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Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria

2023-08Artigo científico Acesso aberto
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dc.contributor.authorGama, Maria Inês
dc.contributor.authorDaly, Anne
dc.contributor.authorAshmore, Catherine
dc.contributor.authorEvans, Sharon
dc.contributor.authorMoreira-Rosário, André
dc.contributor.authorRocha, Júlio César
dc.contributor.authorMacDonald, Anita
dc.contributor.institutionNOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)
dc.contributor.institutionCentro de Investigação em Tecnologias e Serviços de Saúde (CINTESIS)
dc.contributor.pblMDPI AG
dc.date.accessioned2023-09-13T22:18:43Z
dc.date.available2023-09-13T22:18:43Z
dc.date.issued2023-08
dc.descriptionFunding Information: M.I.G. has received a Recordati Rare Disease Grant Ed. 2021–2022 from the Portuguese Society of Metabolic Diseases (SPDM) to develop this work in the worth of 3000€. Funding Information: M.I.G. has received travelling grants from Cambrooke Therapeutics and Nutricia to attend scientific meetings. A.D. received research funding from Vitaflo International, financial support from Nutricia, Mevalia and Vitaflo International to attend study days and conferences. J.C.R. was a member of the European Nutritionist ExpertPanel (Biomarin), the Advisory Board for Applied Pharma Research, Vitaflo, Synlogic, Biomarin and Nutricia, and received honoraria as a speaker from APR, Merck Serono, Biomarin, Nutricia, Vitaflo, Cambrooke, PIAM and Lifediet. S.E. received research funding from Nutricia, and financial support from Nutricia and Vitaflo International to attend study days and conferences. C.A. received honoraria from Nutricia and Vitaflo International to attend study days and conferences. A.M. has received research funding and honoraria from Nutricia, Vitaflo International, and Biomarin. She is a Member of the Advisory Board Element (Danone-Nutricia). The funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript, or in the decision to publish the results. Publisher Copyright: © 2023 by the authors.
dc.description.abstractIntroduction: In phenylketonuria (PKU) changes in dietary patterns and behaviors in sapropterin-responsive populations have not been widely reported. We aimed to assess changes in food quality, mental health and burden of care in a paediatric PKU sapropterin-responsive cohort. Methods: In an observational, longitudinal study, patient questionnaires on food frequency, neophobia, anxiety and depression, impact on family and burden of care were applied at baseline, 3 and 6-months post successful sapropterin-responsiveness testing (defined as a 30% reduction in blood phenylalanine levels). Results: 17 children (10.8 ± 4.2 years) completed 6-months follow-up. Patients body mass index (BMI) z-scores remained unchanged after sapropterin initiation. Blood phenylalanine was stable. Natural protein increased (p < 0.001) and protein substitute intake decreased (p = 0.002). There were increases in regular cow’s milk (p = 0.001), meat/fish, eggs (p = 0.005), bread (p = 0.01) and pasta (p = 0.011) intakes but special low-protein foods intake decreased. Anxiety (p = 0.016) and depression (p = 0.022) decreased in caregivers. The impact-on-family, familial-social impact (p = 0.002) and personal strain (p = 0.001) lessened. After sapropterin, caregivers spent less time on PKU tasks, the majority ate meals outside the home more regularly and fewer caregivers had to deny food choices to their children. Conclusion: There were significant positive changes in food patterns, behaviors and burden of care in children with PKU and their families after 6-months on sapropterin treatment.en
dc.description.versionpublishersversion
dc.description.versionpublished
dc.format.extent509055
dc.identifier.doi10.3390/nu15163603
dc.identifier.issn1422-8599
dc.identifier.otherPURE: 71099130
dc.identifier.otherPURE UUID: b17f4f7c-59c8-4817-bd16-6346a42c0b3c
dc.identifier.otherScopus: 85168748316
dc.identifier.otherPubMed: 37630793
dc.identifier.otherWOS: 001154118800145
dc.identifier.otherWOS: 001057216200001
dc.identifier.urihttp://hdl.handle.net/10362/157769
dc.identifier.urlhttps://www.scopus.com/pages/publications/85168748316
dc.language.isoeng
dc.peerreviewedyes
dc.subjectburden of care
dc.subjectfood literacy
dc.subjectfood patterns
dc.subjectphenylketonuria
dc.subjectsapropterin
dc.subjectFood Science
dc.subjectNutrition and Dietetics
dc.subjectSDG 3 - Good Health and Well-being
dc.titleImpact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuriaen
dc.title.subtitleA 6 Month Follow-Up Reporten
dc.typejournal article
degois.publication.issue16
degois.publication.titleNutrients
degois.publication.volume15
dspace.entity.typePublication
rcaap.rightsopenAccess

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