Long-Term Results of Long-Acting Somatostatin Analog Therapy in Children with Congenital Hyperinsulinism

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.

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.

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.

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.