Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient

Abstract We have recently published on the limited effectiveness of sirolimus as a treatment option for hypoglycaemia as a consequence of hyperinsulinism. Our data oppose the view that mTOR inhibitors provide new opportunities for the treatment of patients with hyperinsulinism. We are not convinced...

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Main Authors:	Indraneel Banerjee, Diva De Leon, Mark J. Dunne
Format:	Article
Language:	English
Published:	BMC 2017-04-01
Series:	Orphanet Journal of Rare Diseases
Subjects:	Sirolimus Congenital hyperinsulinism in Infancy patient mTOR islet hypoglycaemia
Online Access:	http://link.springer.com/article/10.1186/s13023-017-0621-5

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spelling	doaj-435c6286008e464e8d03535bca894e452020-11-24T20:55:59ZengBMCOrphanet Journal of Rare Diseases1750-11722017-04-011211310.1186/s13023-017-0621-5Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patientIndraneel Banerjee0Diva De Leon1Mark J. Dunne2Paediatric Endocrinology, Central Manchester University Hospitals NHS Foundation Trust (CMFT) and The University of ManchesterCongenital Hyperinsulinism Center, Division of Pediatric Endocrinology and Diabetes, The Children’s Hospital of PhiladelphiaPaediatric Endocrinology, Central Manchester University Hospitals NHS Foundation Trust (CMFT) and The University of ManchesterAbstract We have recently published on the limited effectiveness of sirolimus as a treatment option for hypoglycaemia as a consequence of hyperinsulinism. Our data oppose the view that mTOR inhibitors provide new opportunities for the treatment of patients with hyperinsulinism. We are not convinced by the argument that any benefit for some patients outweighs the potential and later long-term problems that accompany mTOR inhibition in the neonate. We also express the opinion that caution must be taken when repurposing/repositioning therapies in the field of rare disease.http://link.springer.com/article/10.1186/s13023-017-0621-5SirolimusCongenital hyperinsulinism in Infancy patientmTORislethypoglycaemia
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Indraneel Banerjee Diva De Leon Mark J. Dunne
spellingShingle	Indraneel Banerjee Diva De Leon Mark J. Dunne Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient Orphanet Journal of Rare Diseases Sirolimus Congenital hyperinsulinism in Infancy patient mTOR islet hypoglycaemia
author_facet	Indraneel Banerjee Diva De Leon Mark J. Dunne
author_sort	Indraneel Banerjee
title	Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
title_short	Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
title_full	Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
title_fullStr	Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
title_full_unstemmed	Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
title_sort	extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
publisher	BMC
series	Orphanet Journal of Rare Diseases
issn	1750-1172
publishDate	2017-04-01
description	Abstract We have recently published on the limited effectiveness of sirolimus as a treatment option for hypoglycaemia as a consequence of hyperinsulinism. Our data oppose the view that mTOR inhibitors provide new opportunities for the treatment of patients with hyperinsulinism. We are not convinced by the argument that any benefit for some patients outweighs the potential and later long-term problems that accompany mTOR inhibition in the neonate. We also express the opinion that caution must be taken when repurposing/repositioning therapies in the field of rare disease.
topic	Sirolimus Congenital hyperinsulinism in Infancy patient mTOR islet hypoglycaemia
url	http://link.springer.com/article/10.1186/s13023-017-0621-5
work_keys_str_mv	AT indraneelbanerjee extremecautionontheuseofsirolimusforthecongenitalhyperinsulinismininfancypatient AT divadeleon extremecautionontheuseofsirolimusforthecongenitalhyperinsulinismininfancypatient AT markjdunne extremecautionontheuseofsirolimusforthecongenitalhyperinsulinismininfancypatient
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Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient

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