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Endocrine Abstracts (2024) 99 RC3.1 | DOI: 10.1530/endoabs.99.RC3.1

ECE2024 Rapid Communications Rapid Communications 3: Adrenal and Cardiovascular Endocrinology | Part I (7 abstracts)

Biochemical control with dose reduction in chronic glucocorticoid therapy over 4 years: A phase III extension study of Chronocort (Efmody) in the treatment of Congenital Adrenal Hyperplasia (CAH)

Richard John M. Ross 1 , Deborah P. Merke 2 , Wiebke Arlt 3 , Aude Brac De La Perriere 4 , Angelica Hirschberg 5 , John D.C. Newell-Price 1 , Alessandro Prete 6 , Aled Rees 7 , Nicole Reisch 8 , Marcus Quinkler 9 , Philippe A. Touraine 10 , Kerry Maltby 11 , Jo Quirke 11 , Naila Aslam 11 , Helen Coope 11 & John Porter 11


1The University of Sheffield, United Kingdom; 2National Institutes of Health, Bethesda, United States; 3MRC Laboratory of Medical Sciences, United Kingdom; 4Louis Pradel Hospital, Bron, France; 5Karolinska Institute, Sweden; 6University of Birmingham, United Kingdom; 7Cardiff University, United Kingdom; 8Klinikum der Universität München, München, Germany; 9Charlottenburg, Berlin, Germany, 10University Hospitals Pitié Salpêtrière - Charles Foix, Paris, France, 11Diurnal, United Kingdom


Background: Management of CAH involves replacing cortisol deficiency and reducing raised adrenal androgens, however the supraphysiological glucocorticoid doses often required to treat hyperandrogenism are associated with poor long-term health outcomes. Modified-release hydrocortisone (MRHC) capsules, Efmody, replicate cortisol diurnal rhythm and improve control of CAH compared to standard glucocorticoid therapy. Here we report changes in glucocorticoid daily dose and 9am 17-hydroxyprogesterone (17-OHP) and androstenedione (A4) in MRHC-treated patients after 48 months in the MRHC single-arm extension study.

Methods: Participants completing MRHC Ph2 and Ph3 studies were eligible to enter a single-arm, open-label extension study. Visits occurred at baseline, weeks 4, 12, 24 and 6-monthly thereafter. MRHC doses were adjusted on the basis of an adrenal insufficiency checklist, and measurement of A4 and 17-OHP at 9am and 1pm, targeting 17-OHP to <36 nmol/l and A4 into the reference range. Participants that completed 48 months in the extension study were reviewed. Participants were considered responders when 9am 17OHP ≤36 nmol/l or A4 ≤7nmol/l and hydrocortisone (HC) dose ≤25 mg/day.

Results: Data were available for 91 participants at baseline and 71 participants (61 with hormone blood results) at 48 months. The median daily HC dose (Inter-Quartile Range [IQR]) at baseline and 48 months were 30 mg (IQR 20–35) and 20 mg (IQR 15–25), respectively (nominal P<0.0001). Geomean (95% CI) 17-OHP was 15.84 (10.32–24.31) and 11.34 (7.048–18.23) nmol/l at baseline and 48 months, respectively. Geomean A4 (95% CI) was 2.242 (1.669–3.012) and 2.092 (1.604–2.727) nmol/l at baseline and 48 months, respectively. Quadrant analysis showed the following:

Dose
≤25 mg/day>25 mg/day
Baseline>36 nmol/l 17-OHP14/91 (15%)21/91 (23%)
≤36nmol/l 17-OHP29/91 (32%)27/91 (30%)
48 months>36 nmol/l 17-OHP15/61 (24%)3/61 (5%)
≤36 nmol/l 17-OHP31/61 (51%)12/61 (20%)
Responder status 51% vs 32% at baseline, P=0.0274 by Fisher’s exact test.

Conclusions: After 48 months of MRHC treatment the median daily HC dose was reduced from a median of 30mg to 20mg and the number of patients achieving responder status based on 9am 17-OHP or A4 while receiving HC ≤25 mg/day increased.

Volume 99

26th European Congress of Endocrinology

Stockholm, Sweden
11 May 2024 - 14 May 2024

European Society of Endocrinology 

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