Modified-release Hydrocortisone in Congenital Adrenal Hyperplasia.

Merke, Deborah P, Mallappa, Ashwini, Arlt, Wiebke, Brac de la Perriere, Aude, Hirschberg, Angelica Lindén, Juul, Anders, Newell-Price, John, Perry, Colin G, Prete, Alessandro, Rees, D Aled, Reisch, Nicole, Stikkelbroeck, Nike, Tourraine, Phillippe, Maltby, Kerry, Treasure, F Peter, Porter, John and Ross, Richard J (2021) Modified-release Hydrocortisone in Congenital Adrenal Hyperplasia. The Journal of clinical endocrinology and metabolism. ISSN 1945-7197. This article is available to all UHB staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their UHB Athens login IDs

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Official URL: https://academic.oup.com/jcem

Abstract

BACKGROUND

Standard glucocorticoid therapy in congenital adrenal hyperplasia regularly fails to control androgen excess, causing glucocorticoid over-exposure and poor health outcomes. We investigated whether modified-release hydrocortisone (MR-HC), which mimics physiologic cortisol secretion, could improve disease control.

METHODS

6-month randomized phase III study, MR-HC versus standard glucocorticoid, followed by single-arm MR-HC extension study. Primary outcomes were change in 24-hour standard deviation score (SDS) of androgen precursor 17-hydroxyprogesterone (17OHP) for phase III, and efficacy, safety and tolerability of MR-HC for the extension study.

RESULTS

The phase III study recruited 122 adult CAH patients. While the study failed its primary outcome at 6 months, there was evidence of better biochemical control on MR-HC, with lower 17OHP SDS at 4 (P=0.007) and 12 (P=0.019) weeks, and between 07:00h to 15:00h (P=0.044) at 6 months. The percentage of patients with controlled 09:00h serum 17OHP (<1200 ng/dl) was 52% at baseline, at 6 months 91% for MR-HC and 71% for standard therapy (P=0.002), and 80% for MR-HC at 18 months extension. The median daily hydrocortisone dose was 25mg at baseline, at 6 months 31mg for standard therapy and 30mg for MR-HC, and after 18 months 20mg MR-HC. Three adrenal crises occurred in phase III, none on MR-HC and 4 in extension study. MR-HC resulted in patient-reported benefit including menses restoration in eight patients (one on standard therapy), and 3 patient and 4 partner pregnancies (none on standard therapy).

CONCLUSION

MR-HC improved biochemical disease control in adults with reduction in steroid dose over time and patient-reported benefit.

Item Type: Article
Additional Information: This article is available to all UHB staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their UHB Athens login IDs
Subjects: WK Endocrine system. Endocrinology
Divisions: Ambulatory Care > Endocrinology
Related URLs:
Depositing User: Jamie Edgar
Date Deposited: 05 Feb 2021 16:12
Last Modified: 05 Feb 2021 16:12
URI: http://www.repository.uhblibrary.co.uk/id/eprint/3952

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