ESPE Yearbook of Paediatric Endocrinology

Nat Rev Endocrinol. 2022; 18(6): 337-352. PMID: 35411073https://pubmed.ncbi.nlm.nih.gov/35411073/Brief Summary: This review summarizes the current knowledge and understanding of the therapeutic challenges and the novel advances in the management of classical congenital adrenal hyperplasia (CAH).This review describes the present and novel therapeutic options for 21OHD C...

To read the full abstract: J Clin Endocrinol Metab. 2018; 103(6):2336-2345Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive disorders characterized by impaired cortisol production in the adrenal cortex. Classic CAH represents the most common cause of primary adrenal insufficiency in childhood (10). Infant mortality in patients with CAH has decreased since the advent of...

To read the full abstract: J Clin Endocrinol Metab 2017;102:1797-1806We know that conventional glucocorticoid therapies fail to mimic the normal diurnal profile of cortisol secretion, which should show an early morning surge in circulating levels, followed by a gradual daytime reduction, and night-time suppression. The early morning surge in ACTH also drives adrenal androgen production, which is e...

J Clin Endocrinol Metab 2021; 106(5): e2063–e2077.https://pubmed.ncbi.nlm.nih.gov/33527139/The authors report the findings of a 6-month, randomized, phase 3 trial, with a single arm extension, to investigate the efficacy, safety and tolerability of modified release hydrocortisone (MC-HC) replacement therapy versus standard glucocorticoid replacement therapy in 122 adult patients with c...