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在正在进行的第3阶段扩展研究中,沃索里提德对患有无形质症的儿童的持续增长促进作用
Ravi Savarirayan1, Melita Irving2, William R Wilcox3
1Murdoch Children's Research Institute, Royal Children's Hospital Victoria, University of Melbourne, Parkville, VIC, Australia.
在患有无形质细胞增生症的儿童中,vosoritide治疗在六年内显示出持续的增长促进和有利的安全性. 与未经治疗的同龄人相比,这种C型尿素类类似物改善了身高增长和身体比例.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 骨发育不良症 骨发育不良症
- 药理干预药理干预
背景情况:
- 骨质疏松症的特点是骨生长减少,这是由于潜在的途径失调造成的.
- 伏索里提德是一种C型尿性类同类物,向这种途径.
- 长期治疗效果和与未接受治疗的儿童进行比较对于了解沃索里提德的疗效至关重要.
研究的目的:
- 为了评估长期治疗效果的vosoritide 在患有无质形成症的儿童.
- 为了比较vosoritide治疗儿童的成长结果与外部未经治疗的对照人群.
主要方法:
- 参与者完成了为期6个月的观察性研究和为期52周的双盲,安慰剂控制试验.
- 符合条件的参与者参加了开放式扩展,每天接受15μg/kg的vosoritide.
- 在CLARITY无形发育研究中,提供了外部未经处理的对照数据进行比较.
主要成果:
- 与未接受治疗的儿童相比,Vosoritide治疗在三年内增加了5.75厘米的额外身高.
- 在接受治疗的儿童中,观察到上下体段比率的显著改善.
- 沃索里提德在持续治疗长达六年的情况下表现出良好的安全性,没有观察到长期危害.
结论:
- 沃索里提德在患有无粒细胞增生症的儿童中耐受良好.
- 经过长达六年的沃索里提德治疗,观察到持续的促进生长的效果.
- 伏索里提德为改善无形质生长提供了一个有前途的治疗选择.
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