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Endocrine Abstracts (2024) 99 P215 | DOI: 10.1530/endoabs.99.P215

ECE2024 Poster Presentations Adrenal and Cardiovascular Endocrinology (95 abstracts)

Incidence of adrenal crisis in Congenital Adrenal Hyperplasia (CAH) patients during a prospective monitored long-term study of modified-release hydrocortisone (MRHC) capsules, (Efmody)

Richard John M. Ross 1 , DeborahP Merke 2 , Ashwini Mallappa 2 , Wiebke Arlt 3 , AudeBrac DeLaPerriere 4 , Angelica Hirschberg 5 , John DC 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, UK; 2National Institutes of Health, Bethesda, USA; 3MRC Laboratory of Medical Sciences, UK; 4Louis Pradel Hospital, Bron, France; 5Karolinska Institute, Sweden; 6University of Birmingham, UK; 7Cardiff University, UK; 8Medizinische Klinik und Poliklinik I | LMU Klinikum, München, Germany; 9Charlottenburg, Berlin, Germany; 10University Hospitals Pitié Salpêtrière - Charles Foix, Paris, France; 11Diurnal, UK


Background: Adrenal crisis is the leading cause of excess mortality in patients with CAH1. Retrospective studies report an adrenal crisis incidence of 5-10/100 patient years (PY), with mortality 0.5/100 PY2. Modified-release hydrocortisone (MRHC) capsules, (Efmody), replicate cortisol diurnal rhythm and improve androgen control in CAH compared to standard glucocorticoid therapy2. Here, we report the incidence of adrenal crisis in CAH patients from a prospective study of MRHC in CAH patients.

Methods: Patients completing MRHC Ph2 and Ph3 studies were eligible to enter a single-arm, open-label extension study. Study visits occurred at baseline, weeks 4, 12, 24 and 6-monthly thereafter. The primary endpoint was the safety of MRHC over time. Adrenal crisis was defined according to Allolio 20153. MRHC doses were adjusted on the basis of an adrenal insufficiency checklist, and measurement of androstenedione (A4) and 17-hydroxyprogesterone (17-OHP) at 0900 and 1300 h.

Results: 91 patients entered the study, mean age 37 years, 68% female, 32% male. 22 discontinued; 11 at patient request, 5 due to pregnancy, 2 undergoing fertility treatment, 2 at physician/sponsor request, 1 due to an AE (carpal tunnel syndrome), and 1 due to death (myocardial infarction). Median treatment duration was 1500 days, range 0.2 to 5.8 years. Median MRHC dose at study entry was 30 mg/day, reducing to 20 mg/day from 24-weeks until end of study. Signs and symptoms of adrenal insufficiency due to under-treatment were reported for 41 (45.1%) participants, most frequently fatigue which was reported at some point by 41.8% of all participants. Signs and symptoms of overtreatment were reported for 25 (27.5%) participants, most frequently sudden weight gain which was reported at some point by 16.5% of all participants. Signs and symptoms of both over- and undertreatment occurred predominantly during the first 24 weeks. The study encompassed 357 participant years, and 18 adverse events considered indicative of adrenal crisis occurred in 7 participants, giving an incidence rate of 5.043 adrenal crises/100 PY.

Conclusions: Data from this longest prospectively monitored study in CAH suggest that the incidence of adrenal crisis on MRHC is at the low end of that reported in retrospective studies and the safety profile of MRHC is otherwise similar to immediate release hydrocortisone.

References: 1. Falhammar H. JCEM 2014 99 e2715-E21.

2. Merke DP. JCEM 2021 106 e2063–e2077.

3. Allolio B. EJE 2015 172 r115-24

Volume 99

26th European Congress of Endocrinology

Stockholm, Sweden
11 May 2024 - 14 May 2024

European Society of Endocrinology 

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