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. 2025 May 9;9(7):bvaf083.
doi: 10.1210/jendso/bvaf083. eCollection 2025 Jul.

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

Ines Ben Rhaiem  1 , Jacques Beltrand  1   2   3   4 , Gaëlle Vermillac  1   3 , Dulanjalee Kariyawasam  1   2   3   4 , Delphine Geraud  5 , Alix Besançon  1 , Cécile Godot  1 , Marianne Berdugo  2   6 , Adeline Alice Bonnard  2   6 , Hélène Cavé  2   7 , Sylvie Nivot-Adamiak  8 , Marie Béatrice Saade  8 , Virginie Ribault  9 , Sabine Baron  10 , Cécile Petit-Bibal  11 , Fabienne Dalla-Vale  12 , Karine Bourdet  13 , Carole Morin  14 , Aurélie Donzeau  15 , Iva Gueorguieva  16 , Pierre-François Souchon  17 , Marc Nicolino  18 , Pascal Barat  19 , Régis Coutant  15 , Kevin Perge  18 , Michel Polak  1   2   3   4
Affiliations

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

Ines Ben Rhaiem et al. J Endocr Soc. .

Abstract

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.

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Figures

Figure 1.
Figure 1.
Patient flowchart.
Figure 2.
Figure 2.
Neuropsychiatric and neuropsychological features in 13 of the 27 patients.
Figure 3.
Figure 3.
Changes in HbA1c values and oral glyburide suspension doses over time. Numbers of patients with available data each year of follow-up.
Figure 4.
Figure 4.
Successive treatments in each patient; the data were recorded at 6-month intervals. Blue: insulin; purple: crushed glyburide tablets; orange: oral glyburide suspension. Insulin was used at diagnosis in 20 (74%) patients, in a median dose of 0.6 IU/kg/day for a median of 3 months. In 13 (48%) patients, crushed glyburide tablets (Daonil or Hemi-Daonil) were given off-label, in a median dose of 0.35 mg/kg/day for a median of 3 months. A combination of insulin and crushed glyburide tablets was used in 8 (29%) patients. All patients eventually received the oral glyburide suspension; this was available as the initial treatment in only 2 patients.
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