在使用真实世界的数据和药理动力学/药理动力学模型模拟的儿童中使用德斯摩素的剂量
Kevin Yen1, Emma Hughes2, Rada Savic2
1Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics, University of California, San Francisco, CA, USA. kevin.yen@ucsf.edu.
为患有AVP缺陷 (AVP-D) 的儿童优化德斯莫普林素 (ddAVP) 剂量至关重要. 建议为1-5岁的儿童提供50mcg的初始门诊剂量,以提高治疗效率并减少夜间排尿.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 药理动力学和药理动力学
- 罕见疾病治疗方法 罕见疾病治疗方法
背景情况:
- 对于阿尔金因血管压素缺乏症 (AVP-D) 的儿科德斯莫普林素 (ddAVP) 剂量显示出显著的变化.
- 目前对儿童的ddAVP的剂量建议是有限的.
- 优化ddAVP治疗方案对于有效治疗儿科患者AVP-D至关重要.
研究的目的:
- 评估和优化在被诊断为AVP缺陷 (AVP-D) 的儿童中使用德斯莫普林素 (ddAVP) 的剂量方案.
- 使用药理动力学和药理动力学 (PK/PD) 模拟来优化剂量.
- 为儿童AVP-D建立基于证据的剂量指南.
主要方法:
- 在ddAVP上对患有AVP-D的儿童 (<18岁) 电子健康记录的回顾性审查.
- 应用已验证的PK/PD模型来模拟ddAVP度和尿液输出.
- 对人口统计学因素 (年龄,体重) 的分析,以告知夜间尿道症的剂量优化.
主要成果:
- 分析了来自53名患有AVP-D的儿童的276份剂量记录.
- 模拟预测,在5岁以下的儿童中,将门诊ddAVP剂量增加到50mcg可以减少尿量.
- 这种剂量调整的目的是实现与保持干燥的人相似的尿液输出模式.
结论:
- 对于患有AVP-D的1-5岁儿童,建议初始门诊剂量至少为50微克.
- 建议使用这种剂量来提高ddAVP在治疗夜间尿尿症方面的疗效.
- 利用现实世界的临床数据可以增强治疗AVP-D等罕见疾病的药物剂量策略.
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