Mpundu-Kaambwa Christine, Chen Gang, Huynh Elisabeth, Russo Remo, Ratcliffe Julie
Institute for Choice, University of South Australia Business School, University of South Australia, Adelaide, Australia.
Centre for Health Economics, Monash Business School, Monash University, Melbourne, Australia.
BMJ Open. 2017 Sep 11;7(9):e015924. doi: 10.1136/bmjopen-2017-015924.
Cerebral palsy is the most common cause of physical disability in children and adolescents and is associated with impairments that may reduce the quality of life (QOL) of this population. Patient-reported outcome measures (PROMs) can facilitate the assessment of the effect of disease and treatment on QOL, from a patient viewpoint. The purpose of this systematic review is to identify PROMs that are used to measure QOL and subjective well-being (SWB) outcomes in young people with cerebral palsy and to evaluate the suitability of these PROMs for application in economic evaluations within this population.
MEDLINE, Scopus, the Cochrane Library, Web of Science Core Collection, EconLit, PsycINFO, CINAHL, EMBASE and Informit will be systematically searched from inception to date of search. Published peer-reviewed, English-language articles reporting PROMs measuring QOL or SWB outcomes in children and adolescents with cerebral palsy will be included. One reviewer will conduct the initial search and screen titles and abstracts for potentially eligible studies. The search will be performed in November 2017. To reduce the likelihood of reviewer selection bias, two other reviewers will independently screen a randomly selected subsample (10%) of the citations. Two reviewers will then retrieve full texts of potentially eligible studies and assess them against predefined inclusion criteria. The suitability of selected PROMs for use in economic evaluations of young people with cerebral palsy will be assessed using the International Society of Quality of Life Research recommended Minimum Standards and the Patient-Centered Outcomes and Comparative Effectiveness Research checklist. A narrative synthesis of extracted data will be presented including study descriptive data, PROMs measurement properties, settings in which they were applied and the valuation methods. Recommendations for practice on the selection of PROMs for use in economic evaluations of children and adolescents with cerebral palsy will be presented.
Ethical approval is not required as the proposed systematic review will not use primary data. The results of this study will be widely disseminated through publication in a peer-reviewed journal and conference presentation(s).
International Prospective Register of Systematic Reviews number: CRD42016049746.
脑瘫是儿童和青少年身体残疾的最常见原因，且与可能降低该人群生活质量（QOL）的损伤相关。患者报告结局测量（PROMs）能够从患者角度促进对疾病及治疗对生活质量影响的评估。本系统评价的目的是识别用于测量脑瘫青少年生活质量和主观幸福感（SWB）结局的PROMs，并评估这些PROMs在该人群经济评价中的适用性。
将从创刊至检索日期对MEDLINE、Scopus、Cochrane图书馆、科学引文索引核心合集、EconLit、PsycINFO、护理学与健康领域数据库、EMBASE和Informit进行系统检索。纳入已发表的、经过同行评审的、报告测量脑瘫儿童和青少年生活质量或主观幸福感结局的PROMs的英文文章。一名评审员将进行初始检索，并筛选标题和摘要以查找潜在符合条件的研究。检索将于2017年11月进行。为降低评审员选择偏倚的可能性，另外两名评审员将独立筛选随机抽取的10%的引文子样本。然后，两名评审员将检索潜在符合条件研究的全文，并根据预定义的纳入标准对其进行评估。将使用国际生活质量研究协会推荐的最低标准以及以患者为中心的结局和比较效果研究清单来评估所选PROMs在脑瘫青少年经济评价中的适用性。将对提取的数据进行叙述性综合，包括研究描述性数据、PROMs测量属性、应用环境及评估方法。将给出关于选择用于脑瘫儿童和青少年经济评价的PROMs的实践建议。
由于拟进行的系统评价不使用原始数据，因此无需伦理批准。本研究结果将通过在同行评审期刊上发表及会议报告进行广泛传播。
国际前瞻性系统评价注册编号：CRD42016049746。
