基于中文版儿童健康相关生活质量量表评价Alport综合征患儿生活质量的病例对照研究

Health-related quality of life in children with Alport syndrome assessed by the Chinese version of Pediatric Quality of Life InventoryTM 4.0 Generic Core Scales:A case control study

目的了解Alport综合征(AS)儿童健康相关生活质量(HRQoL)水平.方法病例对照研究,AS为病例组(来源于2018年第六届中国AS家长联谊会的AS患儿),原发肾病综合征为对照组(北京大学第一医院2017年10月至2018年2月儿科肾脏病房住院的原发性肾病综合征患儿),以自编调查问卷收集研究对象的一般资料,以中文版PedsQL 4.0普适性核心量表为评价生活质量的工具,由经过培训的专人结合纸质版问卷填写说明以一致的话语指导量表填写.结果符合本文纳入和排除标准的61例AS和30例原发肾病综合征患儿进入分析,两组在病程、蛋白尿程度、近1个月病情、住院次数、家庭平均月收入和疾病花费对家庭的影响方面差异有统计学意义.回收AS和原发肾病综合征患儿自评量表54份和27份,对应的家长报告量表分别为61份和30份.不论AS还是原发肾病综合征,患儿自评量表总分及4个维度得分与对应的家长报告得分差异无统计学意义.AS患儿自评量表总分及各维度的得分与原发肾病综合征患儿自评量表的相应得分的差异无统计学意义,AS家长报告量表总分、生理功能、情感功能及角色功能维度得分与原发肾病综合征家长报告量的相应得分差异无统计学意义,但在量表社会功能维度得分AS低于原发肾病综合征,差异有统计学意义.结论基于中文版PedsQL 4.0普适性核心量表测得的AS儿童HRQoL受损并不比原发肾病综合征儿童明显.

Objective To assess the health-related quality of life(HRQoL)of children with Alport syndrome.Methods In this case control study,the cases included children with Alport syndrome who attended the 6th Alport family meeting in China held in 2018,and the controls included children with primary nephrotic syndrome who were admitted to Division of Nephrology,Department of Pediatrics,Peking University First Hospital between October 2017 and February 2018.The demographic,clinical and social data were collected using a self-designed questionnaire.The Chinese version of Pediatric Quality of Life InventoryTM(PedsQLTM)4.0 Generic Core Scales were used to assess HRQoL in the participants.Parallel child self-report(5-18 years)and parent proxy-report(2-18 years)forms was included.The trained research assistants guided the questionnaire filling by the same explanatory words and instructions.Results A total of 91 families(61 families with Alport syndrome and 30 families with primary nephrotic syndrome)were recruited in the study.There were significant differences in the duration of disease,the level of proteinuria,the condition in the last one month,hospitalization frequency,family monthly average income and the impact of disease cost on family between children with Alport syndrome and primary nephrotic syndrome.A total of 81 child self-report forms(54 from Alport syndrome and 27 from primary nephrotic syndrome)and 91 parent proxy-report forms(61 from Alport syndrome and 30 from primary nephrotic syndrome)were collected.There were no significant differences in overall scales and four dimensions between the child-self and parent-proxy reports for both Alport syndrome and primary nephrotic syndrome.Compared with children affected by primary nephrotic syndrome and their parents,no significant differences were observed in overall scales and four subscales of the child-self reports from Alport syndrome,whereas parents of children with Alport syndrome had lower scores in the social subscale.Conclusion Compared with primary nephrotic syndrome,HRQoL of Alport syndrome was not worse in the assessment by the Chinese version of PedsQLTM 4.0 Generic Core Scales.