Development of an inventory to assess perceived barriers related to PKU treatment

Abstract

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.

Investigating the Metabolic Model in Preterm Neonates by Tandem Mass Spectrometry: A Cohort Study

“…Therefore, low Leucine intake and abnormal Phenylalanine metabolism could lead to a low ratio of (Leucine + Isoleucine + Proline-OH) to Phenylalanine. Lack of Leucine could affect the normal physical development of preterm neonates due to insufficient energy supply, and abnormal Phenylalanine metabolism could inhibit the intellectual development by producing excess phenyl pyruvate [55,56]. Eliminating these effects is very import for the growth and development of preterm neonates.…”

Factors that increase risk for poor adherence to phenylketonuria treatment in Brazilian patients

“…A general score (0–25) can be computed for the inventory that indicates the global level of perceived barriers. A higher score indicates a higher level of perceived barriers (Teruya et al, 2020).…”

“…Information concerning perceived barriers to adherence to PKU treatment was collected with the Perceived Barriers to Phenylketonuria Treatment Inventory (Teruya et al, 2020)…”

Burden of phenylketonuria in Latin American patients: A systematic review and meta-analysis of observational studies

“…Six of the included studies were published only as an abstract [18,19,37,57, 58, 67,104], nine studies as a thesis[33,43,49, 65,70,73, 88,93, 96], and the majority (n = 57) were published as full-text in peerreviewed journals[17, 20-23, 28-30, 34-36, 38-42, 44-48, 50-56, 59-64, 66, 68, 69, 71, 72, 74-87, 94, 95, 97-99, 102, 103]. Seven further studies[24, 26,31, 89,91,100,105] were published initially as a thesis followed by a full-text publication[25,27,32,90,92,101, 106]. When information regarding risk of bias or other aspects related to study criteria were unavailable in the methods, we attempted to contact study authors for additional information.Forty of the included studies were conducted inBrazil [18,20,[24][25][26][27][31][32][33]40,42,43,49,50,52,55,59, 60, 62, 63,[65][66][67][68][70][71][72][78][79][80][86][87][88][89][90][91][92]…”

“…Seven further studies[24, 26,31, 89,91,100,105] were published initially as a thesis followed by a full-text publication[25,27,32,90,92,101, 106]. When information regarding risk of bias or other aspects related to study criteria were unavailable in the methods, we attempted to contact study authors for additional information.Forty of the included studies were conducted inBrazil [18,20,[24][25][26][27][31][32][33]40,42,43,49,50,52,55,59, 60, 62, 63,[65][66][67][68][70][71][72][78][79][80][86][87][88][89][90][91][92][93][94][95]97,[99][100][101][102]105, 106], seven in Argentina[19, 22, 23, 28-30, 34, 44, 45, 57, 58], ve in Chile[35, 36, 38,39,77], three each in Colombia [46,53,...…”