Sirolimus Is Effective in Infants with Severe Hyperinsulinemic Hypoglycemia

Severe infantile hypoglycemia from hyperinsulinemia, most often from genetic dysregulation of pancreatic beta cells, is a major therapeutic challenge. When infants do not respond to diazoxide and octreotide along with intravenous glucose infusions, subtotal pancreatectomy is required with inevitable diabetes mellitus later in childhood.

Investigators now report outcomes in four children (born at 33–40 weeks' gestation) with hyperinsulinemic hypoglycemia resistant to diazoxide and octreotide who were treated with sirolimus (starting dose, 0.05 mg per square meter body surface per day, increased to reach a serum trough level of 5–15 ng/mL) beginning at ages 7 to 16 weeks. All infants had symptomatic hypoglycemia with seizures, poor feeding, an…