The Use of Lanreotide in the Treatment of Congenital Hyperinsulinism.

Context: Congenital hyperinsulinism (HI) results in severe, persistent hypoglycemia and is associated with high risk of neurodevelopmental deficits. Sixty percent of HI cases are unresponsive to diazoxide, the only Food and Drug Administration-approved drug. Somatostatin analogs are used off-label as second-line treatment; the long-acting somatostatin analogue, lanreotide, has been used to treat HI over the past decade. Existing reports are limited to small case series.

Objective: To assess the effectiveness and safety of lanreotide in individuals with HI.

Design: Retrospective cohort study of individuals with HI treated with lanreotide between 2015 and 2020.

Setting: The Congenital Hyperinsulinism Center at The Children's Hospital of Philadelphia.

Patients: Fifty-four individuals with hyperinsulinism treated with lanreotide.

Main Outcome Measures: Fasting duration with plasma glucose > 70 mg/dL; frequency of lanreotide-associated side effects.

Results: The median duration of lanreotide therapy was 28.7 (2.8-64.5) months. Thirty-four patients (63%) had HI due to inactivating mutations of the adenosine 5'-triphosphate (ATP) sensitive potassium channel (KATP-HI), and 39% had undergone a pancreatectomy. Of 52 patients receiving other HI therapies, 22 (42%) were able to discontinue other treatments and were managed on lanreotide alone. Fasting duration with plasma glucose > 70 mg/dL was significantly longer during therapy with lanreotide compared to prior to lanreotide initiation (8.6 ± 6.5 vs 5.1 ± 4.7 hours, P = 0.001). The most common side effects were subcutaneous nodules (26%) and gallstones (11%).

Conclusions: Lanreotide is a well-tolerated treatment for patients with HI. It results in a longer duration of fasting and a simplification of treatment regimens.