Janchevska A, Tasic V, Gucev Z, Krstevska-Konstantinova M, Cheong H I
University Children's Hospital Skopje, Medical Faculty, Skopje, Macedonia.
Seoul National University Children's Hospital, Seoul, Korea ; Research Coordination Center for Rare Diseases, Seoul National University Hospital, Seoul, Korea ; Kidney Research Institute, Medical Research Center, Seoul National University College of Medicine, Seoul, Korea.
Balkan J Med Genet. 2015 Apr 10;17(2):81-5. doi: 10.2478/bjmg-2014-0078. eCollection 2014 Dec.
Nephrogenic diabetes insipidus (NDI) is caused by the inability of renal collecting duct cells to respond to arginine vasopressin (AVP)/antidiuretic hormone (ADH). We present the case of a 7-year-old boy with a history of excretion of large amounts of dilute urine and polydipsia since infancy. The boy had several vomiting episodes with mild dehydration during the first 3 years of life. There was no evidence of headaches, dizziness or visual problems. He drinks between 2 and 3 L/day and has 24-hour diuresis of 2 liters, now. He has prepubertal appearance with appropriate weight [+0.85 standard deviation score (SDS)] and height (+0.15 SDS) for his age. His intelligence was also normal. The water deprivation test showed low urine osmolality after 8 hours of dehydration. After desmopressin administration, urine osmolality remained low. Serum osmolality was in the normal range for sex and age before and after desmopressin administration. This indicated a nephrogenic form of diabetes insipidus. Molecular analyses revealed a P286L [p.Pro(CCC)286Leu(CTC)] mutation in the AVPR2 gene, that was inherited from his mother. This patient is the first case with genetically confirmed X-linked inherited form of NDI in the Republic of Macedonia. Molecular analysis confirmed the clinical diagnosis and enabled genetic advice for this family.
肾性尿崩症(NDI)是由肾集合管细胞无法对精氨酸加压素(AVP)/抗利尿激素(ADH)作出反应所致。我们报告一例7岁男孩,自婴儿期起就有大量稀释尿液排出和烦渴的病史。该男孩在生命的头3年中有几次呕吐发作并伴有轻度脱水。没有头痛、头晕或视觉问题的证据。他现在每天饮水2至3升,24小时尿量为2升。他具有青春期前的外貌,体重[+0.85标准差评分(SDS)]和身高(+0.15 SDS)与其年龄相符。他的智力也正常。禁水试验显示脱水8小时后尿渗透压较低。给予去氨加压素后,尿渗透压仍较低。给予去氨加压素前后血清渗透压在性别和年龄的正常范围内。这表明是肾性尿崩症。分子分析显示AVPR2基因存在P286L [p.Pro(CCC)286Leu(CTC)]突变,该突变遗传自他的母亲。该患者是马其顿共和国首例经基因证实的X连锁遗传性肾性尿崩症病例。分子分析证实了临床诊断,并为这个家庭提供了遗传咨询。