长期安全性和对接受西洛斯治疗的患者生长的影响:综合分析
Yang-Yang Wang1, Li-Ping Zou2,3, Kai-Feng Xu4
1Department of Pediatrics, The First Medical Center of PLA General Hospital, Chinese PLA General Hospital, Beijing, 100853, China.
Orphanet journal of rare diseases
|August 15, 2024
概括
长期使用西洛对于儿童的生长和器官功能是安全的,尽管更高的剂量可能会影响生长指标. 诸如口腔炎和高脂血症等不良事件是可以管理的.
科学领域:
- 药理学 药理学是指药理学的学科.
- 儿科医学 儿科医学
- 在瘤学瘤学.
背景情况:
- 赛洛斯向mTOR通路,用于相关疾病.
- 长期安全性数据,特别是在儿科,是有限的.
- 这项研究针对不同年龄组的西罗斯的长期安全性.
研究的目的:
- 评估西洛斯的长期安全性.
- 具体评估其对儿科生长模式的影响.
- 分析对造血,肝脏和功能以及脂质水平的影响.
主要方法:
- 两项前性队列研究 (10年) 的综合分析.
- 1,738名参与者 (5天至69岁) 患有TSC和/或LAM.
- 雪洛斯1 mg/m2/天,剂量根据最低水平 (5-10 ng/ml) 进行调整.
主要成果:
- 没有对儿科生长,器官功能或脂质产生显著的不良影响.
- 更高的剂量显示出增加身高,体重和BMI Z-分数的趋势.
- 常见的不良事件:口腔炎 (52.9%),短暂的细胞衰竭,高脂血症 (第一年).
- 在48.5%的成年女性中发现月经障碍.
结论:
- 长期使用西洛对于儿童的生长和器官功能是安全的.
- 对增长的潜在剂量依赖性影响需要进一步研究.
- 对口腔炎等不良事件的管理策略至关重要.
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