苯丙酮尿症患者报告结局系统评价

Phenylketonuria Patients Report Outcome:A Systematic Review

目的系统评价苯丙酮尿症(phenylketonuria,PKU)的患者报告结局(PRO)。方法系统检索PubMed、EMBASE、The Cochrane Library、Web of Science、Scopus和EBSCO英文数据库以及中国期刊全文数据库发表的有关PKU患者的PRO研究。结果初检确定1680篇相关文献,手工检索确定6篇,经去重、审查标题和全文后,最终纳入32篇。共30种PRO评估工具,其中适用于成年患者25种,未成年患者6种。评估疾病症状量表17种,患者报告抑郁、焦虑情绪较多,评估健康相关生命质量的量表13种,其中PKU-QOL最为常用。结论目前用于PKU患者的PRO量表多是国外量表,中文版本的量表较少,限制了在国内的应用。

Objective To systematically evaluate the reported outcomes of patients with phenylketonuria(PKU).Methods A systematic search was made on PubMed,EMBASE,The Cochrane Library,Web of Science,Scopus and Ebsco English databases,and the Chinese Journal Full-text Database(CNKI) published studies on PKU patient reporting outcomes.Results 1 680 related literatures were identified through preliminary examination,and 6 were identified through manual search.After de duplication,title and full text review,32 were finally included.There were a total of 30 PRO evaluation tools,including 25 for adult patients and 6for juvenile patients.There were 17 scales for evaluating disease symptoms.Patients reported more depression and anxiety.There were 13 scales for evaluating health-related quality of life.Among them,the Phenylketonuria-quality of life(PKU-QOL) scale was the most commonly used.Conclusion The scales currently used to report outcomes for PKU patients are basically foreign scales.The Chinese version of the scales are less,which limit their application in China.