Quality of life and adherence to treatment in early-treated Brazilian phenylketonuria pediatric patients

Autor: E. Vieira Neto, Lilian de Mattos Carvalho, Ida Vanessa Doederlein Schwartz, Ana Paula Vanz, Márcia Gonçalves Ribeiro, Claudia Braga Monteiro, Tássia Tonon, H.S. Maia Filho
Jazyk: angličtina
Rok vydání: 2018
Předmět:
Male
Parents
Questionnaires
Pediatrics
Time Factors
Physiology
Phenylketonurias
First year of life
Fenilcetonuria
Biochemistry
0302 clinical medicine
Secondary Prevention
Phenylketonuria
Reference population
General Pharmacology
Toxicology and Pharmaceutics
Child
lcsh:QH301-705.5
Research Articles
Intelligence Tests
Metabolismo
lcsh:R5-920
Intelligence quotient
General Neuroscience
Age Factors
General Medicine
humanities
Pediatric patient
Treatment Outcome
Female
Analysis of variance
lcsh:Medicine (General)
Brazil
Quality of life
medicine.medical_specialty
congenital
hereditary
and neonatal diseases and abnormalities
Adolescent
Diet therapy
Phenylalanine
Immunology
Biophysics
030209 endocrinology & metabolism
Ocean Engineering
03 medical and health sciences
medicine
Humans
Analysis of Variance
business.industry
Significant difference
Patient compliance
Cell Biology
Fenilalanina
Proxy
Treatment Adherence and Compliance
Socioeconomic Factors
lcsh:Biology (General)
Linear Models
Self Report
business
030217 neurology & neurosurgery
Zdroj: Repositório Institucional da UFRGS
Universidade Federal do Rio Grande do Sul (UFRGS)
instacron:UFRGS
Brazilian Journal of Medical and Biological Research, Vol 51, Iss 2 (2017)
Brazilian Journal of Medical and Biological Research
Brazilian Journal of Medical and Biological Research v.51 n.2 2018
Associação Brasileira de Divulgação Científica (ABDC)
instacron:ABDC
Brazilian Journal of Medical and Biological Research, Volume: 51, Issue: 2, Article number: e6709, Published: 11 DEC 2017
Popis: Early dietary treatment of phenylketonuria (PKU), an inborn error of phenylalanine (Phe) metabolism, results in normal cognitive development. Although health-related quality of life (HRQoL) of PKU patients has been reported as unaffected in high-income countries, there are scarce data concerning HRQoL and adherence to treatment of PKU children and adolescents from Brazil. The present study compared HRQoL scores in core dimensions of Brazilian early-treated PKU pediatric patients with those of a reference population, and explored possible relationships between adherence to treatment and HRQoL. Early-treated PKU pediatric patient HRQoL was evaluated by self- and parent-proxy reports of the Pediatric Quality of Life Inventory (PedsQL) core scales. Adherence to treatment was evaluated by median Phe levels and percentage of results within the therapeutic target range in two periods. Means for total and core scales scores of PedsQL self- and parent proxy-reports of PKU patients were significantly lower than their respective means for controls. Adequacy of median Phe concentrations and the mean percentage of values in the target range fell substantially from the first year of life to the last year of this study. There was no significant difference in mean total and core scale scores for self- and parent proxy-reports between patients with adequate and those with inadequate median Phe concentrations. The harmful consequences for intellectual capacity caused by poor adherence to dietary treatment could explain the observed decrease in all HRQoL scales, especially in school functioning. Healthcare system and financial difficulties may also have influenced negatively all HRQoL dimensions.
Databáze: OpenAIRE
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