Efficacy and safety of sirolimus in a neonate with persistent hypoglycaemia following near-total pancreatectomy for hyperinsulinaemic hypoglycaemia.
Author
Abraham, M. B.
Shetty, V. B.
Price, G.
Smith, N.
Bock, M. de
Siafarikas, A.
Resnick, S.
Whan, E.
Ellard, Sian
Flanagan, Sarah
Davis, E.A.
Jones, T. W.
Hussain, K.
Choong, C. S.
Date
2015-11-01Journal
Journal of Pediatric Endocrinology & Metabolism : JPEMType
Journal ArticleResearch Support, Non-U.S. Gov't
Publisher
De GruyterDOI
10.1515/jpem-2015-0094Rights
Archived with thanks to Journal of pediatric endocrinology & metabolism : JPEMMetadata
Show full item recordAbstract
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.