Glibenclamide oral suspension: Suitable and effective in patients with neonatal diabetes.
Beltrand J,Baptiste A,Busiah K,Bouazza N,Godot C,Boucheron A,Djerada Z,Gozalo C,Berdugo M,Tréluyer J,Elie C,Polak M
Source :
Pediatr Diabetes
2019 Fév 21
Pmid / DOI:
30684309
Abstract
BACKGROUNDResults of genetic have led to off-label glibenclamide treatment in patients with neonatal diabetes (NDM) because of potassium channel mutations. No pediatric form of glibenclamide was available. Glibenclamide was designated an orphan drug designation for NDM and a suspension was developed. As a part of the pediatric plan investigation, we assessed its acceptability, efficiency, and safety.METHODSIn this Phase II, prospective, non-randomized, single-center study, patient received glibenclamide tablets for 1 month then the suspension for 3 months. We assessed acceptability using hedonic scales and patient questionnaires, effectiveness using glycated hemoglobin (HbA1C) assays and safety based on hypo and hyperglycemia, and other adverse events.RESULTSWe included 10 patients (0.1-16.2 years, 6