ESPE Abstracts (2016) 86 P-P1-474

Switching Patients with Congenital Hyperinsulinism from Standard Octreotide to Long-Acting Release Octreotide Preserves Blood Glucose Control and Improves Quality of Life of Their Caregivers

Claudia Pionaa, Evelina Mainesb, Laura Baggioa, Giorgia Gugelmob, Paolo Cavarzerea, Andrea Bordugob & Rossella Gaudinoa

aDivision of Pediatric Endocrinology, Pediatric Clinic, Department of Life and Reproduction Sciences, “Giambattista Rossi” Hospital, University of Verona, Piazzale Ludovico Antonio Scuro, 10, Verona, Italy; bInherited Metabolic Diseases Unit, Department of Pediatrics, Regional Centre for Newborn Screening, Diagnosis and Treatment of Inherited Metabolic Diseases and Congenital Endocrine Diseases, Azienda O, Verona, Italy

Background: Congenital hyperinsulinism (CHI; MIM #256450) is the most common cause of persistent hypoglycaemia in children. Recessive inactivating mutations in KATP channel subunits, encoded by ABCC8 and KCNJ11 genes, are the most common cause of CHI. Mutations of these genes usually cause forms of CHI which in the vast majority of patients are unresponsive to first line medical treatment with diazoxide. Multiple daily standard octreotide injections combined with diet are second line treatment and may permit to avoid near-total pancreatectomy. Treatment with long acting release (LAR) octreotide has been reported as efficient and well tolerated in children affected by CHI. Nevertheless, experience in younger patients is yet poor. We here report two 2-year-old children affected by CHI who were switched from standard octreotide to LAR octreotide treatment.

Aim: The objective was to evaluate the blood glucose control and the quality of life of the families before and after the therapy change.

Method: Standard octreotide therapy was changed to LAR octreotide therapy in 3 months. Blood glucose control was assessed by premeal glucometer measurement and by 7-day continuous glucose monitoring before and after the therapy change. Parents self-reported Paediatric Quality of life was evaluated with a questionnaire before and after the change of therapy.

Results: Blood glucose control did not show significant variations and hypoglycaemic episodes remained rare and isolated. No side effects were observed during a follow up of six months. QoL questionnaire, completed separately by patient’s mother and father, revealed a clear improvement in psychosocial health with agreement between the parents.

Conclusions: LAR octreotide therapy seems to be effective and safe also in young patients affected with CHI. Because LAR octreotide simplifies the medical care of children with CHI, it improves also the quality of life of their caregivers.