ISSN 1662-4009 (online)

ey0019.15-12 | Assorted Conditions | ESPEYB19

15.12. One statistical analysis must not rule them all

EJ Wagenmakers , A Sarafoglou , B Aczel

Nature. 2022;605(7910):423-5. doi: 10.1038/d41586-022-01332-8.PubMed ID: 35581494Brief summary: This Nature comment was prompted by the very wide range of calculated values for the COVID-19 infections ‘reproduction number’ R, produced by different modelling teams despite access to the same datasets on UK’s emerging ‘2nd wave’ in October 2020. Valu...

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...

ey0020.6-6 | Clinical Trials – New Treatments | ESPEYB20

6.6. Crinecerfont, a CRF1 receptor antagonist, lowers adrenal androgens in adolescents with congenital adrenal hyperplasia

Newfield Ron S , Sarafoglou Kyriakie , Fechner Patricia Y , Nokoff Natalie J , Auchus Richard J , Vogiatzi Maria G , Jeha George S , Giri Nagdeep , Roberts Eiry , Sturgeon Julia , Chan Jean L , Farber Robert H

Brief summary: This study evaluated the safety, tolerability and efficacy of Crinecerfont, a CRF1R antagonist in adolescents with classic congenital adrenal hyperplasia (CAH).Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD) is characterized by impaired cortisol synthesis and excess adrenal androgen secretion. Sufficient suppression of adrenal andogen production in classic CAH may be difficult with current formulations...