60%的疗程中发生. 释放后的生存率为83%. ES,瘤诊断和TCZ后感染与死亡率有关.
结论:
- TCZ用于各种儿科住院病症,主要是CRS.
- 新发的透视膜炎在TCZ后是常见的. 死亡风险因素可能受到潜在疾病严重程度的影响.
- 在活跃感染期间给予TCZ并没有独立地与增加的死亡率有关.
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