The CrescNet Registry Achondroplasia Module:现实世界的人口统计数据和未接受治疗和接受索治疗的个体的临床结果
Hormone research in paediatrics
|January 30, 2026
概括
沃索里提德治疗在3年内显著改善了儿童的身高. 克雷斯克网注册模块有效地收集有关这种罕见骨发育不良的现实数据.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 罕见疾病 罕见疾病
- 骨发育不良症 骨发育不良症
背景情况:
- 骨质疏松症是一种罕见的遗传性疾病,导致严重的不成比例的矮身.
- 伏索里提德是唯一被批准用于治疗无形质症的疗法.
- CrescNet注册表于2021年建立了一个专门的模块,以收集Achondroplasia数据.
研究的目的:
- 描述在CrescNet注册表中建立的特定的Achondroplasia模块的设置.
- 报告在3年内对患有无形质细胞增生症的儿童进行索治疗的初步现实结果.
主要方法:
- 该模块在CrescNet网络中的10个欧洲国家实施.
- 收集的数据包括人体测量,发育里程碑,干预措施 (vosoritide,手术),并发症和生活质量.
- 伪名化数据根据年龄和治疗状况进行了集中分析.
主要成果:
- 在32个中心注册了486名参与者; 186人接受了vosoritide.
- 接受伏索里提德治疗的个体在1,2,3年 (P≤0.0001) 的高度标准偏差得分显著增加.
- 实际数据中的增长结果与临床试验结果一致.
结论:
- 在CrescNet模块有效地收集现实世界的数据,以了解骨质疏松症和治疗结果.
- 沃索里提德在现实环境中在改善生长方面表现出一致的有效性.
- 持续的跟踪将为对vosoritide的影响提供更长期的见解.
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