Long-Term Follow-Up of Children With Congenital Hyperinsulinism on Octreotide Therapy

Context: Octreotide, a somatostatin analog, is commonly used in diazoxide unresponsive congenital hyperinsulinism (CHI) patients as a second-line therapy. Objective: The aims of this study were to evaluate the dose range, side effects, and long-term follow-up of octreotide therapy in a large cohort of CHI patients. Setting: The study was conducted at an international referral center for the management of CHI. Patients: Twenty-eight (17 males) diazoxide unresponsive CHI patients (15 biallelic and 10 monoallelic ATP sensitive potassium channel mutation) managed with daily multidose octreotide therapy between 2001 and 2013 participated in the study. Main Outcome Measures: Regular follow-up of auxology, growth factors (serum IGF-1 and IGF binding protein 3 levels), thyroid functions, liver function tests, and hepatobiliary ultrasonography were measured. Results: The median age of CHI diagnosis was 1 week (range 1-80 wk). The mean (+/- SD) dose of octreotide required was 17.8 (+/- 7.5) mu g/kg.d (range 7.5-30 mu g/kg.d). The mean (+/- SD) duration of follow-up on octreotide therapy was 52.4 (+/- 33.8) months (range 6 mo to 9.5 y). Elevation of liver enzymes was the most prevalent side effect (n = 13; 46.4%), which resolved spontaneously. Gallbladder pathology was detected in nine patients (32%). Mean (+/- SD) duration of octreotide therapy before the development of gallbladder pathology was 4.3 (+/- 4.6 mo), whereas 19 patients were free of gallstones after a follow-up of 53.6 +/- 32.9 months on octreotide therapy. There was no relationship between the dose or the duration of octreotide therapy and development of gallbladder pathology or liver dysfunction. Conclusions: Transient elevation of liver enzymes and asymptomatic gallbladder pathology were the most prevalent long-term side effects of octreotide therapy. There was no correlation between the dose or the duration of octreotide therapy and development of liver dysfunction and gallbladder pathology.