对于患有阴性质疏松症的儿童的沃索里提德治疗:第二阶段临床试验
Andrew Dauber1,2, Anqing Zhang3, Roopa Kanakatti Shankar1,2
1Division of Endocrinology, Children's National Hospital, Washington, DC 20010, USA.
EClinicalMedicine
|May 30, 2024
概括
沃索里提德有效地增加了儿童的生长速度 带有下极性质,一种罕见的骨发育不良. 这种C型尿素类型的安全性和有效性与其在无形质形成症中的使用相似.
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
- 药理学 药理学是指药理学的学科.
背景情况:
- 缺色细胞增生症是一种罕见的自体主导骨发育不良症,由FGFR3变异引起,导致不成比例的矮身.
- 目前缺乏治疗低身高的儿童患有下极性质形成症的治疗方法.
- 沃索里提德是一种C型天然尿素类同类物,已被批准用于增长改善.
研究的目的:
- 评估在被诊断为患有下体质增生症的儿童中使用沃索里提德的安全性和有效性.
- 评估沃索里提德对生长速度和身高标准偏差评分 (SDS) 的影响.
主要方法:
- 一个单臂的,第二阶段的,开放的标签试验,涉及26名患有下极性质的儿童.
- 6个月的观察期,然后是12个月的干预期,每天用皮下伏索里提德 (15微克/公斤/天).
- 同主终点包括不良事件,年增长速度的变化和身高SDS.
主要成果:
- 24名参与者接受了沃索里提德;它被很好地容忍,没有严重的不良事件.
- 年增长速度增加了2.26 SD,身高SDS增加了0.36 SD.
- 绝对年化生长速度增加了1.81厘米/年,高高成形特异性高度SDS增加了0.38 SD.
结论:
- 伏索里提德是一种安全有效的治疗方法,用于增加患有下极性质形成症的儿童的生长速度.
- 观察到的疗效在低质粒细胞增生是类似于在无质粒细胞增生患者报告的疗效.
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