Safety and Effectiveness of Oral Glyburide Suspension in Neonatal Diabetes Mellitus: French Retrospective Cohort Study.

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 mutations, 4 had 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 , , 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 ( = .382), prompting in a small OGS dosage decrease. The last recorded HbA1c value was 6.3%, 5.9%, and 7.4% in the , , 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.