疾病修饰疗法和与治疗反应相关的特征在1型糖尿病预防中:系统性审查
Jamie L Felton1,2, Kurt J Griffin3,4, Richard A Oram5,6,7
1Department of Pediatrics, Center for Diabetes and Metabolic Diseases, Indianapolis, IN, USA.
Communications medicine
|October 4, 2023
概括
针对1型糖尿病 (T1D) 的精准医学需要更好的试验设计. 未来的研究需要统一的结果,生物标志物和预先规定的分析来指导T1D的预防和治疗.
科学领域:
- 免疫学 免疫学 免疫学
- 内分泌学 在内分泌学.
- 临床试验 临床试验
背景情况:
- 1型糖尿病 (T1D) 涉及对β细胞的免疫破坏.
- 目前的预防策略面临疾病异质性带来的挑战.
- 精准医学是有效修改T1D疾病所需的.
研究的目的:
- 系统地审查T1D疾病修饰的随机对照试验 (RCT).
- 分析干预措施并确定与治疗反应相关的特征.
- 评估T1D研究中精度分析的质量.
主要方法:
- 来自PubMed/Embase (过去25年) 的RCT (≥50名参与者) 的系统审查.
- 对T1D疾病修饰疗法和治疗反应预测指标的分析.
- 使用Cochrane偏差风险工具进行偏差评估.
主要成果:
- 审查了75份手稿;确定了11项预防试验和60项干预试验.
- 17项干预措施,主要是免疫疗法,比安慰剂更有好处.
- 精确分析往往缺乏预先规范,一致的报告,并倾向于报告积极的发现.
结论:
- 高质量的T1D预防/干预试验,但低质量的精确分析.
- 临床实践的有意义结论受到精确分析质量差的阻碍.
- 未来的试验需要统一的结果,生物标志物和预先规定的分析,用于T1D的精密医学.
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