Efficacy and safety of sirolimus in a neonate with persistent hypoglycaemia following near-total pancreatectomy for hyperinsulinaemic hypoglycaemia.

2.50
Hdl Handle:
http://hdl.handle.net/11287/618154
Title:
Efficacy and safety of sirolimus in a neonate with persistent hypoglycaemia following near-total pancreatectomy for hyperinsulinaemic hypoglycaemia.
Authors:
Abraham, M. B.; Shetty, V. B.; Price, G.; Smith, N.; Bock, M. de; Siafarikas, A.; Resnick, S.; Whan, E.; Ellard, Sian ( 0000-0002-7620-5526 ) ; Flanagan, Sarah; Davis, E.A.; Jones, T. W.; Hussain, K.; Choong, C. S.
Abstract:
Hyperinsulinaemic hypoglycaemia (HH) is characterised by inappropriate insulin secretion and is the most common cause for persistent neonatal hypoglycaemia. The only treatment available for medically unresponsive hypoglycaemia is a near-total pancreatectomy. A neonate with severe HH, due to a homozygous ABCC8 mutation, was not responsive to treatment with maximal doses of diazoxide and subcutaneous daily octreotide, and underwent a near-total pancreatectomy; however, hypoglycaemia persisted. Introduction of sirolimus, an mTOR (mammalian target of rapamycin) inhibitor, obviated the requirement for glucose infusion. Euglycaemia was achieved with no significant adverse events from the drug. Sirolimus therapy was ceased at 13 months of age. No episodes of persistent hypoglycaemia were observed after cessation of sirolimus. This report demonstrates the successful use of sirolimus for persistent hypoglycaemia in the critically ill infant post pancreatectomy. Sirolimus could be considered in patients with severe HH not responsive to conventional medical and surgical therapy. However, the long-term efficacy and safety with this immunosuppressive drug in very young patients are not assured.
Citation:
Efficacy and safety of sirolimus in a neonate with persistent hypoglycaemia following near-total pancreatectomy for hyperinsulinaemic hypoglycaemia. 2015, 28 (11-12):1391-8 J. Pediatr. Endocrinol. Metab.
Publisher:
De Gruyter
Journal:
Journal of Pediatric Endocrinology & Metabolism : JPEM
Issue Date:
1-Nov-2015
URI:
http://hdl.handle.net/11287/618154
DOI:
10.1515/jpem-2015-0094
PubMed ID:
26226122
Additional Links:
http://www.degruyter.com/doi/10.1515/jpem-2015-0094
Type:
Journal Article; Research Support, Non-U.S. Gov't
Language:
en
ISSN:
2191-0251
Appears in Collections:
2015 RD&E publications; Molecular Genetics; Honorary contracts publications

Full metadata record

DC FieldValue Language
dc.contributor.authorAbraham, M. B.en
dc.contributor.authorShetty, V. B.en
dc.contributor.authorPrice, G.en
dc.contributor.authorSmith, N.en
dc.contributor.authorBock, M. deen
dc.contributor.authorSiafarikas, A.en
dc.contributor.authorResnick, S.en
dc.contributor.authorWhan, E.en
dc.contributor.authorEllard, Sianen
dc.contributor.authorFlanagan, Sarahen
dc.contributor.authorDavis, E.A.en
dc.contributor.authorJones, T. W.en
dc.contributor.authorHussain, K.en
dc.contributor.authorChoong, C. S.en
dc.date.accessioned2016-08-10T09:27:23Z-
dc.date.available2016-08-10T09:27:23Z-
dc.date.issued2015-11-01-
dc.identifier.citationEfficacy and safety of sirolimus in a neonate with persistent hypoglycaemia following near-total pancreatectomy for hyperinsulinaemic hypoglycaemia. 2015, 28 (11-12):1391-8 J. Pediatr. Endocrinol. Metab.en
dc.identifier.issn2191-0251-
dc.identifier.pmid26226122-
dc.identifier.doi10.1515/jpem-2015-0094-
dc.identifier.urihttp://hdl.handle.net/11287/618154-
dc.description.abstractHyperinsulinaemic hypoglycaemia (HH) is characterised by inappropriate insulin secretion and is the most common cause for persistent neonatal hypoglycaemia. The only treatment available for medically unresponsive hypoglycaemia is a near-total pancreatectomy. A neonate with severe HH, due to a homozygous ABCC8 mutation, was not responsive to treatment with maximal doses of diazoxide and subcutaneous daily octreotide, and underwent a near-total pancreatectomy; however, hypoglycaemia persisted. Introduction of sirolimus, an mTOR (mammalian target of rapamycin) inhibitor, obviated the requirement for glucose infusion. Euglycaemia was achieved with no significant adverse events from the drug. Sirolimus therapy was ceased at 13 months of age. No episodes of persistent hypoglycaemia were observed after cessation of sirolimus. This report demonstrates the successful use of sirolimus for persistent hypoglycaemia in the critically ill infant post pancreatectomy. Sirolimus could be considered in patients with severe HH not responsive to conventional medical and surgical therapy. However, the long-term efficacy and safety with this immunosuppressive drug in very young patients are not assured.en
dc.language.isoenen
dc.publisherDe Gruyteren
dc.relation.urlhttp://www.degruyter.com/doi/10.1515/jpem-2015-0094en
dc.rightsArchived with thanks to Journal of pediatric endocrinology & metabolism : JPEMen
dc.subjectWessex Classification Subject Headings::Endocrinology::Diabetesen
dc.subjectWessex Classification Subject Headings::Paediatricsen
dc.titleEfficacy and safety of sirolimus in a neonate with persistent hypoglycaemia following near-total pancreatectomy for hyperinsulinaemic hypoglycaemia.en
dc.typeJournal Articleen
dc.typeResearch Support, Non-U.S. Gov'ten
dc.identifier.journalJournal of Pediatric Endocrinology & Metabolism : JPEMen
dc.type.versionPublisheden

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