Endocrine Abstracts

JOINT2666

Background: Premature adrenarche (PA) is common among 5-9 years old children, mainly females. The outcome is usually benign in the absence of a definable cause for androgen excess, but some patients seek management for their symptoms of hyperandrogenism, body odor, axillary or pubic hair but some experience significant distress and seek for treatment for facial acne. Management of hyperandrogenism (including acne) among pubertal and adult females includes “off label” use of spironolactone, a steroidal aldosterone antagonist. licenced for use as a diuretic but has also an anti-androgenic effect in higher doses. As such, it has been used for treatment of prepubertal acne associated with benign PA at the Royal Children Hospital, Melbourne.

Aim: To characterize those patients who received spironolactone compared to a similar population who received standard of care dermatological preparations only, or no treatment at all, and to assess their growth, puberty, short and long term outcome parameters and safety.

Methods: A retrospective case-control study included all patients diagnosed with benign PA between 2000 and 2024. Case/control ratio was 1:2. Data included clinical, growth and laboratory assessments.

Results: Study population included 8 females who were treated with spironolactone, median age 8.3 years (range 7.5,11.7), and 16 female who were not treated with spironolactone, median age 6 years (range 4.2,9.2). The spironolacton group was significantly older at presentation, P <0.001. Spironolactone doses ranged from 25 to 100 mg/day, with most patients stabilizing at 50 mg/day. 75% of those treated experienced initial improvement in acne severity. However, 62.5% required additional therapies as additional topical creams, retinoic acid, oral antibiotics and oral contraceptive pill at follow-up visits, suggesting partial long-term efficacy. Minor adverse events occurred in 25% of treated patients but resolved without discontinuation. No significant electrolyte abnormalities or renal dysfunction were reported. Both groups initiated central puberty at comparable ages (mean 9.2 vs. 9.3 years), indicating that spironolactone did not delay puberty onset. Final heights aligned with mid-parental targets in both cohorts, alleviating concerns about growth suppression. The study noted that most patients stabilized at lower doses (1–1.5 mg/kg) compared to adult acne regimens (2.5 -3mg/kg), potentially balancing efficacy and safety in children.

Conclusions: spironolactone offers a viable option for benign PA-associated acne. Our findings support cautious off-label adoption, while advocating rigorous longitudinal monitoring and further prospective trials to solidify a role for spironolactone in pediatric acne management.