Macrolide-Induced Aldosterone Suppression as a Functional Marker of KCNJ5-Mutated Aldosterone-Producing Adenoma: A Proof-of-Concept Clinical and Experimental Study (MAPA Study)

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ID: 326590
2026
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Abstract
OBJECTIVE: Primary aldosteronism (PA), the most common curable form of hypertension, is frequently caused by aldosterone-producing adenomas (APAs) harboring KCNJ5 mutations that render aldosterone synthesis sensitive to macrolides in vitro. This study aimed to evaluate whether a single dose of roxithromycin reduces plasma aldosterone concentration (PAC) and blood pressure (BP) in patients with KCNJ5-mutated APA, and to characterize the haemodynamic effects of macrolides in mice. DESIGN: Prospective, within-patient pharmacologic challenge. METHODS: At the specialized Hypertension Center, University of Padua, eligible consecutive hypertensive patients screened for PA underwent a pharmacologic challenge with a single oral dose of roxithromycin to investigate the within-patient changes in plasma aldosterone concentration (PAC), active renin, cortisol, and blood pressure (BP). RESULTS: Among 373 challenged patients, 18 had KCNJ5 (G151R or L168R) -mutated APA, 25 had wild-type APA, and 307 had no PA. Roxithromycin reduced PAC (P<0.001) only in APA with KCNJ5 mutation, albeit it did not lower BP values. However, it produced a small BP decrease in non-PA hypertensive patients. Mouse studies showed that this BP-lowering can be due to macrolides-induced attenuation of angiotensin II actions and enhancement of endothelium- and nitric oxide-dependent vasodilation. CONCLUSIONS: The selective suppression of aldosterone secretion with roxithromycin in KCNJ5-mutated APA in vivo, which aligns with ex-vivo mechanistic data, can represent a functional marker of KCNJ5 mutations and could help guide precision subtyping of PA patients. Macrolides also exhibit an aldosterone-independent antihypertensive effect in non-PA patients, which involves nitric oxide-dependent vasodilation.
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openalex_W7204552549 Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Gian Paolo Rossi, Brasilina Caroccia, Alessandro Bressan, Clement Byiringiro, Ana M. Briones, Giulio Ceolotto, Giuseppe Zanotti, Roberto Padrini, Ana Maria Briones, Teresa Maria Seccia
Journal european journal of endocrinology
Year 2026
DOI
10.1093/ejendo/lvag161
URL
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