制定针对安吉尔曼综合征的临床医生报告和护理人员报告的措施,以支持整体,以患者为中心的药物开发
Siobhan Connor-Ahmad1, Jorrit Tjeertes2, Michael Chladek3
1Roche Products Ltd., Welwyn Garden City, AL7 1TW, UK. siobhan.connor-ahmad@roche.com.
Orphanet journal of rare diseases
|June 22, 2023
概括
开发了新的临床医生和护理人员尺度,以评估安吉尔曼综合征 (AS) 症状和功能障碍. 这些工具旨在支持AS的以患者为中心的治疗方法的开发.
科学领域:
- 神经遗传学 神经遗传学
- 临床结果评估 临床结果评估
- 开发以患者为中心的治疗方法
背景情况:
- 安吉尔曼综合征 (AS) 是一种罕见,复杂的神经遗传疾病,影响个人和家庭.
- 验证的措施对于开发以患者为中心的AS疗法至关重要.
- 针对AS的全球印象尺度的开发遵循了FDA的指导,并得到了专家和护理人员的意见.
研究的目的:
- 为开发临床医生和护理人员报告的Angelman综合征全球印象尺度.
- 在AS临床试验中创建可靠的措施来评估关键症状和功能障碍.
- 确保措施是相关的,可以理解的,并捕捉AS的异质性.
主要方法:
- 从AS症状和影响的概念性疾病模型开发了最初的领域.
- 与临床医生,患者倡导者和护理人员进行了两轮认知报告采访.
- 根据对年龄适应性和清晰度的反,改进了尺度项目和措辞.
主要成果:
- 创建了AS-临床医师全球印象 (SAS-CGI) 和护理人员报告的AS尺度 (CASS) 的症状.
- 测量评估AS的发作,睡眠,行为,沟通,运动技能,认知和自我护理.
- 认知总结证实了尺度的相关性,清晰性和可理解性.
结论:
- SAS-CGI和CASS在1-12岁儿童中捕获了多种AS症状.
- 这些临床结果评估被纳入AS临床研究.
- 心理测量属性将被评估,以告知未来的改进.
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