在先天性上腺增生症中的葡萄糖皮质体处方趋势,2017年至2023年
A Roxas1, A Gihawi1,2, M Makarchuk1
1Norwich Medical School, University of East Anglia, Norwich, UK.
Endocrine connections
|March 5, 2026
概括
现代用于先天性上腺增生症 (CAH) 的葡萄糖皮质类药物的使用是有限的,只有5%的患者过渡. 主要障碍包括高成本,监管障碍和供应链问题,影响全球获得CAH治疗的机会.
科学领域:
- 内分泌学 在内分泌学.
- 药理学 药理学是指药理学的学科.
- 儿科内分泌学 儿科内分泌学
背景情况:
- 先天性上腺增生 (CAH) 是一组影响上腺的遗传性疾病.
- 21-基酶缺乏是最常见的CAH形式.
- 现代葡萄糖皮质激素药物比传统治疗方法具有潜在的治疗优势.
研究的目的:
- 研究CAH中现代葡萄糖皮质激素药物 (Acecort®,Alkindi®,Efmody®,Plenadren®) 的使用模式.
- 检查处方趋势,采用障碍以及影响其使用的地理和时间因素.
- 在全球队列中评估从传统葡萄糖皮质激素治疗到现代葡萄糖皮质激素治疗的过渡.
主要方法:
- 这是一项两部分研究,结合了国际先天性上腺增生注册表 (2017-2023) 的回顾性分析和处方中心的定性调查.
- 分析了20个国家的46个中心的治疗方案,处方趋势和障碍.
- 包括儿科和成人患者群体.
主要成果:
- 在2017-2023年期间,只有5%的患者 (790人中有44人) 转换为现代葡萄糖皮质类药物.
- 在高收入西欧国家,采用率最高.
- 观察到年龄特定的处方模式:阿尔金迪® <8年,Efmody® ≥7年. 到2023年,可用性会有所不同:阿尔金迪 (Alkindi®) (54%),埃弗莫迪 (Efmody®) (46%),普莱纳德伦 (Plenadren®) (33%),阿塞科特 (Acecort®) (15%).
结论:
- 在全球范围内,对CAH的现代葡萄皮质类药物的采用仍然非常有限.
- 主要障碍包括立法批准流程,供应链中断和高药费用.
- 在全球范围内,对先进的CAH治疗方法的不平等获得是显而易见的,需要制定战略来克服这些采用挑战.
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