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Endocrine Abstracts (2015) 39 P4 | DOI: 10.1530/endoabs.39.P4

1UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK; 2Great Ormond Street Hospital for Children, London, UK; 3Alder Hey Childrens Hospital, Liverpool, UK; 4Kymos Pharma Services, Barcelona, Spain.


Background: CHI is a cause of severe hypoglycaemia in children. Diazoxide (KATP channel agonist) is used as first-line treatment but is known to cause severe hypertrichosis and reduced appetite in children. Diazoxide unresponsive CHI us treated with daily octreotide subcutaneous injections (3–4 times/day).

Objective and hypotheses: To evaluate the efficacy, safety and pharmacokinetics of long acting Somatostatin analogue (Lanreotide) therapy in CHI patients.

Method: Children with CHI >6 months of age either on high dose diazoxide (causing side effects), or daily octreotide were started on 30 mg Lanreotide administered every 4-weeks. Children >3 years of age had paediatric quality of life (PedsQL) with strengths and difficulties questionnaires (SDQ) pre- and 1-year post-Lanreotide. Plasma Lanreotide concentrations measured by radioimmunoassay collected at different time points after administration and subsequently prior to each dose for 6 months.

Results: 30 children were commenced on Lanreotide and three had to stop treatment. Out of 27 children, 19 were on daily octreotide injections and eight on diazoxide. Five children have stopped overnight continuous feeds. 23 children had diffuse hyperinsulinism, three were protein sensitive and one had focal lesion (had three surgeries). Pharmacokinetic data on 21 children showed highest median value (25th–75th interquartile range) of Lanreotide concentration was 14.93 ng/ml (4.39–31.6) at +4 h of first dose. The median values (25th–75th interquartile range) prior to 2nd, 3rd, 4th, 5th, 6th and 12th doses were 0.88 ng/ml (0.66–1.32), 1.09 ng/ml (0.89–1.35), 1.21 ng/ml (0.87–1.49), 0.79 ng/ml (0.67–1.55), 1.35 ng/ml (1.19–1.86) and 1.44 ng/ml (1.08–2.18) respectively. PedsQL showed significant change in total health and psychosocial score and significant reduction in overall stress in the SDQ after 1-year post-Lanreotide (P<0.05).

Conclusion: This study demonstrates that lanreotide can be used as an alternative to diazoxide and octreotide therapy in CHI patients with a significant improvement in blood glucose control and quality of life. There is cumulative effect in lanreotide concentration after each dose. Our 2.5 years follow-up data shows no adverse effects on growth.

Volume 39

43rd Meeting of the British Society for Paediatric Endocrinology and Diabetes

British Society for Paediatric Endocrinology and Diabetes 

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