与使用C型尿素类同类物相关的高三症:一个病例报告
Juan Llerena Clinton1, Tatiana Sa Pacheco Carneiro Magalhaes2, Ana Paula Bordallo3
1Departamento de Genética Médica, Instituto Fernandes Figueira, Fundação Oswaldo Cruz, Rio de Janeiro, Brazil.
一种新的副作用,高三症,观察到在治疗 vosoritide 的 achondroplasia 患者. 这种以过度毛发生长为特征的疾病在停止治疗后会消失,不被认为是严重的.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
背景情况:
- 无形质是最常见的骨发育不良症.
- 伏索里提德是一种C型天然尿素类同类物,已被批准用于增强无色多发症患者的生长.
- 超三症是一种新发现的与索治疗相关的不良事件.
研究的目的:
- 报告一种新的不良事件,高三症,在接受vosoritide的无色多发症患者中.
- 描述vosoritide诱导的高三的临床特征和解决方案.
- 为了告知临床决策和患者咨询关于vosoritide治疗.
主要方法:
- 一个由来自巴西的18名无骨质疏松症患者 (年龄在2-10岁) 治疗vosoritide的病例系列.
- 儿科内分泌学家对高三症的临床诊断.
- 监测病毒化迹象,骨老化或雄激素水平升高的迹象.
主要成果:
- 在18名患者中,有11名患者 (61.1%) 在接受了至少3个月的沃索里提德治疗后出现过高三症.
- 超三症呈现为脸上,手臂,腹部,背部和腿部的有色素,薄,短发.
- 没有患者表现出病毒化或激素异常的迹象;在停止治疗后,高三症得到缓解.
结论:
- 超三症是一种意想不到的,非严重的不良事件与vosoritide 相关的 achondroplasia 患者.
- 这一发现需要在开始vosoritide治疗之前与家人讨论.
- 需要进一步的现实世界数据来理解病理生理学和指导治疗决策.
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