ESPE Abstracts (2015) 84 P-2-493

aUniversity College London Institute of Child Health, London, UK; bGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK; cAlder Hey Children’s Hospital NHS Trust, Liverpool, UK; dKymos Pharma Services, Barcelona, Spain

Background: Congenital hyperinsulinism (CHI) causes severe hypoglycaemia in children. Diazoxide and daily octreotide injections are first and second-line of treatment for CHI respectively. Diazoxide can cause severe hypertrichosis resulting in parental anxiety and compliance issues.

Objective and hypotheses: To evaluate the efficacy, safety and pharmacokinetics of Lanreotide therapy in CHI patients.

Method: Patients >6 months of age either on high dose diazoxide (causing side effects), or daily octreotide were started on 30 mg Lanreotide 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 were collected at times 0,+1,+2,+4,+24 and +96 hours post 1st dose and subsequently prior to each dose for 6 months.

Results: 29 children were commenced on Lanreotide and three had to stop treatment. 18/26 were on daily octreotide and eight on diazoxide. Five children have stopped overnight continuous feeds. Out of 26 children, 22 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 1st 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 lanreotide is safe and effective 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.

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