肝脏糖原储存疾病的以个人为中心的结果:基于国际共识的标准结果集的开发
Ruben J Overduin1, Andrea B Haijer-Schreuder1, Frederiec K Withaar2
1Department of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands; UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, The Netherlands.
一个共识确定了14个肝脏糖原储存疾病 (GSD) 的关键健康结果,改善护理和临床试验. 这一标准集专注于以患者为中心的结果,以更好地管理GSD.
科学领域:
- 医学研究 医学研究
- 临床结果 临床结果
- 患者为中心的护理
背景情况:
- 肝脏糖原储存疾病 (GSD) 需要标准化的结果措施,以获得有效的医疗保健和临床试验.
- 目前的GSD管理缺乏基于共识的一套以人为中心的健康结果.
- 改善健康结果需要包括患者报告的结果措施.
研究的目的:
- 开发一种基于共识的,以人为中心的,肝 GSD 的健康结果标准.
- 确定肝 GSD 管理的关键健康结果和案例组合变量.
- 提高肝脏GSD患者的护理质量和临床试验设计.
主要方法:
- 一个国际性的,代性的名义上的共识过程,涉及到各种各样的利益相关者.
- 参与者包括患有GSD的个人,护理人员,医疗保健提供者,行业代表和基于价值的医疗保健专家.
- 数据收集涉及确定和优先考虑关键健康结果和案例组合变量.
主要成果:
- 为肝脏GSD测量建立了一套标准的14个推的健康结果.
- 关键结果包括治愈,GSD相关事件,血糖和代谢控制,并发症,生活质量和获得护理.
- 此外,还确定了29个案例混合变量的综合清单,以及测量频率的建议.
结论:
- 一套基于国际共识的,以人为中心的肝脏GSD健康结果标准已经成功开发出来.
- 该标准化套件旨在用于医疗机构,患者登记册和临床试验.
- 确定的结果旨在提高GSD研究和患者护理的一致性和相关性.
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