ESPE Abstracts (2016) 86 FC5.3

Glibentek, a New Suspension of Glibenclamide for Patients with Neonatal Diabetes, is as Effective and more Convenient than Crushed Tablets

Jacques Beltranda,b, Cécile Godota, Kanetee Busiaha,b, Zoubir Djeradac, Sabine Barond, Claire Le Tallece, Raphael Tessierf, Virginie Ribaultg, Maryse Cartignyh, Henri Brueli, Claire Gozaloc, Jean-Marc Tréluyerb,j, Caroline Eliej & Michel Polaka,b

aUnité d’endocrinologie, gynécologie et diabétologie pédiatrique, Hôpital universitaire Necker - Enfants Malades, Assistance Publique Hôpitaux de Paris, Paris, France; bFaculté de Médecine Paris Descartes, Université Sorbonne Paris Cité, Paris, France; cLaboratoire de Pharmacologie -Toxicologie, Hôpital Maison Blanche, CHU Reims, Reims, France; dService de pédiatrie, CHU Nantes, Nantes, France; ePédiatrie, diabétologie, CHU Toulouse Purpan, Toulouse, France; fService de pédiatrie, CHU Brest, Brest, France; gService de pédiatrie médicale, CHU Caen, Caen, France; hService de pédiatrie, Hôpital Jeanne De Flandres, Lille, France; iService de pédiatrie, Hôpital Jacques Monod, Montivilliers, France; jUnité de recherche clinique, centre d’investigation Clinique, Hôpital universitaire Necker Enfants Malades Paris, Assistance Publique-Hôpitaux de Paris, Paris, France

Background: Glibenclamide has proven to be efficient for patients with neonatal diabetes owing to potassium channel mutations. Anyway its pharmaceutical form is not suitable for young children or infants. The tablets dosage is too high for most infants and must be crushed and diluted before administration. We developed a suspension of glibenclamide (EMA Orphean drug designation january 2016) fitting recommendations of drug administration to allow a precise dosage and designed to be more practical for patients and their families.

Objective and hypotheses: To determine if a suspension of glibenclamide is more suitable and as efficient as tablets in children with neonatal diabetes.

Method: Patients where switched from crushed tablets to suspension during 3 months. Acceptability and efficiency were measured (self administrated questionnaires, hedonic scales and HbA1C) and compared to tablets ones. Adverse events were recorded.

Results: 10 patients (6 boys) with KCNJ11 mutation, median age 2.7 years (0.3–16.2), median duration of glibenclamide therapy 2.3 years (0.01–11.3). Switch from glibenclamide tablets to glibentek didn’t affect metabolic control (Median change in HbA1C: −0.35%, −1.3 to 0). Median dosage was 0.25 mg/k/day (0.075–0.77) before the switch and 0.12 mg/kg/day (0.06–0.56) 3 months after. 100% of parents were satisfied with the suspension (vs 50% for tablets). 60% reported administration difficulties with the tablets but none with the suspension. Infants and young children were more satisfied by the suspension than teenagers that could swallow the whole tablet. Safety record was excellent: no severe hypoglycaemia, sporadic abominal pains in one patient, transient rise in hepatic transaminases in another. Parents from infants and young children (n=5) preferred keeping the suspension than going back to tablets.

Conclusion: Glibenclamide suspension is as effective as tablets and more practical for infants and young children with neonatal diabetes. ( NCT02375828).