Background: Sulfonylureas (SU) have proven to be effective in patients with monogenic diabetes owing to potassium channel mutation. They allow the discontinuation of insulin and a good metabolic control. Long-term data arguing for a persistent beneficial effect of SU are missing.
Objective and hypotheses: SU provide a good metabolic control maintained over time in patients with neonatal diabetes.
Method: From a French cohort of 34 patients (GENEODIA study French Neonatal Glibenclamide Study Group), we selected patients transferred successfully (no insulin during the 12 months after the switch) for more than five years (before 2010). Data recorded retrospectively: safety, diabetes complication, SU dosage and HbA1C before switch and at last follow-up visit.
Results: About 28 patients were eligible (18 boys 24 KCNJ11 mutations 4 ABCC8 mutations). Complete data before switch were available for 24 patients and after switch for 17 patients. Median time till SU start was 9.32 years (5.812.15) and median follow-up time 6.6 years (1.411.5 years). Median age at transfer from insulin to SU was 4.9 years (0.2336.5). Median HbA1C before transfer was 7.4% (5.310.3) and 6.1% (5.18.1%) at last visit. Median change was −1.4% (−4 to +0.3%), P<0.001. Median glibenclamide dosage was 0.16 mg/kg/day (0.0250.66) at last visit. Safety was good; no episodes of renal or hepatic failure and no development of retinopathy or nephropathy were reported. Insulin was re introduced permanently in 1 patient (3 years after SU transfer) and transiently in another (1 year after transfer and during 4 years).
Conclusion: While prescribed off label, SU display a beneficial metabolic effect maintained over time with an extremely good safety profile in patients with neonatal diabetes owing to potassium channel mutation.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology