高效疗法作为儿科发病多发性硬化症的第一线治疗方法
Nail Benallegue1,2, Fabien Rollot3,4,5,6, Sandrine Wiertlewski2,7
1Department of Paediatric Neurology, Universitaire Angers, CHU Angers, Angers, France.
JAMA neurology
|February 12, 2024
概括
高效疗法 (HETs) 与中度有效疗法 (METs) 相比,显著减少儿科发病多发性硬化症 (POMS) 的复发. 最初的HET提供了更好的结果和耐受性,这表明它们应该优先考虑患有POMS的儿童.
科学领域:
- 神经学 神经学
- 儿科 儿科 儿科
- 免疫学 免疫学 免疫学
背景情况:
- 儿科发病多发性硬化症 (POMS) 治疗策略缺乏共识,历史上使用过适度有效的疗法 (MET).
- 高效疗法 (HETs) 的使用不断扩大,需要评估它们在POMS中的实际有效性.
研究的目的:
- 为了比较HET作为初始治疗与MET在POMS中降低疾病活性方面的现实世界协会.
- 评估初始治疗选择对复发率,MRI活动,残疾进展和治疗耐受性的影响.
主要方法:
- 对于530名曾经接受过治疗但患有复发性缓解性POMS的儿童进行了回顾性队列研究,这些儿童来自法国复发性结核病观察所 (OFSEP) 队列 (2010-2022年).
- 初始HET与MET的比较,分析到第一次复发的时间,年复发率 (ARR),MRI活动和扩展残疾状况尺度 (EDSS) 的进展.
- 以共变量调整的统计建模,考虑非线性和相互作用.
主要成果:
- 无论是HET还是MET,都能在2年内降低第一次复发的风险.
- 在5年内,HET显示首次复发风险降低了54%,MRI活动减少.
- HET与更好的耐受性相关,并且由于缺乏疗效或不耐受性而导致治疗中止的风险明显降低 (MET高6倍).
结论:
- 与MET相比,POMS的初始HET与复发风险降低,更低的治疗转换率和更好的中期耐受性有关.
- 根据这些发现,建议优先考虑POMS儿童的初始HET.
- 仍需要进行长期安全性研究,以充分了解POMS初始HET使用的影响.
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