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Impact of fluconazole in vasopressin 2 receptor-related congenital arginine vasopressin resistance

Item Type:Article
Title:Impact of fluconazole in vasopressin 2 receptor-related congenital arginine vasopressin resistance
Creators: Mortensen, Line A., Bistrup, Claus, Rittig, Søren, Hertz, Jens Michael, Schmidt-Ott, Kai M. ORCID logoORCID: https://orcid.org/0000-0002-7700-7142, Klussman, Enno ORCID logoORCID: https://orcid.org/0000-0003-4004-5003, Hinze, Christian ORCID logoORCID: https://orcid.org/0000-0003-2526-1621, Jensen, Boye L. and Hinrichs, Gitte R.
Abstract:INTRODUCTION: Arginine vasopressin resistance (AVP-R), previously called nephrogenic diabetes insipidus is a rare inherited disorder characterized by excessive urine output (often over 10 l/d) and a risk of life-threatening dehydration, resulting from kidney collecting duct insensitivity to arginine vasopressin. Currently, no curative treatment exists. Preclinical studies suggest that repurposing of anti-fungal fluconazole enhances AQP2-mediated water transport. Here, we present results from a clinical intervention study evaluating the effect of fluconazole on 24-hour urine output and urine concentrating ability in patients with congenital AVP-R caused by a pathogenic variant in the AVPR2 gene encoding the receptor of AVP. METHODS: We performed an open-label, non-randomized intervention in five adult males with AVPR2-related AVP-R. Participants received oral fluconazole 400 mg daily for 14 days, with baseline and follow-up assessments including water deprivation test. The primary endpoints were 24-hour urine output and urinary concentrating ability. Secondary endpoints included urinary and plasma osmolality, surrogate copeptin secretion, electrolytes, and micturition frequency. Safety and treatment adherence were evaluated weekly. RESULTS: Median fluconazole concentration following treatment were 13.7 µg/ml (range 12.1–24.4) in plasma and 28.6 µg/ml (range 22.4–34.7) in urine, respectively. Median change in 24-h urine output was +0.8 l (range −3.6 to +5.7) and in urine osmolality −3 mOsm/kg (range −78 to +25), with no change in micturition frequency. During water deprivation, body weight decreased −2.0 to −2.8 kg at baseline vs. −2.1 to −3.1 kg after treatment, urine osmolality changed −91 to +222 vs. −12 to +193 mOsm/kg, and plasma copeptin increased 4.8 to 16.8 vs. 4.2 to 12.9 pmol/l. CONCLUSIONS: Fluconazole treatment did not reduce urine output or improve urine concentrating ability in adult patients with congenital AVP-R.
Keywords:Antifungal Agents, Arginine Vasopressin, Aquaporins, AVPR2, Polyuria, Vasopressin Resistance
Source:Kidney International
ISSN:0085-2538
Publisher:Elsevier / International Society of Nephrology
Date:24 September 2026
Official Publication:https://doi.org/10.1016/j.kint.2026.08.024
PubMed:View item in PubMed

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