Development and validation of TreatHSP-QoL – a patient-reported outcome measure for health-related quality of life in hereditary spastic paraplegia

Author:

Malina Jekaterina1ORCID,Huessler Eva-Maria2,Jöckel Karl-Heinz2,Boog-Whiteside Eva2,Jeschonneck Nicole1,Schröder Bernadette2,Schüle Rebecca3,Kühl Tobias2,Klebe Stephan1ORCID

Affiliation:

1. University Hospital Essen: Universitatsklinikum Essen

2. University of Duisburg-Essen: Universitat Duisburg-Essen

3. University Hospital Heidelberg: UniversitatsKlinikum Heidelberg

Abstract

Abstract Background. Hereditary spastic paraplegia (HSP) is a rare neurodegenerative disease that lacks specific and validated patient-centered outcome measures (PCOMs). We aimed to develop and validate a health-related quality of life (HRQoL) questionnaire specific to HSP (“TreatHSP-QoL”) that could be used as a PCOM. Results. The pilot-items of the TreatHSP-QoL (45 five-level Likert scale items, with values per item between 0–4) were developed based on a qualitative data analysis of 54 semi-structured interviews, conducted in person with 36 HSP patients and 18 caregivers. It was then reduced and modified through the validation process to 25 items. The main validation was performed using the online questionnaire in 242 HSP patients and 56 caregivers. The exploratory factor analysis defined five subdomains. Cronbach’s alpha ranged from 0.57–0.85 for the subdomains and reached 0.85 for the total score. The test-retest correlation reached 0.86. Correlations with the EuroQol-5 Dimension (5 levels) (EQ-5D-5L) and Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) questionnaires varied strongly among the subdomains, with the total scores reaching 0.53 and − 0.45, respectively. The caregiver-patient response correlation ranged between 0.64–0.82 for subdomains and reached 0.65 for the total score. Conclusions. TreatHSP-QoL can be used in high-quality clinical trials and clinical practice as a disease-specific PCOM (i.e., HRQoL measure) and is also applicable as a proxy questionnaire. Score values between 0-100 can be reached, where higher value represents better HRQoL. The variable correlations to the EQ-5D-5L and FARS-ADL support the specificity for HSP. All in all, the results demonstrate good validity and reliability for this new patient-centered questionnaire for HSP.

Publisher

Research Square Platform LLC

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3