Octreotide efficacy and safety in children with hyperinsulinism: evidence from two Chinese centers

Octreotide is recommended as a second-line treatment for patients with congenital hyperinsulinism (CHI), especially for those who do not respond to diazoxide or surgical intervention. Studies on the adverse effects of octreotide in large cohorts are still scarce. We evaluated the safety of octreotide in CHI patients by reviewing cases from the two largest centres in China that specialize in the management of this condition. Our study analysed adverse events in 122 CHI patients on octreotide, with a 93% success rate for the therapy alone. The mean maximum dose of octreotide was 13.1 ± 6.5 µg/kg/day, with no difference in required doses between diffuse and focal lesion patients. Common side effects were hepatobiliary injuries (20.5%), gastrointestinal symptoms (31.1%), and transient hyperglycaemia (49.2%), with one case of necrotizing enterocolitis. Adverse event rates increased in patients treated with intermediate octreotide dose to those treated with higher doses, rising from 58% at doses of 5-10 µg/kg/day to 100% at doses exceeding 20 µg/kg/day. Patients experiencing adverse events received significantly higher doses.

Octreotide effectively maintained blood glucose levels in diazoxide-unresponsive CHI patients without serious adverse effects, across all subtypes. Our study suggests that intermediate octreotide dosing is associated with a lower incidence of adverse events. We recommend close monitoring of patients receiving octreotide, especially during the initiation phase and when higher doses are administered.

• Octreotide is a commonly used second line treatment for patients with congenital hyperinsulinism. • The administration of octreotide is frequently associated with gastrointestinal adverse events.

• Our study indicates that octreotide is used without serious adverse effects to maintain blood glucose levels in infants with congenital hyperinsulinism who are otherwise in good health. • The incidence of adverse events in patients treated with octreotide is dose-dependent.