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

J Pediatr Endocrinol Metab. 2015 Nov 1;28(11-12):1391-8. doi: 10.1515/jpem-2015-0094.

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.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Congenital Hyperinsulinism / surgery*
  • Humans
  • Hypoglycemia / drug therapy*
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Infant, Newborn
  • Male
  • Pancreatectomy*
  • Sirolimus / adverse effects
  • Sirolimus / therapeutic use*
  • Treatment Outcome

Substances

  • Immunosuppressive Agents
  • Sirolimus