ISSN 1662-4009 (online)

ey0020.12-8 | Steroids | ESPEYB20

12.8. Interpretation of steroid biomarkers in 21-hydroxylase deficiency and their use in disease management

K Sarafoglou , DP Merke , N Reisch , H Claahsen-van der Grinten , H Falhammar , RJ Auchus

Brief summary: In this Minireview, one pediatric and one adult case with 21-hydroxylase deficiency (21OHD) are discussed with respect to different clinical questions and steroid biomarkers reflecting their diagnosis, treatment and disease control. Basics of the disease mechanisms with different aspects throughout life (childhood, adulthood, sex, fertility and pregnancy) are discussed to lay grounds for the interpretation and use of laboratory data, including the newer 11-oxyge...

ey0019.8-8 | Clinical Trials – New Treatments | ESPEYB19

8.8. Crinecerfont lowers elevated hormone markers in adults with 21-hydroxylase deficiency congenital adrenal hyperplasia

AR Auchus , K Sarafoglou , PY Fechner , MG Vogiatzi , EA Imel , SM Davis , N Giri , J Sturgeon , E Roberts , JL Chan , RH Farber

J Clin Endocrinol Metab. 2022; 107(3): 801-812. PMID: 34653252 https://pubmed.ncbi.nlm.nih.gov/34653252/Brief Summary: This clinical trial evaluated the safety and efficacy of crinecerfont, a CRF1R antagonist, in suppressing adrenal androgen secretion in adult patients with classic congenital adrenal hyperplasia (CAH) during a treatment period of 14 days.Class...

ey0019.8-9 | Clinical Trials – New Treatments | ESPEYB19

8.9. Tildacerfont in adults with classic Congenital Adrenal Hyperplasia: Results from two phase 2 studies

K Sarafoglou , CN Barnes , M Huang , EA Imel , IJ Madu , DP Merke , D Moriarty , S Nakhle , RS Newfield , MG Vogiatzi , RJ Auchus

J Clin Endocrinol Metab. 2021; 106(11): e4666-e4679. PMID: 34146101https://pubmed.ncbi.nlm.nih.gov/34146101/Brief Summary: These clinical trials evaluated the safety and efficacy of tildacerfront, a CRF1R antagonist, in suppressing adrenal androgen secretion in adult patients with classical Congenital adrenal hyperplasia (CAH) during a treatment period of 12 weeks.<p class="abs...