Observational studyOrphanet journal of rare diseases2025
Psychometric properties of perceived barriers to PKU treatment inventory in a Brazilian national sample.
Observational study in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundMetabolic 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.
methodsThis 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.
resultsAdequate 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.
conclusionThe 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. CLINICAL TRIAL NUMBER: Not applicable.
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