Long-term outcomes in patients with congenital adrenal hyperplasia treated with hydrocortisone modified-release hard capsules

Arlt, Wiebke, Bracde la Perriere, Aude, Hirschberg, Angelica, Merke, Deborah, Newell-Price, John, Prete, Alessandro, Rees, Aled ORCID: https://orcid.org/0000-0002-1165-9092, Reisch, Nicole, Touraine, Philippe, Bendfeldt, Hanna, Porter, John, Coope, Helen and Ross, Richard 2025. Long-term outcomes in patients with congenital adrenal hyperplasia treated with hydrocortisone modified-release hard capsules. European journal of endocrinology 193 (1) , pp. 76-84. 10.1093/ejendo/lvaf130

Preview

PDF - Published Version Available under License Creative Commons Attribution. Download (829kB) | Preview

Abstract

Background: Hydrocortisone modified-release hard capsules (MRHC, development name Chronocort) replace the physiological overnight cortisol rise and improve the biochemical control of congenital adrenal hyperplasia (CAH). Aim: This study aims to evaluate long-term safety, tolerability, and efficacy of MRHC. Methods: This is an open-label follow-on study. Results: Ninety-one patients with classic CAH, mean age 37 years, 68% female, 32% male, entered the study and 22 discontinued. Median treatment duration was 4 years (range 0.2-5.8). Median hydrocortisone dose at study entry was 30 mg/day and reduced to 20 mg/day after 24 weeks and stayed stable thereafter until 48 months (P < .0001). Disease control improved on MRHC for the steroid disease markers serum 17-hydroxyprogesterone (17OHP) (P < .03) and androstenedione (A4) (P < .002). After 4 years, the majority of patients had a 17OHP < 4-fold upper limit of normal (ULN) (71%) and an A4 <ULN (90%). Measurement of 17OHP and A4 at 09:00 h and 13:00 h gave similar results. Of the 37 women < 50 years of age who were not on contraceptives over the whole study period, 5 became pregnant (13.5%). Of the men, 13.8% (4/29) had a partner pregnancy. Seven patients had an adrenal crisis with 1 patient reporting 8 of these giving an incidence of 3.9 crises per 100 patient years. Conclusions: Modified-release hard capsule treatment resulted in hydrocortisone dose reduction followed by a stable dose with improved biochemical control associated with fertility. Biochemical control could be reliably monitored by a single blood sample taken between 09:00 and 13:00 h. The incidence of adrenal crises was below that reported previously in patients with CAH.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Schools > Medicine
Publisher:	Oxford University Press
ISSN:	0804-4643
Date of First Compliant Deposit:	26 June 2025
Date of Acceptance:	13 June 2025
Last Modified:	14 Jul 2025 11:34
URI:	https://orca.cardiff.ac.uk/id/eprint/179085

Actions (repository staff only)

Edit Item