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

Detalhes bibliográficos
Autor(a) principal: Gama, Maria Inês
Data de Publicação: 2023
Outros Autores: Daly, Anne, Ashmore, Catherine, Evans, Sharon, Moreira-Rosário, André, César Rocha, Júlio, MacDonald, Anita
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10362/157769
Resumo: Funding 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.
id RCAP_8e404c5bc9f8dc0ee237a67fe5682a02
oai_identifier_str oai:run.unl.pt:10362/157769
network_acronym_str RCAP
network_name_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository_id_str 7160
spelling Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with PhenylketonuriaA 6 Month Follow-Up Reportburden of carefood literacyfood patternsphenylketonuriasapropterinFood ScienceNutrition and DieteticsSDG 3 - Good Health and Well-beingFunding 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.Introduction: 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.NOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)Centro de Investigação em Tecnologias e Serviços de Saúde (CINTESIS)RUNGama, Maria InêsDaly, AnneAshmore, CatherineEvans, SharonMoreira-Rosário, AndréCésar Rocha, JúlioMacDonald, Anita2023-09-13T22:18:43Z2023-082023-08-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10362/157769eng1422-8599PURE: 71099130https://doi.org/10.3390/nu15163603info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2024-03-11T05:40:04Zoai:run.unl.pt:10362/157769Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T03:56:52.075983Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
A 6 Month Follow-Up Report
title Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
spellingShingle Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
Gama, Maria Inês
burden of care
food literacy
food patterns
phenylketonuria
sapropterin
Food Science
Nutrition and Dietetics
SDG 3 - Good Health and Well-being
title_short Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
title_full Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
title_fullStr Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
title_full_unstemmed Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
title_sort Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
author Gama, Maria Inês
author_facet Gama, Maria Inês
Daly, Anne
Ashmore, Catherine
Evans, Sharon
Moreira-Rosário, André
César Rocha, Júlio
MacDonald, Anita
author_role author
author2 Daly, Anne
Ashmore, Catherine
Evans, Sharon
Moreira-Rosário, André
César Rocha, Júlio
MacDonald, Anita
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv NOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)
Centro de Investigação em Tecnologias e Serviços de Saúde (CINTESIS)
RUN
dc.contributor.author.fl_str_mv Gama, Maria Inês
Daly, Anne
Ashmore, Catherine
Evans, Sharon
Moreira-Rosário, André
César Rocha, Júlio
MacDonald, Anita
dc.subject.por.fl_str_mv burden of care
food literacy
food patterns
phenylketonuria
sapropterin
Food Science
Nutrition and Dietetics
SDG 3 - Good Health and Well-being
topic burden of care
food literacy
food patterns
phenylketonuria
sapropterin
Food Science
Nutrition and Dietetics
SDG 3 - Good Health and Well-being
description Funding 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.
publishDate 2023
dc.date.none.fl_str_mv 2023-09-13T22:18:43Z
2023-08
2023-08-01T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10362/157769
url http://hdl.handle.net/10362/157769
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 1422-8599
PURE: 71099130
https://doi.org/10.3390/nu15163603
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron:RCAAP
instname_str Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron_str RCAAP
institution RCAAP
reponame_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
repository.mail.fl_str_mv
_version_ 1799138152413134848