Background: Long-acting somatostatin analogues have been reported to be an effective treatment option to prevent severe hypoglycaemia in children with severe diffuse congenital hyperinsulinism (CHI). Possible side effects include gallstones, growth retardation and necrotizing enterocolitis (NEC), the latter occurring in particular cases of newborns treated with octreotide. So far only short-acting octreotide is being used in early infancy, requiring multiple injections daily or continuous infusion. Long-acting lanreotide autogel® (LAN-ATG), that has to be applied only once a month, has not been reported as a treatment option in early infancy.
Objective and hypotheses: To assess safety and efficacy of treatment with LAN-ATG in early infancy in patients with CHI, that did not sufficiently respond to diazoxide, octreotide and nutritional treatment.
Method: Off-label use of LAN-ATG (510 mg/kg body weight) in four infants with CHI without any risk factors for NEC, starting at an age of 23 months. CHI was due to homozygous K-ATP-channel mutations (n=2) and due to Beckwith-Wiedemann syndrome (n=2). Evaluation of blood glucose concentrations, incidence of hypoglycaemia and need of concomitant drugs.
Results: Mean blood glucose concentrations, 7 days before compared to 7 days after the first LAN-ATG administration increased by 13.5 mg/dl (range 715 mg/dl). Frequency of hypoglycaemia <60mg/dl decreased by 13% (range 0.127%). Glucose infusions, octreotide and glucagon treatment could be successfully stopped in all cases 320 days after first LAN-ATG injection. In all but one patient, carbohydrate intake could be reduced, by a mean of 6 g/kg body weight/d (range 1.7512.8 g/kg body weight/d). Over an treatment period of in total 26 patient-months of LAN-ATG application, no serious adverse effects occurred.
Conclusion: During treatment of CHI with LAN-ATG in early infancy no severe side effects were observed and treatment was efficacious in patients not responding to current treatment regimen as alternative to surgery.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology