Sirolimus therapy in infants with severe hyperinsulinemic hypoglycemia

Area of Science:

• Pediatric Endocrinology
• Neonatal Medicine
• Pharmacology

Background:

• Hyperinsulinemic hypoglycemia is a primary cause of persistent neonatal hypoglycemia.
• Subtotal pancreatectomy is the standard treatment for diazoxide-unresponsive cases.
• Alternative therapies are needed for infants refractory to current treatments.

Observation:

• Four infants with severe, diazoxide- and octreotide-resistant hyperinsulinemic hypoglycemia were treated with sirolimus.
• Maximal diazoxide and octreotide doses were administered prior to sirolimus initiation.
• The study monitored glycemic response and adverse events over a 1-year follow-up period.

Findings:

• All four infants showed a clear glycemic response to sirolimus.
• One patient required a supplementary low dose of octreotide for sustained normoglycemia.
• No major adverse events were reported during the 1-year follow-up period.

Implications:

• Sirolimus represents a viable therapeutic option for severe hyperinsulinemic hypoglycemia refractory to conventional treatments.
• This finding may reduce the need for surgical intervention like pancreatectomy in affected neonates.
• Further research is warranted to establish optimal dosing and long-term efficacy of sirolimus in neonatal hypoglycemia.