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"MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial
BACKGROUND: Phenylketonuria (PKU) is an autosomal recessive disorder of phenylalanine metabolism. The inability to convert phenylalanine (Phe) into tyrosine causes Phe to accumulate in the body. Adherence to a protein restricted diet, resulting in reduced Phe levels, is essential to prevent cognitiv...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2011
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3133536/ https://www.ncbi.nlm.nih.gov/pubmed/21708003 http://dx.doi.org/10.1186/1750-1172-6-48 |
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author | ten Hoedt, Amber E Hollak, Carla EM Boelen, Carolien CA van der Herberg-van de Wetering, N Ada P ter Horst, Nienke M Jonkers, Cora F Wijburg, Frits A Bosch, Annet M |
author_facet | ten Hoedt, Amber E Hollak, Carla EM Boelen, Carolien CA van der Herberg-van de Wetering, N Ada P ter Horst, Nienke M Jonkers, Cora F Wijburg, Frits A Bosch, Annet M |
author_sort | ten Hoedt, Amber E |
collection | PubMed |
description | BACKGROUND: Phenylketonuria (PKU) is an autosomal recessive disorder of phenylalanine metabolism. The inability to convert phenylalanine (Phe) into tyrosine causes Phe to accumulate in the body. Adherence to a protein restricted diet, resulting in reduced Phe levels, is essential to prevent cognitive decline. Frequent evaluation of plasma Phe levels and, if necessary, adjustment of the diet are the mainstay of treatment. We aimed to assess whether increased self-management of PKU patients and/or their parents is feasible and safe, by providing direct online access to blood Phe values without immediate professional guidance. METHODS: Thirty-eight patients aged ≥ 1 year participated in a 10 month randomized controlled trial. Patients were randomized into a study group (1) or a control group (2). Group 2 continued the usual procedure: a phone call or e-mail by a dietician in case of a deviant Phe value. Group 1 was given a personal "My PKU" web page with a graph of their recent and previous Phe values, online general information about the dietary treatment and the Dutch PKU follow-up guidelines, and a message-box to contact their dietician if necessary. Phe values were provided on "My PKU" without advice. Outcome measures were: differences in mean Phe value, percentage of values above the recommended range and Phe sample frequency, between a 10-month pre-study period and the study period in each group, and between the groups in both periods. Furthermore we assessed satisfaction of patients and/or parents with the 'My PKU' procedure of online availability. RESULTS: There were no significant differences in mean Phe value, percentage of values above recommended range or in frequency of blood spot sampling for Phe determination between the pre-study period and the study period in each group, nor between the 2 groups during the periods. All patients and/or parents expressed a high level of satisfaction with the new way of disease management. CONCLUSIONS: Increased self-management in PKU by providing patients and/or parents their Phe values without advice is feasible and safe and is highly appreciated. TRIAL REGISTRATION: The trial was registered with The Netherlands National Trial Register (NTR #1171) before recruitment of patients. |
format | Online Article Text |
id | pubmed-3133536 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2011 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-31335362011-07-12 "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial ten Hoedt, Amber E Hollak, Carla EM Boelen, Carolien CA van der Herberg-van de Wetering, N Ada P ter Horst, Nienke M Jonkers, Cora F Wijburg, Frits A Bosch, Annet M Orphanet J Rare Dis Research BACKGROUND: Phenylketonuria (PKU) is an autosomal recessive disorder of phenylalanine metabolism. The inability to convert phenylalanine (Phe) into tyrosine causes Phe to accumulate in the body. Adherence to a protein restricted diet, resulting in reduced Phe levels, is essential to prevent cognitive decline. Frequent evaluation of plasma Phe levels and, if necessary, adjustment of the diet are the mainstay of treatment. We aimed to assess whether increased self-management of PKU patients and/or their parents is feasible and safe, by providing direct online access to blood Phe values without immediate professional guidance. METHODS: Thirty-eight patients aged ≥ 1 year participated in a 10 month randomized controlled trial. Patients were randomized into a study group (1) or a control group (2). Group 2 continued the usual procedure: a phone call or e-mail by a dietician in case of a deviant Phe value. Group 1 was given a personal "My PKU" web page with a graph of their recent and previous Phe values, online general information about the dietary treatment and the Dutch PKU follow-up guidelines, and a message-box to contact their dietician if necessary. Phe values were provided on "My PKU" without advice. Outcome measures were: differences in mean Phe value, percentage of values above the recommended range and Phe sample frequency, between a 10-month pre-study period and the study period in each group, and between the groups in both periods. Furthermore we assessed satisfaction of patients and/or parents with the 'My PKU' procedure of online availability. RESULTS: There were no significant differences in mean Phe value, percentage of values above recommended range or in frequency of blood spot sampling for Phe determination between the pre-study period and the study period in each group, nor between the 2 groups during the periods. All patients and/or parents expressed a high level of satisfaction with the new way of disease management. CONCLUSIONS: Increased self-management in PKU by providing patients and/or parents their Phe values without advice is feasible and safe and is highly appreciated. TRIAL REGISTRATION: The trial was registered with The Netherlands National Trial Register (NTR #1171) before recruitment of patients. BioMed Central 2011-06-27 /pmc/articles/PMC3133536/ /pubmed/21708003 http://dx.doi.org/10.1186/1750-1172-6-48 Text en Copyright ©2011 ten Hoedt et al; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/2.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Research ten Hoedt, Amber E Hollak, Carla EM Boelen, Carolien CA van der Herberg-van de Wetering, N Ada P ter Horst, Nienke M Jonkers, Cora F Wijburg, Frits A Bosch, Annet M "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial |
title | "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial |
title_full | "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial |
title_fullStr | "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial |
title_full_unstemmed | "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial |
title_short | "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial |
title_sort | "my pku": increasing self-management in patients with phenylketonuria. a randomized controlled trial |
topic | Research |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3133536/ https://www.ncbi.nlm.nih.gov/pubmed/21708003 http://dx.doi.org/10.1186/1750-1172-6-48 |
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