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Development of an inventory to assess perceived barriers related to PKU treatment
Katia Irie Teruya1, Eduardo Remor2, Ida Vanessa Doederlein Schwartz3
1Institute of Psychology, Universidade Federal do Rio Grande do Sul. Rua Ramiro Barcelos, 2600 (sala 219), Porto Alegre, Rio Grande do Sul, 90035003, Brazil.
Insights
Phenylketonuria (PKU) patients with poor metabolic control report more treatment barriers. This study developed an inventory to assess these barriers, finding they correlate with higher phenylalanine levels in PKU patients.
Area of Science:
- Metabolic Disorders
- Genetics and Heredity
- Patient-Reported Outcomes
Background:
- Phenylketonuria (PKU) management in Brazil shows suboptimal metabolic control, particularly from childhood.
- Understanding patient-perceived barriers is crucial for improving treatment adherence and outcomes in PKU.
- Existing research lacks a comprehensive tool to identify barriers specific to PKU treatment from multiple stakeholder perspectives.
Purpose of the Study:
- To develop and psychometrically evaluate an inventory for identifying perceived barriers to PKU treatment.
- To explore sociodemographic and medical predictors of perceived barriers in PKU patients.
- To examine the association between the number of perceived barriers and treatment adherence in PKU.
Main Methods:
- Development of a novel inventory to assess perceived barriers to PKU treatment.
- Administration of the inventory to PKU patients (≥13 years) and caregivers of younger patients (6-17 years).
- Analysis of psychometric properties and correlation of inventory scores with phenylalanine levels from medical records.
Main Results:
- The developed inventory effectively identified perceived barriers across different age groups and PKU severities.
- Adolescents reported fewer barriers than adults, and patients with better metabolic control reported fewer barriers.
- A positive association was found between the number of perceived barriers and blood phenylalanine concentration, indicating poorer adherence.
Conclusions:
- The newly developed inventory demonstrates validity in assessing perceived barriers in PKU treatment.
- Findings underscore the significance of perceived barriers in influencing metabolic control and adherence in PKU.
- Further research is warranted to explore and address the barriers faced by PKU patients.
Background:
According to studies of phenylketonuria (PKU), the Brazilian population's metabolic control shows unsatisfactory indexes from childhood. Research on patients' perceived difficulties or barriers to adherence to treatment can help us to comprehend how these outcomes are associated. The present study aimed to: (1) describe the development of an inventory for identifying the most frequent and relevant perceived barriers to PKU treatment from the perspective of patients, caregivers, and healthcare professionals; (2) evaluate certain psychometric characteristics of the new measure; and, (3) explore potential predictors (sociodemographic and medical characteristics) that may contribute to increasing the number of perceived barriers and examine whether the number of barriers is associated with the degree of adherence shown by the patient.
Results:
Participants in the study were 23 patients with PKU (M age = 18.0 years; SD = 7.3; range 6 to 34 years; 69% early-treated) in classical (n = 11) and mild (n = 12) form, and 11 caregivers. The inventory, developed to ascertain perceived barriers to treatment, was completed by patients (≥ 13 years) and caregivers of patients aged 6 to 17 years. Analyses were conducted to investigate whether barrier inventory scores were associated with adherence to treatment as measured by phenylalanine levels in patients' medical records. Scores on the inventory differed across the patient age groups: adolescents had lower scores (i.e. reported fewer barriers) compared with those of adults (U = 8.000, p = 0.008); patients with better recent metabolic control also reported fewer perceived barriers than did patients with poor adherence (U = 20.000, p = 0.009); and the number of perceived barriers was positively associated with recent blood phenylalanine concentration (Kendall's taub = 0.41; p = 0.001).
Conclusions:
These results suggest that the inventory has merit in assessing perceived barriers and support the need for further research on barriers perceived by PKU patients.
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