Lanreotide well-tolerated in patients with congenital hyperinsulinism

Treatment with lanreotide appears safe in patients with congenital hyperinsulinism (HI), reports a study. In addition, its use leads to a longer fasting duration and a simplification of treatment regimens.

“Somatostatin analogues are used off-label as second-line treatment,” the researchers said. “[T]he long-acting somatostatin analogue, lanreotide, has been used to treat HI over the past decade.”

Individuals with HI treated with lanreotide between 2015 and 2020 at the Congenital Hyperinsulinism Center at The Children’s Hospital of Philadelphia, US, were included in this retrospective cohort study to examine the effectiveness and safety of the drug. Outcomes measured included fasting duration with plasma glucose >70 mg/dL and frequency of lanreotide-related side effects.

Lanreotide therapy lasted for a median of 28.7 months. Of the patients, 34 (63 percent) had HI due to inactivating mutations of the adenosine 5′-triphosphate (ATP) sensitive potassium channel (K_ATP-HI), and 39 percent had undergone a pancreatectomy.

Fifty-two patients received other HI therapies, of which 22 (42 percent) discontinued other treatments and switched to lanreotide alone. Fasting duration with plasma glucose >70 mg/dL went on for a significantly longer duration during therapy with lanreotide than prior to lanreotide initiation (8.6 vs 5.1 hours; p=0.001).

Of note, subcutaneous nodules (26 percent) and gallstones (11 percent) were the most common side effects.

“Congenital HI results in severe, persistent hypoglycaemia and is associated with high risk of neurodevelopmental deficits,” the researchers said. “Sixty percent of HI cases are unresponsive to diazoxide, the only Food and Drug Administration–approved drug.”