LAUSR

Abstract In patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency, long-acting glucocorticoids (GCs) and/or multiple daily dose short-acting GCs are historically suggested to disease management. However, these treatment strategies are generally associated with GC overexposure, which may induce metabolic syndrome (MS) and impairment of quality of life (QoL), and poor treatment compliance (TC). Dual-release hydrocortisone (DR-HC) with a generally once-daily administration has never been evaluated in cohorts of patients with CAH. The current study aimed at investigating the impact of the switch from conventional GCs to DR-HC on 12-month metabolic and androgens profile, QoL and TC in 39 CAH patients (26F, 13M, 18-50 years, 25 treated with immediate release hydrocortisone (IR-HC), 7 with prednisone, 3 with cortisone acetate, 3 with dexamethasone (DMX), one with IR-HC and DMX). The same cohort, stably treated with conventional GCs, was evaluated during the 12 months before the switch. In the switched cohort, at 12-month-follow-up total (p=0.028) and LDL-cholesterol (p=0.001) were significantly improved, and the diagnosis of MS, performed in two patients at baseline, was not confirmed in one patient. Interestingly, no significant increase in adrenal androgens and no clinical worsening of symptoms and signs related to hyperandrogenism in females were observed. QoL did not significantly changed, whereas TC significantly improved (p=0.001) during the year of DR-HC treatment. Conversely, no significant changes were observed during the control period before the treatment switch. In conclusion, in CAH patients the switch from conventional GCs to DR-HC appears able to induce a beneficial effect on lipid profile, MS and TC, surprisingly maintaining an optimal control of androgen levels.