Impact on Diet Quality and Burden of Care in Sapropterin Dihydrochloride Use in Children with Phenylketonuria
Autor(a) principal: | |
---|---|
Data de Publicação: | 2023 |
Outros Autores: | , , , , , |
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 |