ESPE Abstracts

Introduction: Octreotide is recommended as a second-line treatment for patients with congenital hyperinsulinism (CHI), particularly for those who do not respond to diazoxide orsurgical treatment. While the studies on the adverse effects ofoctreotide are still scarce in large cohorts.

Methods: A retrospective study was conducted on CHIpatients who were treated with octreotide in the two largestcenters in China. The study collected and analyzed adverseevents of 122 patients with CHI and octreotide administration.

Results: The success rate with octreotide therapy alone was93% (106/114). The median maximum dose of octreotideadministered was 12.0 μg/kg/d. There was no significantdifference in the dose of octreotide required between patientswith monogenic and syndromic forms of CHI, or betweenpatients with KATP channel mutations and GCK heterozygousmutations. Additionally, no statistical difference was observedin the dose of octreotide required by patients with diffuse andfocal lesions. The study found that the common adverseevents were gastrointestinal symptoms (20.5%), hepatobiliaryinjuries (31.1%), and transient hyperglycemia (49.2%). Onlyone patient developed necrotizing enterocolitis. Our study found that adverse events were experienced by 58%, 69%, 94%, and 100% of patients who received octreotide at doses of 5-10, 10-15, 15-20, and >20 ug/kg/d, respectively. Patientswho experienced adverse events had a significantly highermean dose of octreotide compared to those who did not.

Conclusion: Octreotide was effective in treating CHI patientswho were unresponsive to diazoxide, regardless of theirhistologic subtype. Our study found that patients treated withlower octreotide dose experienced fewer adverse events. Therefore, we recommend that patients receiving octreotide therapy be closely monitored, particularly those receiving higher doses.