Background: Octreotide, a long-acting somatostatin analogue, is commonly used in diazoxide unresponsive congenital hyperinsulinism (CHI) patients as a second line medication. However, there are no large studies evaluating long-term follow-up CHI patients on octreotide therapy.
Objective and hypotheses: To evaluate the dose range, side effects and long-term follow-up in CHI patients on daily octreotide injections.
Method: Twenty-eight (17 males and 11 females) diazoxide unresponsive CHI patients managed with daily multidose octreotide therapy between 2001 and 2013 at Great Ormond Street Hospital for Children NHS Trust were evaluated. Regular follow up of auxology, hormonal, liver function tests, and hepatobiliary ultrasonography results were reviewed from hospital notes.
Results: The median age of diagnosis was 1 week (range: 180 weeks) and the mean dose of octreotide commenced was 17.8±7.5 μg/kg per day (range: 7.533.7 μg/kg per day). The mean duration of follow-up on octreotide therapy was 52.4±33.8 months (range: 6 months9.5 years). Elevation of liver enzymes was the most prevalent side effect of octreotide therapy (n=13, 46.4%) which resolved spontaneously. Gallstone (n=6, 21.2%) and gall bladder sludge (n=3, 10.7%) were detected in nine out of 28 (32%) patients. There was no relation between the dose and duration of octreotide therapy with either elevation of liver enzyme or development of gall bladder pathology. However, the younger age for commencement of octreotide was found to be related with the higher rate of elevation in liver enzymes. GH deficiency and hypothyroidism were not detected during long-term follow-up on octreotide therapy.
Conclusion: Transient elevation of liver enzymes and asymptomatic gallbladder pathology are the most prevalent long-term side effect of octreotide therapy in children with CHI. There is no correlation between dose or duration of octreotide therapy and development of liver dysfunction and gallbladder pathology.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology