New advances in the treatment of neonatal diabetes mellitus with sulfonylureas

HU Xiaoyan; XIANG Jinbo; ZHU Xiaoxia; LI Zheng; CAO Tingting; DING Ting; XU Ziran; LI Jingbo; YANG Youjun

doi:10.6039/j.issn.1001-0408.2026.09.22

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New advances in the treatment of neonatal diabetes mellitus with sulfonylureas

更新时间：2026-05-14

- New advances in the treatment of neonatal diabetes mellitus with sulfonylureas
- ZHONGGUO YAOFANG Vol. 37, Issue 9, Pages: 1236-1240(2026)
- 作者机构：
  
  三峡大学附属仁和医院儿科，湖北宜昌　443001
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2026.09.22
  CLC： R977.15;R587.1
- Received：22 January 2026，
  
  Revised：2026-03-31，
  
  Accepted：02 April 2026，
  
  Published：15 May 2026
- 稿件说明：
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胡小燕,向金波,朱晓霞,等.磺酰脲类药物治疗新生儿糖尿病的研究新进展 [J].中国药房,2026,37(09):1236-1240.

HU Xiaoyan,XIANG Jinbo,ZHU Xiaoxia,et al.New advances in the treatment of neonatal diabetes mellitus with sulfonylureas[J].ZHONGGUO YAOFANG,2026,37(09):1236-1240.
胡小燕,向金波,朱晓霞,等.磺酰脲类药物治疗新生儿糖尿病的研究新进展 [J].中国药房,2026,37(09):1236-1240. DOI： 10.6039/j.issn.1001-0408.2026.09.22.

HU Xiaoyan,XIANG Jinbo,ZHU Xiaoxia,et al.New advances in the treatment of neonatal diabetes mellitus with sulfonylureas[J].ZHONGGUO YAOFANG,2026,37(09):1236-1240. DOI： 10.6039/j.issn.1001-0408.2026.09.22.

摘要

新生儿糖尿病（NDM）是一种罕见的单基因遗传病，主要由

KCNJ11

和

ABCC8

基因突变导致胰岛素分泌不足引起。磺酰脲类药物（以格列本脲为代表）通过精准关闭突变的胰岛β细胞ATP敏感性钾通道，恢复胰岛素分泌，已成为治疗NDM的标准疗法。临床研究证实，磺酰脲类药物可使超过90%的患者成功从终身胰岛素注射治疗成功转换为口服药物治疗，从而实现长期稳定的血糖控制，并能一定程度地改善神经系统预后。在安全性方面，磺酰脲类药物出现严重低血糖事件较为罕见，胃肠道反应轻微，长期治疗耐受性良好，且对儿童生长发育无不利影响。未来通过进一步完善“快速基因诊断-早期干预-研发专用剂型-长期随访”的全链条管理路径，磺酰脲类药物的临床应用有望为NDM患者提供最优化的治疗方案和最大化的健康获益。

Abstract

Neonatal diabetes mellitus （NDM） is a rare monogenic disorder primarily caused by insufficient insulin secretion resulting from mutations in the

KCNJ11

and

ABCC8

genes. Sulfonylureas， represented by glibenclamide， have become the standard therapy for this type of NDM by precisely closing the mutated ATP-sensitive potassium channels in pancreatic β cells， thereby restoring insulin secretion. Clinical studies confirm that sulfonylureas enable over 90% of patients to successfully transition from insulin to oral treatment， achieving long-term stable glycemic control and improving neurological outcomes to a certain extent. In terms of safety， severe hypoglycemia induced by sulfonylureas is relatively rare and gastrointestinal reactions are mild； moreover， sulfonylureas show good long-term tolerability， and have no adverse effects on child growth and development. In the future， by further refining the full-chain management pathway of “rapid genetic diagnosis-early intervention-specialized dosage forms-long-term follow-up”， the clinical application of sulfonylureas is expected to provide NDM patients with an optimized treatment regimen and maximize their health benefits.

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references

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