Context: Neonatal diabetes mellitus (NDM) is a rare condition usually related to an identifiable genetic cause. Sulfonylurea therapy can ensure metabolic control, obviating the need for insulin while also improving neurodevelopmental outcomes. An oral glyburide suspension (OGS; AMGLIDIA) designed for pediatric use was introduced recently to eliminate the drawbacks of using crushed tablets.
Objective: To evaluate the long-term effectiveness and safety of the OGS for NDM.
Design: Retrospective cohort study.
Setting: Fifteen centers in France.
Patients: Consecutive patients started on OGS for NDM in 2015 through 2024.
Intervention: OGS therapy.
Main outcome measures: Glycated hemoglobin (HbA1c) values during OGS therapy; growth; and serious adverse events.
Results: Of 27 patients, 22 had KCNJ11 mutations, 4 had ABCC8 mutations, and 1 had a 6q24 anomaly. Median follow-up during OGS therapy was 2.7 years (range, 2 months-9 years). At baseline, median HbA1c was 6.5% (5.8-7.9) overall and 6.5%, 6.4%, and 8.9% in the KCNJ11, ABCC8, and 6q24 subgroups, respectively. The median starting glyburide dose was 0.15 mg/kg/day (range, 0.1-0.185). HbA1c decreased nonsignificantly over time in all subgroups (P = .382), prompting in a small OGS dosage decrease. The last recorded HbA1c value was 6.3%, 5.9%, and 7.4% in the KCNJ11, ABCC8, and 6q24 subgroups, respectively. Serious adverse events were rare, with hypoglycemia in 2 patients during periods of decreased food intake and diarrhea in 1 patient.
Conclusion: The OGS was effective in maintaining excellent metabolic control in the long term. The safety profile was good. The OGS should be considered for the first-line treatment of NDM.
Keywords: AMGLIDIA; K-ATP channels; long-term efficacy; neonatal diabetes mellitus; pediatric oral glyburide suspension; safety.
© The Author(s) 2025. Published by Oxford University Press on behalf of the Endocrine Society.