Early treatment of neonatal diabetes with oral glibenclamide in an extremely preterm infant.

Autor:	Galderisi A; Hôpital Universitaire Necker-Enfants Malades, Service d'endocrinologie Gynécologie et Diabétologie Pédiatrique Hôpital Necker-Enfants Malades Paris France.; Department of Woman and Child's Health University of Padova Padova Italy., Kermorvant-Duchemin E; Department of Neonatal Medicine Hôpital Universitaire - Enfants Malades, Université Paris Cité Paris France.; Inserm, Centre de Recherche des Cordeliers, Sorbonne University Paris Cité University, Physiopathology of Ocular Diseases: Therapeutic Innovations Paris France., Daruich A; Inserm, Centre de Recherche des Cordeliers, Sorbonne University Paris Cité University, Physiopathology of Ocular Diseases: Therapeutic Innovations Paris France.; Ophthalmology Department Necker-Enfants Malades University Hospital, Assistance Publique Hôpitaux de Paris, Paris Cité University Paris France., Bonnard AA; Département de Génétique Hôpital Universitaire Robert Debré Paris France.; INSERM UMR_S1131 - Institut de Recherche Saint-Louis Paris France., Lapillonne A; Hôpital Universitaire Necker-Enfants Malades, Service de Pédiatrie et Réanimation Néonatales Université Paris Cité Paris France., Aubelle MS; Neonatal Intensive Care Unit of Port-Royal APHP. Centre - Université Paris Cité, APHP Paris France., Perrella B; Neonatal Intensive Care Unit of Port-Royal APHP. Centre - Université Paris Cité, APHP Paris France., Vial Y; Département de Génétique Hôpital Universitaire Robert Debré Paris France.; INSERM UMR_S1131 - Institut de Recherche Saint-Louis Paris France., Cave H; Département de Génétique Hôpital Universitaire Robert Debré Paris France.; INSERM UMR_S1131 - Institut de Recherche Saint-Louis Paris France., Berdugo M; Inserm, Centre de Recherche des Cordeliers, Sorbonne University Paris Cité University, Physiopathology of Ocular Diseases: Therapeutic Innovations Paris France., Jarreau PH; Neonatal Intensive Care Unit of Port-Royal APHP. Centre - Université Paris Cité, APHP Paris France., Polak M; Hôpital Universitaire Necker-Enfants Malades, Service d'endocrinologie Gynécologie et Diabétologie Pédiatrique Hôpital Necker-Enfants Malades Paris France.; Institut IMAGINE, INSERM U1163 Paris France.; Institut Cochin INSERM U1016 Paris France.; Centre des maladies endocriniennes rares de la croissance et du développement Hôpital universitaire Necker-Enfants malades Paris France., Beltrand J; Hôpital Universitaire Necker-Enfants Malades, Service d'endocrinologie Gynécologie et Diabétologie Pédiatrique Hôpital Necker-Enfants Malades Paris France.; Institut Cochin INSERM U1016 Paris France.; Centre des maladies endocriniennes rares de la croissance et du développement Hôpital universitaire Necker-Enfants malades Paris France.
Jazyk:	angličtina
Zdroj:	JIMD reports [JIMD Rep] 2023 Jan 29; Vol. 64 (2), pp. 161-166. Date of Electronic Publication: 2023 Jan 29 (Print Publication: 2023).
DOI:	10.1002/jmd2.12358
Abstrakt:	Early treatment of neonatal diabetes with sulfonylureas has been proven to produce marked improvements of neurodevelopment, beside the demonstrated efficacy on glycemic control. Several barriers still prevent an early treatment in preterm babies including the limited availability of suitable galenic form of glibenclamide. We adopted oral glibenclamide suspension (Amglidia) for the early treatment of neonatal diabetes due to an homozygous variant of KCNJ11 gene c.10C>T [p.Arg4Cys] in an extremely preterm infant born at 26 + 2 weeks' of gestational age. After ~6 weeks of insulin treatment with a low glucose intake (4.5 g/kg/day), the infant was switched to Amglidia 6 mg/ml diluted in maternal milk, via nasogastric tube (0.2 mg/kg/day) progressively reduced to 0.01 mg/kg/day (after ~3 months). While on glibenclamide, the patient exhibited a mean daily growth of 11 g/kg/day. The treatment was suspended at month 6 of birth (weight 4.9 kg [5th-10th centile], M3 of c.a.) for normalization of glucose profile. During the treatment, the patient exhibited a stable glucose profile within the range of 4-8 mmol/L in the absence of hypo or hyperglycemic episodes with 2-3 blood glucose tests per day. The patient was diagnosed with retinopathy of prematurity Stade II in Zone II without plus disease at 32 weeks, with progressive regression and complete retinal vascularization at 6 months of birth. Amglidia could be regarded as the specific treatment for neonatal diabetes even in preterm babies due to its beneficial effect on the metabolic and neurodevelopmental side. Competing Interests: Michel Polak has been the scientific advisor for the AMGLIDIA development, none for the others. The other authors declare that they have no conflict of interest. (© 2023 The Authors. JIMD Reports published by John Wiley & Sons Ltd on behalf of SSIEM.)
Databáze:	MEDLINE
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