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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.

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。

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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.

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