ESPE Abstracts (2021) 94 P1-25

1Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom; 2Department of Paediatrics, Nizwa Hospital, Nizwa, Oman; 3Department of Pediatrics, Division of Endocrinology, Jim Pattison Children’s Hospital, Saskatoon, Canada; 4Dept of Mathematics, University of Manchester, Manchester, United Kingdom; 5Institute of Biomedical and Clinical Science, University of Exeter Medical School, Exeter, United Kingdom; 6Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom


Background: Hypoglycaemia due to congenital hyperinsulinism (CHI) is the commonest cause of severe, recurrent hypoglycaemia in childhood. Previous follow up studies have focused on neurodevelopmental status which is noted to be delayed in as many as 48% of cases. There has been less emphasis on other long-term outcomes in patients with CHI. Given the requirement for high volume carbohydrate in most patients, there are concerns regarding the adverse effects on weight. However, there are no long-term studies describing feeding and auxology outcomes in children with CHI. We aimed to provide a description of auxological, feeding and neurodevelopmental outcomes in a longitudinal cohort to better understand the natural history and long-term outcomes of children with CHI.

Method: We reviewed medical records for all patients with confirmed CHI over a three-year period in a single specialist centre. Notes were reviewed for follow up data on auxology, feeding problems, medications and neurodevelopmental outcomes up to five years of age. Linear interpolation was used to impute missing values of height and weight data. Non-parametric tests were performed using SPSS 25.

Results: Seventy patients were recruited to the study. Mean weight at birth was -1.0 standard deviation scores (SDS) for age and sex (-3.2 for small for gestational age (SGA) patients and +0.4 for appropriate for gestational age (AGA) patients), while mean height at three months was -1.5 SDS. Both weight and height trended to the population mean over the follow-up period and by five years of age mean weight was -0.1 SDS and height +0.1 SDS. Feeding difficulties, defined as the requirement for non-oral enteral feeding, were present in 12 patients (17%) at three months, reducing to two patients (3%) by five years. At age five years, 11 patients (15%) had neurodevelopmental delay and of these only one was severe. Earlier resolution of disease was predicted by lower maximum early diazoxide dose (P = 0.007) and being born SGA (P = 0.009) as expected.

Conclusion: We have provided the first description of auxological natural history data in a longitudinal cohort of patients with CHI. After five years, in spite of feeding difficulties and carbohydrate loading in early life, auxology parameters are reassuringly normal in follow-up. A lower than expected rate of neurodevelopmental delay could be attributed to prompt early treatment. Early maximum diazoxide dose and being born SGA can be used in discussions with families to prognosticate possible resolution of disease.

Volume 94

59th Annual ESPE (ESPE 2021 Online)

Online,
22 Sep 2021 - 26 Sep 2021

European Society for Paediatric Endocrinology 

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