Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy

We first introduced the concept of the mTOR pathway’s involvement in congenital hyperinsulinism of infancy (CHI), based largely on morphoproteomic observations and clinical outcomes using sirolimus (rapamycin) as a therapeutic agent in infants refractory to octreotide and diazoxide treatment. Subseq...

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Detalles Bibliográficos
Autores principales: Brown, Robert E., Senniappan, Senthil, Hussain, Khalid, McGuire, Mary F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5732475/
https://www.ncbi.nlm.nih.gov/pubmed/29246172
http://dx.doi.org/10.1186/s13023-017-0735-9
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author Brown, Robert E.
Senniappan, Senthil
Hussain, Khalid
McGuire, Mary F.
author_facet Brown, Robert E.
Senniappan, Senthil
Hussain, Khalid
McGuire, Mary F.
author_sort Brown, Robert E.
collection PubMed
description We first introduced the concept of the mTOR pathway’s involvement in congenital hyperinsulinism of infancy (CHI), based largely on morphoproteomic observations and clinical outcomes using sirolimus (rapamycin) as a therapeutic agent in infants refractory to octreotide and diazoxide treatment. Subsequent publications have verified the efficacy of such treatment in some cases but limited and variable in others. We present further evidence of a constant but variable role for the mTOR pathway in the biology of CHI and provide a strategy that allows for the short-term testing of sirolimus in individual CHI patients.
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spelling pubmed-57324752017-12-21 Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy Brown, Robert E. Senniappan, Senthil Hussain, Khalid McGuire, Mary F. Orphanet J Rare Dis Letter to the Editor We first introduced the concept of the mTOR pathway’s involvement in congenital hyperinsulinism of infancy (CHI), based largely on morphoproteomic observations and clinical outcomes using sirolimus (rapamycin) as a therapeutic agent in infants refractory to octreotide and diazoxide treatment. Subsequent publications have verified the efficacy of such treatment in some cases but limited and variable in others. We present further evidence of a constant but variable role for the mTOR pathway in the biology of CHI and provide a strategy that allows for the short-term testing of sirolimus in individual CHI patients. BioMed Central 2017-12-16 /pmc/articles/PMC5732475/ /pubmed/29246172 http://dx.doi.org/10.1186/s13023-017-0735-9 Text en © The Author(s). 2017 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Letter to the Editor
Brown, Robert E.
Senniappan, Senthil
Hussain, Khalid
McGuire, Mary F.
Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy
title Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy
title_full Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy
title_fullStr Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy
title_full_unstemmed Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy
title_short Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy
title_sort morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mtor pathway in the biology of congenital hyperinsulinism of infancy
topic Letter to the Editor
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5732475/
https://www.ncbi.nlm.nih.gov/pubmed/29246172
http://dx.doi.org/10.1186/s13023-017-0735-9
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