苯丙酮尿症患儿治疗依从性现状及其相关因素分析

Treatment adherence and associated factors among children with phenylketonuria

摘要

目的 分析苯丙酮尿症(PKU)患儿治疗依从性情况及社会经济因素、个体特征和治疗行为与依从性的相关关系及关联路径,为改善其预后提供参考依据。方法 本研究为回顾性研究。选取2016年4月1日—2023年12月31日在山西省儿童医院(山西省妇幼保健院)新生儿疾病筛查中心确诊并按要求坚持随访至2024年12月31日的169例PKU患儿为研究对象,按照是否遵循治疗规范[近1年血苯丙氨酸(Phe)检测次数≥6次]且Phe水平在目标区间的比例≥70%,将患儿分为依从性好组(n=78)与依从性差组(n=91)。采用单因素分析和多因素logistic回归分析筛选与依从性相关的因素,运用结构方程模型(SEM)进行路径分析。结果 母亲大专及以上文化(OR=2.492,95%CI:1.133～5.477)、居住地为城市(OR=2.625,95%CI:1.272～5.418)、家庭月收入4 000～8 000元(OR=2.245,95%CI:1.018～4.925)和＞8 000元(OR=4.055,95%CI:1.070～15.370)与PKU患儿依从性好呈正相关(P<0.05);患儿年龄越大(OR=0.456,95%CI:0.240～0.871)、开始饮食治疗时间越晚(OR=0.958,95%CI:0.930～0.987),PKU患儿依从性好的可能性越低(P＜0.05)。SEM表明社会经济因素、开始饮食治疗时间与依从性直接相关,标化路径系数分别为0.614、-0.186;社会经济因素又通过开始饮食治疗时间与依从性呈间接相关,标化路径系数为0.044。结论 家庭社会经济水平与PKU患儿依从性直接相关,又通过开始饮食治疗时间与依从性呈间接相关,可据此为改善患儿依从性制定具有针对性的策略提供线索。

Abstract

Objective To assess treatment adherence among children with phenylketonuria (PKU), and to examine the associations and pathways linking socioeconomic factors, individual characteristics, and treatment-related behaviors with adherence, so as to provide evidence for improving PKU outcomes. Methods This retrospective study included 169 children with PKU who were diagnosed at the Newborn Screening Center of Shanxi Children′s Hospital (Shanxi Maternal and Child Health Care Hospital) between April 1, 2016, and December 31, 2023, and who remained in follow-up through December 31, 2024.Good treatment adherence was defined as meeting both of the following criteria: at least six blood phenylalanine (Phe) measurements in the past year and Phe concentrations within the target range in ≥70% of measurements.The participants were classified into good-adherence (n=78) and poor-adherence (n=91) groups.Univariate and multivariable logistic regression analyses were used to identify factors associated with treatment adherence, and structural equation modeling (SEM) was used for path analysis. Results Maternal education of junior college level or higher (OR=2.492, 95%CI: 1.133 - 5.477), urban residence (OR=2.625, 95%CI: 1.272 - 5.418), and a monthly household income of CNY 4 000 - 8 000 (OR=2.245, 95%CI: 1.018 - 4.925) or >CNY 8 000 (OR=4.055, 95%CI: 1.070 - 15.370) were associated with higher odds of good treatment adherence(P<0.05).Older age (OR=0.456, 95%CI: 0.240 - 0.871) and a longer interval before initiation of dietary treatment (OR=0.958, 95%CI: 0.930 - 0.987) were associated with lower odds of good treatment adherence (P<0.05).SEM showed that socioeconomic factors and time to initiation of dietary treatment were directly associated with treatment adherence, with standardized path coefficients of 0.614 and -0.186, respectively.Socioeconomic factors were also indirectly associated with treatment adherence through time to initiation of dietary treatment (standardized path coefficient=0.044). Conclusions Family socioeconomic status is directly associated with treatment adherence in children with PKU and is also indirectly associated with adherence through the timing of dietary treatment initiation.The results may help guide targeted interventions to improve treatment adherence.

关键词

苯丙酮尿症 / 治疗依从性 / 结构方程模型 / 儿童

Key words

phenylketonuria / treatment adherence / structural equation modeling / children

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基金资助

山西省儿童医院(山西省妇幼保健院)院级基金项目(ZXKTGIIT-2025018)