Abstract

Background and objectives

Congenital hyperinsulinism (CHI) is a rare condition caused by dysregulation of pancreatic insulin secretion, leading to severe hypoglycemia in children. In diazoxide-unresponsive patients, somatostatin analogs are recommended as a treatment option. The aim of the present study was to evaluate the clinical characteristics, growth patterns, side effect profiles, and long-term follow-up of CHI patients who received octreotide long-acting release (octreotide-LAR) therapy.

Methods

In the study, data from 23 CHI patients (F/M: 9/14) who received octreotide-LAR therapy between 2010 and 2025 were collected retrospectively. Data regarding glycemic control, growth parameters, and treatment-related adverse events were collected. The initial dose of octreotide-LAR was calculated as the total monthly dose of short-acting octreotide and was thereafter adjusted based on blood glucose monitoring results.

Results

The median age at diagnosis was 15 days (2–120), and the most common presenting symptom was hypoglycemic seizures (78.2%). Genetic mutations were identified in 14 patients (60.8%). Octreotide-LAR therapy was initiated at a median age of 14 (25^th-75^th quartiles: 9–24) months, and the mean treatment duration was 71.6±38.2 months (min-max: 7–162). Initial dose was calculated as total monthly dose of short acting octreotide which was adjusted per blood glucose monitoring results. Mean growth velocity during treatment was 6.4±1.1 cm/year with no concern of negative growth outcome. Neurodevelopmental delay was observed in 10 patients (43.4%). Apart from gallstones in three patients and mild, transiently elevated liver enzyme levels in six patients, no serious treatment-related side effects were observed.

Conclusion

In our cohort, long-term octreotide-LAR therapy achieved normoglycemia in the majority of patients without negativegrowth outcome. Transient elevations in liver enzymes and cholelithiasis were the most frequently observed side effects, indicating a safety profile comparable to that of short-acting octreotide. These results suggested that octreotide-LAR therapy is an effective and safe treatment option for diazoxide-unresponsive patients with CHI.