Continuous Glucose Monitoring-Derived Glycemic Phenotyping of Childhood Hypoglycemia due to Hyperinsulinism: A Year-long Prospective Nationwide Observational Study.

Autor:	Worth C; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK.; Department of Computer Science, University of Manchester, Manchester, UK., Auckburally S; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK.; Faculty of Health and Medicine, Lancaster University, Lancaster, UK., Worthington S; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK., Ahmad S; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK., O'Shea E; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK., Senniappan S; Department of Paediatric Endocrinology, Alder Hey Children's Hospital, Liverpool, UK., Shaikh G; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, UK., Dastamani A; Department of Paediatric Endocrinology, Great Ormond Street Hospital, London, UK., Ferrara-Cook C; Crinetics Pharmaceuticals, San Diego, CA, USA., Betz S; Crinetics Pharmaceuticals, San Diego, CA, USA., Salomon-Estebanez M; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK., Banerjee I; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK.; Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
Jazyk:	angličtina
Zdroj:	Journal of diabetes science and technology [J Diabetes Sci Technol] 2024 Nov 20, pp. 19322968241255842. Date of Electronic Publication: 2024 Nov 20.
DOI:	10.1177/19322968241255842
Abstrakt:	Background: The glycemic characterization of congenital hyperinsulinism (HI), a rare disease causing severe hypoglycemia in childhood, is incomplete. Continuous glucose monitoring (CGM) offers deep glycemic phenotyping to understand disease burden and individualize patient care. Typically, CGM has been restricted to severe HI only, with performance being described in short-term, retrospective studies. We have described CGM-derived phenotyping in a prospective, unselected national cohort providing comprehensive baseline information for future therapeutic trials. Methods: Glycemic frequency and trends, point accuracy, and patient experiences were drawn from a prospective, nationwide, observational study of unselected patients with persistent HI using the Dexcom G6 CGM device for 12 months as an additional monitoring tool alongside standard of care self- monitoring blood glucose (SMBG). Findings: Among 45 patients with HI, mean age was six years and 53% carried a genetic diagnosis. Data confirmed higher risk of early morning (03:00-07:00 h) hypoglycemia throughout the study period and demonstrated no longitudinal reduction in hypoglycemia with CGM use. Device accuracy was suboptimal; 17 500 glucose levels paired with SMBG demonstrated mean absolute relative difference (MARD) 25% and hypoglycemia detection of 40%. Patient/parent dissatisfaction with CGM was high; 50% of patients discontinued use, citing inaccuracy and pain. However, qualitative feedback was also positive and families reported improved understanding of glycemic patterns to inform changes in behavior to reduce hypoglycemia. Interpretation: This comprehensive study provides unbiased insights into glycemic frequency and long-term trends among patients with HI; such data are likely to influence and inform clinical priorities and future therapeutic trials. Competing Interests: Declaration of Conflicting InterestsThe author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: IB has received research and educational grants from Zealand, Merck, Diurnal, and Hanmi Pharmaceuticals; these grants did not have conflict of interest with the study presented herewith. At present, IB is the Chair of the Communications Committee of the European Society of Paediatric Endocrinology (ESPE), the Chair of the National Institute of Health Research (NIHR) Clinical Studies Group (CSG) for the British Society of Paediatric Endocrinology and Diabetes (BSPED), and a member of the Rare Disease Advisory Group (RDAG) in NHS England. He has advised the National Institute of Health and Care Excellence (NICE) on the development of Head Injury guidelines and has advisory roles with the following patient organizations: Congenital Hyperinsulinism International, Congenital Hyperinsulinism Charity, Pituitary Foundation, and Living with Congenital Adrenal Hyperplasia. MS-E is the national clinical lead for pediatric endocrinology in NHS England. She has received research funding from Zealand and Hanmi Pharmaceuticals. MS-E has advisory roles with Congenital Hyperinsulinism International and Congenital Hyperinsulinism Charity. AD has received research funding from Resolute and Hanmi and Zealand Pharmaceuticals. She has advisory roles with Congenital Hyperinsulinism International and Congenital Hyperinsulinism Charity. SS has received research funding from Zealand Pharmaceuticals. He has advisory roles with Congenital Hyperinsulinism International and Congenital Hyperinsulinism Charity. CW is the national trainee lead for the children’s specialty group within the NIHR and has advisory roles with Congenital Hyperinsulinism International and Congenital Hyperinsulinism Charity. SB is a founder and shareholder of Crinetics Pharmaceuticals and is a Board Observer for Radionetics Oncology and Cellular Longevity. CFC is Senior Director of Clinical Research at Crinetics Pharmaceuticals. EOS, SW, SHA, and SA declare no potential conflicts of interest.
Databáze:	MEDLINE
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