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1
Precision therapy for Developmental delay, Epilepsy and Neonatal Diabetes syndrome in the era of genomics.基因组学时代发育迟缓、癫痫和新生儿糖尿病综合征的精准治疗
Med J Armed Forces India. 2024 Dec;80(Suppl 1):S337-S340. doi: 10.1016/j.mjafi.2024.01.007. Epub 2024 Feb 26.
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本文引用的文献

1
Effectiveness and safety of long-term treatment with sulfonylureas in patients with neonatal diabetes due to KCNJ11 mutations: an international cohort study.KCNJ11 基因突变致新生儿糖尿病患者磺脲类药物长期治疗的有效性和安全性:一项国际队列研究。
Lancet Diabetes Endocrinol. 2018 Aug;6(8):637-646. doi: 10.1016/S2213-8587(18)30106-2. Epub 2018 Jun 4.
2
DEND Syndrome with Heterozygous KCNJ11 Mutation Successfully Treated with Sulfonylurea.携带杂合型KCNJ11突变的DEND综合征经磺脲类药物成功治疗
J Korean Med Sci. 2017 Jun;32(6):1042-1045. doi: 10.3346/jkms.2017.32.6.1042.
3
Neonatal Diabetes and the K Channel: From Mutation to Therapy.新生儿糖尿病与钾通道:从突变到治疗
Trends Endocrinol Metab. 2017 May;28(5):377-387. doi: 10.1016/j.tem.2017.02.003. Epub 2017 Mar 3.
4
Update of mutations in the genes encoding the pancreatic beta-cell K(ATP) channel subunits Kir6.2 (KCNJ11) and sulfonylurea receptor 1 (ABCC8) in diabetes mellitus and hyperinsulinism.糖尿病和高胰岛素血症中编码胰腺β细胞K(ATP)通道亚基Kir6.2(KCNJ11)和磺脲类受体1(ABCC8)的基因突变的更新
Hum Mutat. 2009 Feb;30(2):170-80. doi: 10.1002/humu.20838.
5
A novel mutation causing DEND syndrome: a treatable channelopathy of pancreas and brain.一种导致DEND综合征的新型突变:一种可治疗的胰腺和脑部通道病。
Neurology. 2007 Sep 25;69(13):1342-9. doi: 10.1212/01.wnl.0000268488.51776.53. Epub 2007 Jul 25.
6
ATP-sensitive potassium channels--neonatal diabetes mellitus and beyond.ATP敏感性钾通道——新生儿糖尿病及其他情况
N Engl J Med. 2006 Aug 3;355(5):507-10. doi: 10.1056/NEJMe068142.
7
Switching from insulin to oral sulfonylureas in patients with diabetes due to Kir6.2 mutations.因Kir6.2突变导致糖尿病的患者从胰岛素转换为口服磺脲类药物治疗。
N Engl J Med. 2006 Aug 3;355(5):467-77. doi: 10.1056/NEJMoa061759.
8
Activating mutations in the ABCC8 gene in neonatal diabetes mellitus.新生儿糖尿病中ABCC8基因的激活突变
N Engl J Med. 2006 Aug 3;355(5):456-66. doi: 10.1056/NEJMoa055068.
9
Mutations in KCNJ11, which encodes Kir6.2, are a common cause of diabetes diagnosed in the first 6 months of life, with the phenotype determined by genotype.编码Kir6.2的KCNJ11基因突变是出生后6个月内诊断出的糖尿病的常见病因,其表型由基因型决定。
Diabetologia. 2006 Jun;49(6):1190-7. doi: 10.1007/s00125-006-0246-z. Epub 2006 Apr 12.
10
Permanent neonatal diabetes due to mutations in KCNJ11 encoding Kir6.2: patient characteristics and initial response to sulfonylurea therapy.由编码Kir6.2的KCNJ11基因突变引起的永久性新生儿糖尿病:患者特征及对磺脲类药物治疗的初始反应
Diabetes. 2004 Oct;53(10):2713-8. doi: 10.2337/diabetes.53.10.2713.

基因组学时代发育迟缓、癫痫和新生儿糖尿病综合征的精准治疗

Precision therapy for Developmental delay, Epilepsy and Neonatal Diabetes syndrome in the era of genomics.

作者信息

Badal Sachendra, Sondhi Vishal, Sannalli Kiran, Ram Mohan Karthik, Roy Shuvendu, Yadav Ashok K, Kotwal Narendra

机构信息

Pediatric Neurologist (Pediatrics), Command Hospital (Southern Command), Pune, India.

Pediatric Neurologist (Pediatrics), 92 Base Hospital, C/o.56 APO, India.

出版信息

Med J Armed Forces India. 2024 Dec;80(Suppl 1):S337-S340. doi: 10.1016/j.mjafi.2024.01.007. Epub 2024 Feb 26.

DOI:10.1016/j.mjafi.2024.01.007
PMID:39734857
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11670654/
Abstract

Neonatal diabetes mellitus is a rare disorder with prevalence of one in 400,000 live births that's defined by persistent hyperglycaemia within the first six months of life. Neonatal diabetes is heterogeneous and can be transient or permanent. Developmental delay, Epilepsy and Neonatal Diabetes (DEND) syndrome is characterised by developmental delay, epilepsy, and neonatal diabetes. The most common cause is activating mutations of KCNJ11 or ABCC8 genes, which encode Kir6.2 and SUR1 respectively. KATP channels are expressed in the brain, nerves, muscles, and pancreatic b-cells, implying an association with the neurological features observed in patients. Neonate patients with early onset/neonatal onset diabetes are often misdiagnosed as type 1 DM and do not require lifelong insulin therapy. Whenever associated with neurological features DEND syndrome should be suspected which is a channelopathy affecting pancreas and brain and is amenable to precision therapy. Oral sulfonylureas show promising results in not only attaining euglycemia, but also in controlling seizures and ameliorating developmental delay.

摘要

新生儿糖尿病是一种罕见疾病,在40万例活产婴儿中的患病率为1/400000,其定义为出生后前六个月持续高血糖。新生儿糖尿病具有异质性,可为暂时性或永久性。发育迟缓、癫痫与新生儿糖尿病(DEND)综合征的特征为发育迟缓、癫痫和新生儿糖尿病。最常见的病因是KCNJ11或ABCC8基因的激活突变,这两个基因分别编码Kir6.2和SUR1。KATP通道在脑、神经、肌肉和胰腺β细胞中表达,这表明其与患者所观察到的神经学特征有关。早发型/新生儿期发病的糖尿病新生儿患者常被误诊为1型糖尿病,且不需要终身胰岛素治疗。每当伴有神经学特征时,应怀疑DEND综合征,这是一种影响胰腺和脑的离子通道病,适合进行精准治疗。口服磺脲类药物不仅在实现血糖正常方面显示出有前景的结果,而且在控制癫痫发作和改善发育迟缓方面也有效果。