Ahmed Khattab1, Ian Marshall. 1. Division of Pediatric Endocrinology, Rutgers-Robert Wood Johnson Medical School, Child Health Institute of New Jersey, New Brunswick, New Jersey, USA.
Abstract
PURPOSE OF REVIEW: The most common enzyme defect associated with congenital adrenal hyperplasia (CAH) is 21-hydroxylase deficiency (21OHD). Glucocorticoid therapy aiming to suppress adrenocorticotrophic hormone (ACTH)-mediated hyperandrogenemia and to replace glucocorticoid deficiency, if indicated, remains the first line of management in CAH with or without mineralocorticoid replacement therapy and salt supplementation. We review interventions that may address unmet needs in the management of CAH. Although the objective of this review is to highlight some potential benefits of supplemental therapies, the authors do not recommend for or against the use of the reviewed therapies. In the review, the terms 'male' and 'female' refer to 'genetic male (46,XY)' and 'genetic female (46,XX)' respectively. RECENT FINDINGS: Supplemental therapies, some of which appear to be promising, attempt to address CAH-associated morbidity but long-term efficacy and safety data are still lacking. SUMMARY: We highlight main ideas behind the use of interventions that target an improvement in physiological glucocorticoid replacement, adult height outcome, and management of female genital virilization in CAH.
PURPOSE OF REVIEW: The most common enzyme defect associated with congenital adrenal hyperplasia (CAH) is 21-hydroxylase deficiency (21OHD). Glucocorticoid therapy aiming to suppress adrenocorticotrophic hormone (ACTH)-mediated hyperandrogenemia and to replace glucocorticoid deficiency, if indicated, remains the first line of management in CAH with or without mineralocorticoid replacement therapy and salt supplementation. We review interventions that may address unmet needs in the management of CAH. Although the objective of this review is to highlight some potential benefits of supplemental therapies, the authors do not recommend for or against the use of the reviewed therapies. In the review, the terms 'male' and 'female' refer to 'genetic male (46,XY)' and 'genetic female (46,XX)' respectively. RECENT FINDINGS: Supplemental therapies, some of which appear to be promising, attempt to address CAH-associated morbidity but long-term efficacy and safety data are still lacking. SUMMARY: We highlight main ideas behind the use of interventions that target an improvement in physiological glucocorticoid replacement, adult height outcome, and management of female genital virilization in CAH.
Authors: Viktoria Stachanow; Uta Neumann; Oliver Blankenstein; Davide Bindellini; Johanna Melin; Richard Ross; Martin J Whitaker; Wilhelm Huisinga; Robin Michelet; Charlotte Kloft Journal: Front Pharmacol Date: 2022-03-17 Impact factor: 5.810