Hawton Katherine, Galloway Louise, Harmer Matthew, Kumaran Anitha, Kharbanda Mira, Platt Caroline, Candler Toby
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, UK
University of Bristol, Bristol, UK.
Arch Dis Child Educ Pract Ed. 2025 May 16;110(3):107-112. doi: 10.1136/archdischild-2024-327310.
A 1-month-old male infant presented unwell with a fever and shock. Blood tests showed hypernatraemia, hyperchloraemia and raised urea and creatinine. Initially, he was treated for dehydration secondary to sepsis. However, high urine output combined with low urine osmolality and high plasma osmolality was suggestive of a disorder of arginine vasopressin (AVP), previously called diabetes insipidus (DI). On further endocrine testing, thyroxine (T4) level was low with an inappropriately normal thyroid-stimulating hormone level with no other anterior pituitary hormone abnormalities, a normal MRI head and ophthalmological assessment. Desmopressin, a synthetic form of AVP, was commenced, however, there was an inadequate response despite dose escalation, leading to a diagnosis of AVP resistance (previously nephrogenic DI) rather than AVP deficiency (previously cranial DI). Copeptin, an AVP precursor peptide and surrogate marker, was significantly elevated. A renal tubulopathy genetic screen demonstrated a likely pathogenic hemizygous variant in the AVP receptor 2 gene, which has previously been associated with X-linked vasopressin resistance. This case demonstrates the challenge of differentiating between AVP deficiency and resistance in infancy and the value of copeptin and genetic testing in confirming diagnosis. We outline an approach to fluid management in AVP disorders.
一名1个月大的男婴因发热和休克就诊。血液检查显示高钠血症、高氯血症以及尿素和肌酐升高。起初,他因败血症继发的脱水接受治疗。然而,高尿量、低尿渗透压和高血浆渗透压提示存在精氨酸加压素(AVP)紊乱,以前称为尿崩症(DI)。进一步的内分泌检查显示,甲状腺素(T4)水平低,促甲状腺激素水平正常但不恰当,且无其他垂体前叶激素异常,头颅MRI和眼科评估均正常。开始使用去氨加压素,即AVP的合成形式,然而,尽管剂量增加,反应仍不充分,从而诊断为AVP抵抗(以前称为肾性尿崩症)而非AVP缺乏(以前称为中枢性尿崩症)。copeptin,一种AVP前体肽和替代标志物,显著升高。肾小管病基因筛查显示AVP受体2基因可能存在致病的半合子变异,该变异以前与X连锁加压素抵抗有关。本病例表明了婴儿期区分AVP缺乏和抵抗的挑战以及copeptin和基因检测在确诊中的价值。我们概述了AVP紊乱的液体管理方法。