Katia Irie Teruya, Eduardo Remor, Ida Vanessa Doederlein Schwartz
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引用次数: 0
Abstract
Background: Metabolic control in the phenylketonuria (PKU) population is challenging. Perceived barriers to treatment adherence have been shown to be associated with metabolic control outcomes. The objective of this study was to evaluate the psychometric properties of the Perceived Barriers to PKU Treatment Inventory with patients and caregivers in different regions of Brazil.
Methods: This is a cross-sectional, observational, non-probability sampling study. The inventory has 25 dichotomous items and two versions, patients and caregivers. Data collection in the present study included online assessment (n = 241) and presential mode (n = 42). Sixty-three PKU patients and 220 caregivers of individuals with PKU participated in this study. Internal consistency reliability, convergent validity, known-group validity, and discriminant validity of both versions (patient and proxy report) were evaluated. Furthermore, score norms based on the current sample are presented. A content analysis of the responses recorded in the open-ended inventory was also conducted.
Results: Adequate reliability and evidence for convergent validity, known- groups validity, and discriminant validity were observed in this sample. Optimal cutoff points were identified through ROC curves (score > 9 for the patient version and score > 6 for the caregiver-proxy version), allowing for the identification of the risk of poor metabolic control due to barriers to dietary adherence. Furthermore, the qualitative analysis showed the relevance of the 25 items and the importance of addressing socio-economic and health system factors that have a significant economic impact on patients' and families' lives.
Conclusion: The findings indicate that the Perceived Barriers to PKU Treatment Inventory is valuable for screening barriers that may contribute to increasing the difficulty of treatment management. And to identify patients and caregivers who may require targeted support to improve dietary adherence and metabolic control.
期刊介绍：
Orphanet Journal of Rare Diseases is an open access, peer-reviewed journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high-quality reviews on specific rare diseases. In addition, the journal may consider articles on clinical trial outcome reports, either positive or negative, and articles on public health issues in the field of rare diseases and orphan drugs. The journal does not accept case reports.
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