Rofail Diana, Froggatt Daniel, de la Torre Rafael, Edgin Jamie, Kishnani Priya, Touraine Renaud, Whitwham Sarah, Squassante Lisa, Khwaja Omar, D'Ardhuy Xavier Liogier
Roche Products Limited, Welwyn Garden City, UK.
Hospital del Mar Medical Research Institute (IMIM), 08003, Barcelona, Spain.
Adv Ther. 2017 Aug;34(8):2058-2069. doi: 10.1007/s12325-017-0591-y. Epub 2017 Aug 9.
To date, there is little research on health-related quality of life (HRQoL) in Down syndrome (DS), and existing research is variable with regard to reported HRQoL in DS. There are also no HRQoL measures developed specifically to be used with individuals with Down syndrome.
A multi-national, longitudinal, 24-week non-interventional study was conducted in adolescents and adults with DS. HRQoL was assessed (n = 90) using the parent-report KIDSCREEN-27 questionnaire.
HRQoL domain scores were found to be similar to those in the KIDSCREEN-27 European normative group data set on the Physical Well-being, Psychological Well-being, Autonomy and Parent Relations domains. Compared with the normative data set, the adolescent participants with DS in the current study were found to have lower scores on the Social Support and Peers domain and higher scores than the normative group on the School Environment domain. The test-retest reliability of the KIDSCREEN-27 was also examined using the intraclass correlation coefficient (ICC) in a subgroup of stable participants. The KIDSCREEN-27 demonstrated poor-to-moderate test-retest reliability; however, test-retest reliability was assessed using a long time interval between assessment time points.
The findings of this study underline that further research is needed to better understand the nature of HRQoL in DS. Further research using a shorter time interval between assessment time points to examine test-retest reliability is also required.
F. Hoffmann-La Roche Ltd.
迄今为止，关于唐氏综合征（DS）患者健康相关生活质量（HRQoL）的研究较少，且现有研究中关于DS患者报告的HRQoL存在差异。也没有专门为唐氏综合征患者开发的HRQoL测量方法。
对患有DS的青少年和成人进行了一项为期24周的多国家纵向非干预性研究。使用家长报告的儿童生活质量量表-27（KIDSCREEN-27）问卷评估HRQoL（n = 90）。
发现HRQoL领域得分与儿童生活质量量表-27欧洲常模数据集在身体健康、心理健康、自主性和亲子关系领域的得分相似。与常模数据集相比，本研究中患有DS的青少年参与者在社会支持和同伴领域的得分较低，而在学校环境领域的得分高于常模组。还在一组稳定参与者中使用组内相关系数（ICC）检查了儿童生活质量量表-27的重测信度。儿童生活质量量表-27显示出较差到中等的重测信度；然而，重测信度是在评估时间点之间使用较长时间间隔进行评估的。
本研究结果强调，需要进一步研究以更好地了解DS患者HRQoL的本质。还需要进一步研究，在评估时间点之间使用更短的时间间隔来检查重测信度。
F. Hoffmann-La Roche Ltd.
