Endocrine Abstracts

Background: Lanreotide, a prolonged-release somatostatin analogue, has been used off-label for nearly a decade to treat congenital hyperinsulinism (CHI) cases resistant to diazoxide. Lanreotide’s side-effects include diarrhea, topical allergic reactions, hepatitis, gallstones, growth suppression, hypothyroidism, and gastrointestinal dysmotility. However, there are limited case reports documenting its safety in infants with CHI.

Objective: To evaluate the safety of lanreotide treatment initiated during infancy (<9 months) for managing CHI.

Methods: A longitudinal observational study was conducted at our Highly Specialized CHI Center, documenting side-effects in CHI patients treated with lanreotide from 2014 to 2023. Side-effects were defined as anomalies identified in liver function tests (LFT), abdominal ultrasound, growth parameters, thyroid function tests, or allergic reactions post-lanreotide administration.

Results: Twelve infants diagnosed with CHI started lanreotide before 9 months of age. All had KATP channelopathy (compound heterozygous or paternally inherited recessive variant), except one with a GCK pathogenic variant. Median age at lanreotide initiation was 6 months (range 3-9 months), with median follow-up age of 30 months (range 11-102 months). Acute side-effects were observed in 17% (2/12), predominantly abdominal symptoms, with 8% (1/12) experiencing topical allergic reactions at the injection site. Long-term side-effects were documented in 10 patients; 2 discontinued within 6 months due to ineffectiveness. None developed necrotizing enterocolitis (NEC) despite lanreotide initiation at 3 months. Primary side-effects included elevated liver enzymes (3 AST, 0 GGT, 1 ALT) and coagulation abnormalities in 2 patients. One patient developed gallstones. Height SDS decreased from 0.62 to -0.26 without IGF-1 suppression. No cases of central hypothyroidism were reported.

Conclusion: Long-term use of lanreotide for managing CHI was linked to liver function abnormalities and an elevated risk of gallstones. Nevertheless, discontinuation of lanreotide treatment was deemed unnecessary based on our findings. Our data endorse the safety of initiating lanreotide treatment during infancy (<9 months) for managing CHI. However, diligent monitoring of liver function tests (LFT) and growth is imperative for all patients receiving lanreotide.