ECEESPE2025 Poster Presentations Fetal and Neonatal Endocrinology (15 abstracts)
The pharmaceutical-grade oral Glyburide = Glibenclamide suspension, AMGLIDIA, is safe and efficacious in French patients with neonatal diabetes mellitus
Ines Ben Rhaiem 1 , Jacques Beltrand 2,3,3 , Gaelle Vermillac 4 , Kariyawasam Dulanjalee 1 , Delphine Geraud 5 , Alix Besançon 1 , Cécile Godot 1 , Marianne Berdugo 6 , Adeline Alice Bonnard 7 , Hélène Cavé 8 , AMGLIDIA Study Group 1 & Michel Polak 2
1Hôpital universitaire Necker Enfants Malades, Paris, France; 2Université Paris Cité, Hôpital Universitaire Necker Enfants Malades, Pediatric endocrinology, gynecology and diabetology, Paris, France; 3Institut Cochin, Inserm U 10116, Paris, France; 4INSERM U1016, Institut Cochin, Paris, France; 5Arcachon Hospital, Pediatrics, La Teste-de-Buch, France; 6INSERM U1138, Centre de recherche des Cordeliers, Paris, France; 7Hôpital universitaire Robert-Debré, Genetics, Paris, France; 8Université Paris Cité, Hôpital Universitaire Robert Debré, Genetics, Paris, France
JOINT279
Context: Neonatal diabetes mellitus (NDM), a rare form of diabetes, is often linked to mutations in the K-ATP, KCNJ11 and ABCC8 genes or a genetic anomaly in chromosome 6q24. Management with pharmaceutical grade pediatric-adapted oral glyburide suspension (OGS), AMGLIDIA® has proven effective in restoring insulin secretion and stopping insulin injection.
Objective: This study aimed to evaluate its efficacy, effectiveness and safety.
Methods: Observational nation-wide retrospective study. A cohort of 27 patients affected by NDM in France was collected and analyzed annually over a median follow-up period of 2.7 years and up to 9 years of treatment.
Results: At baseline, the median HbA1c was 6,5% [5,8; 7,9]; 6,5% for KCNJ11 mutated patients, 6,4% for ABCC8 mutated patients, and 8.9% for the patient with chromosome 6 anomaly. All groups showed a non-significant (P=0,382) reduction in HbA1c levels with time to 6,27% in the KCNJ11 cases, 5,93% in the ABCC8 mutated cases and 7.4% in the 6q24 anomaly patient. At baseline, the overall median dose of the OGS was 0,15 mg/kg/day ranging from 0,1 to 0,185. Serious adverse events were minimal, with hypoglycemia reported in only 2 patients and diarrhea in 1.
Conclusion: A very good HbA1c was maintained, with a non-significant decrease in dose requirements over time, up to 9 years and the overall safety profile was favourable. Long-term use of AMGLIDIA® allowed an excellent metabolic control and a reassuring and manageable safety profile, making it a viable first line and long-term treatment in neonatal diabetes mellitus.
Volume 110
Article tools
My recent searches
My recently viewed abstracts
Authors
Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
© Bioscientifica 2025 |
Privacy policy |
Cookie settings
BiosciAbstracts
Bioscientifica Abstracts is the gateway to a series of products that provide a permanent, citable record of abstracts for biomedical and life science conferences.
Find out more