Efficacy of octreotide in 26 cases of congenital hyperinsulinism

Abstract

Objective To analyze the efficacy and safety of octreotide treatment in patients with congenital hyperinsulinism(CHI). Methods Twenty-six patients with CHI hospitalized in Beijing Children's Hospital from February 2002 to January 2016 who received octreotide treatment were selected as the research subjects. The efficacy, side effects and prognosis of octreotide treatment of congenital hyperinsulinism in the 26 patients were analyzed retrospectively. Results In 26 patients, 16 cases(61.4%) showed good response to octreotide and 5 patients(19.2%) were unresponsive to octreotide, and the effect of octreotide in the other 5 cases (19.2%) was not clear. Seven cases of 16 patients who were responsive to octreotide received detection of CHI-related genes, and six infants were found carrying ABCC8 gene mutations. Fourteen patients had tried diazoxide treatment, 1 case had good response and 8 cases showed no response, the efficacy in the other 5 cases was not clear. Four cases received 18F-L-Dopa-positron emission tomography(PET) scan of pancreas, and three of them were found with focal type CHI and were cured with partial pancreatectomy. After application of octreotide, varying degrees of side effects were recorded in 9 patients. Conclusions Octreotide has good therapeutic effect for poorly-controlled CHI with diazoxide. Children with CHI poorly-controlled with diazoxide should undergo the pancreas 18F-L-Dopa-PET scan to confirm the histological type of the CHI, and different levels of pancreatectomy should be performed when needed. Key words: Octreotide; Diazoxide; Congenital hyperinsulinism; Treatment outcome