在患有多发性硬化症的儿童中优化药物选择:我们知道什么,还有什么没有答案?
Rabporn Suntornlohanakul1,2, E Ann Yeh3,4,5
1Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, 555 University Avenue, Toronto, ON, M5G 1X8, Canada.
Paediatric drugs
|December 26, 2024
概括
儿科发病的多发性硬化症 (MS) 比成人发病的MS更具炎症性,影响认知能力. 使用高效疗法的早期治疗可能会改善患有多发性硬化症的儿童的治疗结果.
科学领域:
- 儿科神经学 儿科神经学
- 神经免疫学 神经免疫学
- 临床治疗学 临床治疗学
背景情况:
- 儿科发病多发性硬化症 (POMS) 与成人发病多发性硬化症 (AOMS) 相比,呈现出更炎症的过程和更大的残疾影响.
- 管理方面的挑战包括独特的儿科需求和对青少年的有限的批准疾病修饰疗法 (DMT).
- 目前的批准包括fingolimod (美国FDA) 和fingolimod, teriflunomide, dimethyl fumarate (EMA),许多药物被非标签使用.
研究的目的:
- 审查支持在POMS中使用DMT的文献,包括观察数据.
- 要突出临床转向早期使用高效疗法 (HETs) 在POMS的临床转变.
- 提出建议的治疗算法,并讨论儿童多发性硬化症护理的特殊考虑.
主要方法:
- 对POMS的DMT研究的综合文献综述.
- 对观察证据和临床实践趋势的分析.
- 综合数据,提出治疗算法和管理策略.
主要成果:
- 有证据表明,在POMS中早期启动HET与改善的认知和运动结果有关.
- 观察性研究支持儿童患者对各种DMT的非标签使用.
- 临床实践中明显出现了转向更早,更积极的治疗策略的转变.
结论:
- 个性化治疗方法对于POMS管理至关重要.
- 特别考虑的因素包括家庭动态,坚持和过渡到成人照顾.
- 进一步的研究对于优化MS儿童的长期结果至关重要.
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