Great Ormond Street Hospital for Children, London, UK.
Nephron Physiol. 2010;116(4):p23-9. doi: 10.1159/000320117. Epub 2010 Aug 20.
BACKGROUND/AIMS: Nephrogenic diabetes insipidus (NDI) is a serious condition with large water losses in the urine and the risk of hypernatremic dehydration. Unrecognized, repeated episodes of hypernatremic dehydration can lead to permanent brain damage. Primary NDI is due to mutations in either AVPR2 or AQP2. NDI can also occur as a secondary complication, most commonly from obstructive uropathy or chronic lithium therapy. We observed NDI in patients with inherited tubulopathies and aimed to define the clinical and molecular phenotype.
We reviewed the medical notes of 4 patients with clinical NDI and an underlying molecularly confirmed diagnosis of nephropathic cystinosis, Bartter syndrome, nephronophthisis and apparent mineralocorticoid excess, respectively.
The patients all failed to concentrate their urine after administration of 1-desamino[8-D-arginine] vasopressin. None had an identifiable mutation in AVPR2 or AQP2, consistent with secondary NDI. Patients experienced repeated episodes of hypernatremic dehydration, and in 2 cases, NDI was initially thought to be the primary diagnosis, delaying recognition of the underlying problem.
The recognition of this potential complication is important as it has direct implications for clinical management. The occurrence of NDI in association with these conditions provides clues for the etiology of aquaporin deficiency.
背景/目的:肾源性尿崩症(NDI)是一种严重的疾病,会导致大量尿液流失和高钠血症脱水的风险。如果未被识别,反复发作的高钠血症脱水可能导致永久性脑损伤。原发性 NDI 是由于 AVPR2 或 AQP2 的突变引起的。NDI 也可能作为继发性并发症发生,最常见于梗阻性尿路病或慢性锂治疗。我们观察到遗传性肾小管病患者存在 NDI,并旨在定义其临床和分子表型。
我们回顾了 4 名具有临床 NDI 且分子上确诊为先天性胱氨酸贮积症、巴特综合征、肾性尿崩症和表观盐皮质激素过多症的患者的病历。
这些患者在给予 1-脱氨基[8-D-精氨酸]血管加压素后均无法浓缩尿液。没有一个患者在 AVPR2 或 AQP2 中存在可识别的突变,符合继发性 NDI。患者反复发生高钠血症脱水,其中 2 例最初被误诊为原发性 NDI,从而延迟了对潜在问题的识别。
认识到这种潜在的并发症非常重要,因为它对临床管理有直接影响。这些情况下发生的 NDI 为 aquaporin 缺乏症的病因提供了线索。