五类儿童疾病人群自答与代答健康效用值的差异

Difference of Health Utility Values Between Self-reported and Proxy-reported Assessment of Childhood Population with Five Categories of Diseases

目的系统比较对我国儿童健康威胁较大的5类疾病,其患者自答与代答健康效用值间的差异,为相关药物经济学评价提供参考。方法检索中国期刊全文数据库、中文期刊全文数据库、万方数据知识服务平台、PubMed、Web of science、Embase和Cochrane Library数据库,搜集同时报道儿童患者或未患病儿童的自答和代答健康效用值的文献,检索时限截止至2019年12月31日。并选取加权均值差为效应尺度,按照疾病类型,进而对应用不同健康测量工具以及不同代答者测量的儿童自答和代答效用值进行Meta分析。结果共纳入16篇文献。Meta分析结果显示,在疾病类型方面,未患病儿童(WMD=-0.034,95%CI:-0.047~-0.021)和极低体重儿(WMD=-0.023,95%CI:-0.044~-0.001)自答结果显著低于代答;哮喘(WMD=0.034,95%CI:0.029~0.039)、慢性肺部疾病(WMD=0.030,95%CI:0.003~0.057)和脊柱裂患儿(WMD=0.044,95%CI:0.010~0.078)自答结果显著高于代答。不同健康测量工具以及不同代答者的分析结果显示,在代答健康测量工具应用健康效用量表2(HUI2)或欧洲生命质量五维量表(EQ-5D)时,肿瘤患儿代答者类型选择父母时,自答和代答结果差异无统计学意义(P>0.05)。结论儿童患者自答和代答效用值间存在差异,具体区分疾病类型、健康效用测量工具和代答者类型时,差异的大小和方向有所不同。儿童健康相关生命质量或药物经济学评价研究者应优先选择儿童自答健康效用值,或自答和代答结果间无显著性差异的代答效用值。

Objective To systematically compare the differences between self-reported and proxy-reported health utility values with five categories of diseases that pose a great threat to children’s health in China,and to provide reference for related pharmacoeconomic evaluations.Methods CNKI,VIP,Wanfang Data,PubMed,Web of science,Embase and Cochrane Library databases were searched to collect literature that simultaneously reported the health utility values of self-reported and proxy-reported of child patients or the general population,and the retrieval period was up to December 31,2019.Meta-analysis according to diseases was performed and the weighted mean difference was selected as the effect size.Subgroup analysis was performed according to measuring instruments and proxy types.Results A total of 16 articles were included.The results of Meta analyses showed that in terms of disease types,the self-reported values of children with very low birth weight(WMD=-0.023,95%CI:-0.044--0.001)and general children population(WMD=-0.034,95%CI:-0.047--0.021)were significantly lower than those of proxy-reports.Self-reported of children with asthma(WMD=0.034,95%CI:0.029-0.039),chronic lung disease(WMD=0.030,95%CI:0.003-0.057)and spina bifida(WMD=0.044,95%CI:0.010-0.078)had significantly higher self-reported utility values than those of proxy-reports.The results of subgroup analyses showed that when selecting HUI2 or EQ-5 D as the instruments of proxy-reports,and selecting parents to report for children with tumor,there were no significant difference between self and proxy reports(P>0.05).Conclusion Overally,there are differences between the results of self-reports and proxy-reports of child patients,however,the size and direction of the differences were varied when differentiating between the diseases,the measuring instruments and the proxy types.Researchers of child health-related quality of life or pharmacoeconomic evaluation should give priority to selecting children’s self-reported health utility value,or a proxy utility value with no significant difference between self-reported and proxy results.