儿童和青少年威尔逊病的药物治疗依从性：来自土耳其的队列研究。
Adherence to medical treatment for Wilson's disease in children and adolescents: a cohort study from Turkey.
机构信息
Division of Pediatric Gastroenterology, Medical Park Göztepe Hospital, Bahçeşehir University, İstanbul, Turkey.
Department of Clinical Pharmacy, Faculty of Pharmacy, Hacettepe University, Ankara, Turkey.
出版信息
Orphanet J Rare Dis. 2024 Mar 7;19(1):105. doi: 10.1186/s13023-024-03113-0.
BACKGROUND
This study aimed to assess medication adherence and demographic, clinical, and psychopathological parameters such as quality of life, depression, and anxiety levels that can affect pediatrics with Wilson's Disease (WD).
METHODS
A prospective cohort study was conducted at an outpatient clinic in Turkey among pediatric patients (2 to 18 years) with WD between November 2022 and April 2023. The Medication Adherence Report Scale (MARS-5) as a subjective and Medication Possession Ratio (MPR) as an objective assessment were scored. Physical, genetic and biochemical parameters, the Pediatric Quality of Life Inventory (PedsQL) for both parents and patients, Childhood Depression Inventory, State Trait Anxiety Inventory were also administered.
RESULTS
A total of 30 pediatric outpatients who were prescribed D-penicillamine (n = 27) or trientine (n = 3) as chelators and zinc (n = 29) and pyridoxine (n = 19) as supplements were included. Proteinuria (n = 3), skin rash (n = 2), and gastrointestinal upset (n = 2) were observed. When the correlation between MARS-5 and duration of follow-up was examined, a significant negative correlation was found (p = 0.014). According to MPRs, non-adherence rates (missed doses ≥ 20%) were 29.6%, 17.2% and 5.3% for D-penicillamine, zinc and pyridoxine, respectively. PedsQL scores were higher than those of parents, with a positive correlation between them (p < 0.001). Also, there was a significant positive correlation between PedsQL and State Anxiety Inventory (p < 0.001). Comparing the change in urinary copper levels between different levels of treatment knowledge, significant differences were observed between high- and low levels (p = 0.043).
CONCLUSIONS
Overall, nonadherence rates were 23.3% based on MARS-5 and 5.3-29.6% based on MPR. It is essential to consider factors such as the duration of follow-up, biochemical parameters, treatment knowledge, quality of life and anxiety as potential influencers of medication adherence.
背景
本研究旨在评估影响威尔逊病（WD）儿科患者药物依从性的人口统计学、临床和精神病理学参数，如生活质量、抑郁和焦虑水平。
方法
2022 年 11 月至 2023 年 4 月，在土耳其的一家门诊诊所对 2 至 18 岁的 WD 儿科患者进行了一项前瞻性队列研究。使用药物依从性报告量表（MARS-5）进行主观评估，使用药物持有率（MPR）进行客观评估。还评估了身体、遗传和生化参数、父母和患者的儿科生活质量量表（PedsQL）、儿童抑郁量表、状态特质焦虑量表。
结果
共纳入 30 名接受二巯丁二酸（n=27）或曲恩汀（n=3）螯合剂和锌（n=29）及吡哆醇（n=19）作为补充剂治疗的儿科门诊患者。观察到蛋白尿（n=3）、皮疹（n=2）和胃肠道不适（n=2）。当 MARS-5 与随访时间之间的相关性进行检查时，发现存在显著的负相关（p=0.014）。根据 MPR，二巯丁二酸、锌和吡哆醇的不依从率（漏服剂量≥20%）分别为 29.6%、17.2%和 5.3%。PedsQL 评分高于父母，两者之间呈正相关（p<0.001）。此外，PedsQL 与状态焦虑量表之间存在显著正相关（p<0.001）。比较不同治疗知识水平之间尿铜水平的变化，发现高水平和低水平之间存在显著差异（p=0.043）。
结论
总体而言，根据 MARS-5 评估的不依从率为 23.3%，根据 MPR 评估的不依从率为 5.3-29.6%。考虑随访时间、生化参数、治疗知识、生活质量和焦虑等因素对药物依从性的潜在影响非常重要。
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