PERSPECTIVE: Treatment with hydrocortisone modified-release capsules in children and adolescents with congenital adrenal hyperplasia: an expert opinion.

Recommended treatment for classic congenital adrenal hyperplasia (CAH) in children is hydrocortisone. This therapy is intended to replace cortisol deficiency, but also to reduce the increased production of androgens and their precursors. The aim is to minimize the undesirable side effects of both cortisol deficiency and androgen excess. Short-acting conventional immediate-release hydrocortisone formulations does not mimic physiological diurnal rhythm and often complicate therapy adjustment, mandating frequent administration of often supraphysiological doses, typically 3-4 times daily, including nocturnal dosing. To simulate the physiological diurnal cortisol pattern, a delayed- and sustained-release hydrocortisone preparation has been developed and its efficacy was validated through phase 2 and 3 trials in adult patients. Regulatory approval has been extended to encompass both adult and adolescent patients aged 12 years and older. This manuscript aims to provide treatment principles formulated by two expert centers specialized in pediatric CAH therapy regarding the utilization of recently registered hydrocortisone modified-release capsules in the daily management and stress dosing regimen for adolescents with CAH. It elucidates proposed dosing strategies, therapeutic surveillance protocols and the prospective accumulation of data for the assessment of treatment efficacy during childhood.