Busiah K, Vaivre-Douret L, Yachi C, Cavé H, Polak M
Endocrinologie, gynécologie et diabétologie pédiatrique, hôpital Necker-Enfants malades (AP-HP), affiliation IMAGINE, 149 rue de Sèvres, 75015 Paris, France; Université Paris-Descartes, Sorbonne-Paris-Cité, 12 rue de l'École-de-Médecine, 75270 Paris Cedex 06, France; Inserm U845, faculté de médecine Cochin, 24 rue du faubourg Saint-Jacques, 75014 Paris, France.
Université Paris-Descartes, Sorbonne-Paris-Cité, 12 rue de l'École-de-Médecine, 75270 Paris Cedex 06, France; Inserm UMR-S0669, université Paris-Sud, 15 rue Georges-Clemenceau, 91400 Orsay, France.
Arch Pediatr. 2013 Dec;20 Suppl 4:S117-26. doi: 10.1016/S0929-693X(13)71425-8.
Neonatal diabetes mellitus is a rare condition (1/90,000 to 1/260,000 live births) defined as mild-to-severe hyperglycemia within the first year of life. Permanent neonatal diabetes mellitus requires lifelong therapy, whereas transient form resolves early in life but may relapse later on. Two main physiopathological mechanisms may explain this disease: β cell functional impairment or absence (pancreas agenesis or β cells destruction). The main genetic causes of β cells impairment are 6q24 abnormalities and mutations in ABCC8 or KCNJ11 potassium channel (KATP channel) genes. Compared to the KATP subtype, the 6q24 subtype had specific features: developmental defects involving the heart, kidneys, or urinary tract, intrauterine growth restriction, and early diagnosis. Remission of neonatal diabetes mellitus occurred in 51% of probands at a median age of 17 weeks. Recurrence was common at pubertal age, with no difference between the 6q24 and KATP-channel groups (82% vs 86%, p=0.36, respectively). Patients with mutations in ABCC8 or KCNJ11 genes had developmental delay with or without epilepsy but also developmental coordination disorder (particularly visual-spatial dyspraxia) or attention deficits in all of those who underwent in-depth neuropsychomotor investigations.
新生儿糖尿病是一种罕见病症(发病率为1/90,000至1/260,000活产儿),定义为出生后第一年内出现轻至重度高血糖。永久性新生儿糖尿病需要终身治疗,而暂时性新生儿糖尿病在生命早期可缓解,但后期可能复发。两种主要的生理病理机制可解释这种疾病:β细胞功能障碍或缺失(胰腺发育不全或β细胞破坏)。β细胞功能障碍的主要遗传原因是6q24异常以及ABCC8或KCNJ11钾通道(KATP通道)基因突变。与KATP亚型相比,6q24亚型具有特定特征:涉及心脏、肾脏或泌尿道的发育缺陷、宫内生长受限以及早期诊断。51%的先证者在17周龄时出现新生儿糖尿病缓解。复发在青春期很常见,6q24组和KATP通道组之间无差异(分别为82%和86%,p = 0.36)。ABCC8或KCNJ11基因突变的患者存在发育迟缓,伴或不伴有癫痫,而且在所有接受深入神经心理运动检查的患者中还存在发育协调障碍(尤其是视觉空间失用症)或注意力缺陷。