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Endocrine Abstracts (2022) 86 OC4.5 | DOI: 10.1530/endoabs.86.OC4.5

SFEBES2022 Oral Communications Adrenal and Cardiovascular (6 abstracts)

Comparison of prednisolone and modified-release hydrocortisone capsules in the treatment of congenital adrenal hyperplasia: dose and disease control

Aled Rees 1 , Deborah Merke 2 , Wiebke Arlt 3 , Aude Pierriere 4 , Angelica Hirschberg 5 , Anders Juul 6 , John Newell-Price 7 , Colin Perry 8 , Alessandro Prete 3 , Nicole Reisch 9 , Monica Stikkelbroeck 10 , Philippe Touraine 11 , Helen Coope 12 , Alexander Lewis 12 , John Porter 12 & Richard Ross 7


1University Hospital Wales, Cardiff, United Kingdom; 2NIH, Bethesda, United state of America; 3University of Birmingham, Birmingham, United Kingdom; 4Hopital Louis Pradel, Bron, France; 5Karolinska, Stockholm, Sweden; 6Rigshospitalet, Copenhagen, United Kingdom; 7University of Sheffield, Sheffield, United Kingdom; 8Queen Elizabeth University Hospital, Glasgow, United Kingdom; 9Medizinische Klinik und Poliklinik IV, Klinikum der Universität München, Munich, Germany; 1010Radboud Univ Nijmegen Med Ctr, Nijmegen, Netherlands; 11GH Pitie Salpetriere, Paris, France; 12Diurnal, Cardiff, United Kingdom


Introduction: First-line treatment for congenital adrenal hyperplasia (CAH) is hydrocortisone1. When adequate control is not achieved, prednisolone (or its prodrug prednisone) are often used. However, there has been no formal comparison of disease control in CAH comparing prednis(ol)one vs hydrocortisone and patients are often on a glucocorticoid dose that exceeds the guideline recommended dose of hydrocortisone (≤25 mg/day)1,2. We report an interim analysis of CAH control in patients switched from prednis(ol)one to Modified-Release Hydrocortisone (MRHC) capsules, (Efmody, Diurnal Ltd, Cardiff UK), in an open label safety extension study.

Methods: Patients who completed the phase 3 MRHC study3, were invited to join an open label extension study. We analysed results from patients with a complete dataset at Phase 3 baseline on prednis(ol)one and a minimum of 18 months follow up in the extension study (n=30). Control of CAH was defined as 9am 17-OHP <36 nmol/l and dose was reported according to whether it exceeded 25 mg/day hydrocortisone dose equivalent (prednis(ol)one dose x 5)1,3. Quadrant analysis was performed and Fisher’s exact test applied.

Results: More patients had controlled 17-OHP on a physiological glucocorticoid dose on MRHC than prednis(ol)one, 57% vs 27% P=0.04.

Dose
Prednis(ol)one ≤25 mg/day>25 mg/day
Androgen control>36nmol/l 17-OHP8/30 (27%)5/30 (16%)
<36nmol/l 17-OHP8/30 (27%)9/30 (30%)
Dose
MRHC ≤25 mg/day>25 mg/day
Androgen control>36nmol/l 17-OHP5/30 (16%)0/30 (0%)
<36nmol/l 17-OHP17/30 (57%)8/30 (27%)

Conclusions: Patients who are poorly controlled on prednis(ol)one and/or taking a dose above the recommended dose for adrenal replacement can benefit from a switch to MRHC capsules.

References: 1. Speiser PW. JCEM 2018 103(11):4043–4088. 2. Arlt W. JCEM 2010 95(11): 5110–51213. 3. Merke DP. JCEM 2021(106):e2063-e2077.

Volume 86

Society for Endocrinology BES 2022

Harrogate, United Kingdom
14 Nov 2022 - 16 Nov 2022

Society for Endocrinology 

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