Background: Congenital hyperinsulinism (CHI) is cause of severe hypoglycaemia. Octreotide (somatostatin analogue), given as four times daily s.c. injections or via a pump, is used as second line treatment in diazoxide unresponsive CHI patients.
Objective and hypotheses: The aim of our study was to evaluate the use of a long acting somatostatin analogue (Lanreotide) in patients with CHI.
Method: Diffuse CHI patients above three years of age, on high dose diazoxide (causing side effects) or daily octreotide injections were recruited. The starting dose of Lanreotide was 30 mg 4 weekly. Blood glucose was monitored when weaning diazoxide or octreotide after the first dose of Lanreotide. Every patient had their auxology, endocrinology and ultrasound abdomen monitored. Quality of life questionnaires were also completed.
Results: 16 children (eight boys and eight girls) with CHI were recruited. The median age was 6 years (315 years). Lanreotide treatment was stopped in three children (one had atypical diffuse disease, one had profuse diarrhoea and one showed partial response and developed hypoglycaemic episodes despite increasing the dose to 60 mg). Out of the 13 patients on Lanreotide, ten were on daily octreotide injections and three on diazoxide. Seven (54%) and 5 (38%) patients came off their current treatment within 4 weeks and 812 weeks respectively after administering first Lanreotide injection. However, one patient continues to be on a small dose of diazoxide and on Lanreotide dose of 60 mg. Eight patients had mutations in ABCC8/KCNJ11. Two children have come off their continuous overnight feeds and five of them are currently being weaned. The preliminary data on the quality of life suggests that both parents and children are very satisfied with the response.
Conclusion: Lanreotide is safe and an effective alternative to octreotide/diazoxide therapy in patients with diffuse CHI, offering an improved quality of life. However, some children may not show complete response and monitoring of side effects is equally important. Also, long-term follow-up is necessary in this group of children.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology