Outcomes after medical treatment for primary aldosteronism: an international consensus and analysis of treatment response in an international cohort.

Background: Primary aldosteronism can be treated medically but there is no standardised method to evaluate treatment outcomes. We aimed to develop criteria for assessing the outcomes of targeted medical treatment of primary aldosteronism, analyse outcomes across an international cohort, and identify factors associated with a complete treatment response.

Methods: An international panel of 31 primary aldosteronism experts used the Delphi method to reach consensus on the definition of complete, partial, or absent biochemical and clinical outcomes of medical treatment of primary aldosteronism. Clinical data at baseline and 6-12 months post-treatment were collected from patients with primary aldosteronism who started targeted medical treatment between 2016 and 2021 at 28 participating centres.

Findings: Consensus was reached for defining complete, partial, or absent biochemical or clinical response. Of 1258 patients (with a mean age of 52 years [SD 11·5] and of whom 610 [48·5%] were female and 648 [51·5%] were male), 1057 (84·0%) had biochemical outcome data (559 [52·9%] had a complete biochemical response). The median daily dose of spironolactone was significantly higher for those with a complete biochemical response than for those without (40 mg [IQR 25-50] vs 25 mg [20-50]; p=0·011). Of the 1248 patients with clinical outcome data, 228 [18·3%] had a complete clinical response whereas 227 (18·2%) had an absent response. Patients with a complete clinical response were more likely than those with partial or absent clinical response to be women (OR 2·099, 95% CI 1·485-2·968; p<0·001), require lower doses of antihypertensive drugs at baseline (0·687, 0·603-0·782; p<0·001), and were less likely to have microalbuminuria or left ventricular hypertrophy (0·584, 0·391-0·873; p=0·009).

Interpretation: The Primary Aldosteronism Medical Treatment Outcome (PAMO) criteria represent an internationally developed outcome standard that can guide clinical practice and research into primary aldosteronism. Efforts to optimise treatment intensity and minimise factors associated with an absent treatment response are needed to improve patient outcomes.

Funding: None.

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