Ille Jasenka, Putarek Natasa Rojnić, Radica Ana, Hattersley Andrew, Ellard Sian, Dumić Miroslav
Klinika za pedijatriju Medicinskog fakulteta Sveucilista u Zagrebu, KBC Zagreb.
Lijec Vjesn. 2010 Mar-Apr;132(3-4):90-3.
Neonatal diabetes mellitus is a rare metabolic disorder with an estimated incidence of 1:300.000 to 400.000 newborns, and less than 50% of the neonates have permanent neonatal diabetes mellitus (PNDM). Recently, activating mutation in the KCNJ11 gene encoding Kir6.2 subunit of the adenosin triphosphate-sensitive potassium (K(ATP)) channel has been described as the most frequent cause of PNDM. Under physiological circumstances K(ATP) channel closure plays a central role in glucose-stimulated insulin secretion from pancreatic beta cells. Sulphonylurea drugs stimulate insulin secretion by binding to and closing K(ATP) channels and thus bypassing beta cell metabolism stimulate the same chain of reactions as glucose. We describe a boy diagnosed with PNDM at the age of 3 months when insulin therapy was started, and at the age of 4.5 years KCNJ11 gene was sequenced and found that the boy carried a de novo activating R201H mutation. Insulin therapy was successfully switched to low doses of oral glibenclamide. Accordingly, it is important to emphasize that every person diagnosed with diabetes before six months of life, however old they actually are, should be tested for K(ATP) mutations which is offered via the website www.diabetesgenes.org.
新生儿糖尿病是一种罕见的代谢紊乱疾病,估计发病率为1:300000至400000新生儿,且不到50%的新生儿患有永久性新生儿糖尿病(PNDM)。最近,编码三磷酸腺苷敏感性钾(K(ATP))通道Kir6.2亚基的KCNJ11基因激活突变被描述为PNDM最常见的病因。在生理情况下,K(ATP)通道关闭在胰腺β细胞葡萄糖刺激的胰岛素分泌中起核心作用。磺脲类药物通过与K(ATP)通道结合并使其关闭来刺激胰岛素分泌,从而绕过β细胞代谢,刺激与葡萄糖相同的反应链。我们描述了一名3个月大开始胰岛素治疗的男孩,4.5岁时对KCNJ11基因进行测序,发现该男孩携带一种新发的激活型R201H突变。胰岛素治疗成功转换为低剂量口服格列本脲。因此,重要的是要强调,每个在6个月前被诊断为糖尿病的人,无论其实际年龄多大,都应该通过网站www.diabetesgenes.org进行K(ATP)突变检测。