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An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria

Dietary restriction of phenylalanine combined with a protein substitute prevents intellectual disability in patients with phenylketonuria (PKU). However, current protein substitutes are associated with low adherence owing to unpalatability and burdensome administration regimens. This prospective, ob...

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Autores principales: MacDonald, Anita, Ashmore, Catherine, Daly, Anne, Pinto, Alex, Evans, Sharon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7551143/
https://www.ncbi.nlm.nih.gov/pubmed/32899129
http://dx.doi.org/10.3390/nu12092686
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author MacDonald, Anita
Ashmore, Catherine
Daly, Anne
Pinto, Alex
Evans, Sharon
author_facet MacDonald, Anita
Ashmore, Catherine
Daly, Anne
Pinto, Alex
Evans, Sharon
author_sort MacDonald, Anita
collection PubMed
description Dietary restriction of phenylalanine combined with a protein substitute prevents intellectual disability in patients with phenylketonuria (PKU). However, current protein substitutes are associated with low adherence owing to unpalatability and burdensome administration regimens. This prospective, observational acceptability study in children with PKU assessed the use of a prolonged-release protein substitute designed with an ethyl cellulose and arginate coating masking the bitter taste, smell and reducing the osmolarity of free amino acids. The study product was mixed with the subject’s food or drink and replaced ≥1 dose per day of the subject’s usual protein substitute for 7 days. Seven of 13 subjects were able to take their prescribed dose over the 7 day period. Most subjects mixed the test protein substitute with food or fruit juice. Reduced blood phenylalanine levels (n = 5) and improved phenylalanine/tyrosine ratio (n = 4) were recorded from baseline to Day 7, respectively. Four subjects reported fewer gastrointestinal symptoms compared to baseline. There were no cases of diarrhoea, constipation, bloating, nausea or vomiting. No adverse reactions were reported. In conclusion, the novel prolonged-release protein substitute was taken in a different way to a typical protein substitute and enabled satisfactory blood phenylalanine control. The study product was well tolerated; subjects experienced fewer gastrointestinal symptoms than with their previous treatment. Although the results of this pilot study provide reassuring data, longer-term studies evaluating adherence and blood phenylalanine control are necessary.
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spelling pubmed-75511432020-10-16 An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria MacDonald, Anita Ashmore, Catherine Daly, Anne Pinto, Alex Evans, Sharon Nutrients Article Dietary restriction of phenylalanine combined with a protein substitute prevents intellectual disability in patients with phenylketonuria (PKU). However, current protein substitutes are associated with low adherence owing to unpalatability and burdensome administration regimens. This prospective, observational acceptability study in children with PKU assessed the use of a prolonged-release protein substitute designed with an ethyl cellulose and arginate coating masking the bitter taste, smell and reducing the osmolarity of free amino acids. The study product was mixed with the subject’s food or drink and replaced ≥1 dose per day of the subject’s usual protein substitute for 7 days. Seven of 13 subjects were able to take their prescribed dose over the 7 day period. Most subjects mixed the test protein substitute with food or fruit juice. Reduced blood phenylalanine levels (n = 5) and improved phenylalanine/tyrosine ratio (n = 4) were recorded from baseline to Day 7, respectively. Four subjects reported fewer gastrointestinal symptoms compared to baseline. There were no cases of diarrhoea, constipation, bloating, nausea or vomiting. No adverse reactions were reported. In conclusion, the novel prolonged-release protein substitute was taken in a different way to a typical protein substitute and enabled satisfactory blood phenylalanine control. The study product was well tolerated; subjects experienced fewer gastrointestinal symptoms than with their previous treatment. Although the results of this pilot study provide reassuring data, longer-term studies evaluating adherence and blood phenylalanine control are necessary. MDPI 2020-09-03 /pmc/articles/PMC7551143/ /pubmed/32899129 http://dx.doi.org/10.3390/nu12092686 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
MacDonald, Anita
Ashmore, Catherine
Daly, Anne
Pinto, Alex
Evans, Sharon
An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria
title An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria
title_full An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria
title_fullStr An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria
title_full_unstemmed An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria
title_short An Observational Study Evaluating the Introduction of a Prolonged-Release Protein Substitute to the Dietary Management of Children with Phenylketonuria
title_sort observational study evaluating the introduction of a prolonged-release protein substitute to the dietary management of children with phenylketonuria
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7551143/
https://www.ncbi.nlm.nih.gov/pubmed/32899129
http://dx.doi.org/10.3390/nu12092686
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