Enhanced well-being in patients with congenital adrenal hyperplasia on modified-release hydrocortisone compared to conventional glucocorticoid replacement

Hannah Choi; Selina Ziegler; Hanna Nowotny; Lea Tschaidse; Matthias Auer; Ann-Christin Welp; Martha Merrow; Nicole Reisch-Pawlu

doi:10.1530/endoabs.99.P8

P8

Prev Next

Section Contents Cite

Endocrine Abstracts (2024) 99 P8 | DOI: 10.1530/endoabs.99.P8

ECE2024 Poster Presentations Adrenal and Cardiovascular Endocrinology (95 abstracts)

Enhanced well-being in patients with congenital adrenal hyperplasia on modified-release hydrocortisone compared to conventional glucocorticoid replacement

Hannah Choi ¹ , Selina Ziegler ¹ , Hanna Nowotny ¹ , Lea Tschaidse ¹ , Matthias Auer ¹ , Ann-Christin Welp ¹ , Martha Merrow ² & Nicole Reisch-Pawlu ¹

Views

Author affiliations

¹LMU University Hospital, LMU Munich, Department of Medicine IV; ²Faculty of Medicine, LMU Munich, Institute of Medical Psychology

Background: Conventional glucocorticoid (GC) replacement for congenital adrenal hyperplasia (CAH) induces unphysiological cortisol spikes. In contrast, modified-release hydrocortisone (MR-HC, Efmody) offers a more accurate imitation of the circadian cortisol release, particularly emphasizing the morning surge. The study compares quality of life and sleep-wake cycles in patients with CAH with conventional GC treatment and after a switch to MR-HC.

Methods: In this single-center study we included 41 patients with CAH (28 females, 13 males; 34 classic CAH, 7 non-classic CAH) with a median age of 33 years (IQR 12,75) and a median BMI of 24.67 kg/m² (IQR 5.44). Baseline treatment distribution included 15 patients on HC with a median dose of 25 mg (IQR 10), 20 patients on prednisolone with a median hydrocortisone dose equivalence (HDE) of 27.5 mg (IQR 12.5), and 6 patients on mixed regimens with a median HDE of 30 mg (IQR 15). These patients were switched to MR-HC (Efmody) with a median dose of 25 mg (IQR 10). The following questionnaires were used to gather biopsychosocial data before and ≥3 months after the switch: a self-developed therapy-satisfaction survey, SF-36, Global Fatigue Index, WHOQOL-BREF, PSQI, and MCTQ. In addition, actigraphy watch data was collected from 12 patients using MotionWatch 8 (CamNtech), both before (8 patients with HC, 4 patients with prednisolone) and after the switch to MR-HC, for a median duration of 20 days.

Results: Patients on MR-HC showed significantly increased satisfaction with their replacement regimen, compared to those on conventional GCs (P=0,0048). Patients considered MR-HC more advantageous in managing CAH (P=0,0225). SF-36 survey results indicated less impairment due to mental issues (P=0,0041) and better mental well-being (P=0,0003). Additionally, patients reported an increased subjective health status after a switch to MR-HC compared to one year prior (P=0,0457). However, actigraphy watch data was not different between the two groups, neither the other surveys.

Conclusion: Patients seem to be more satisfied with MR-HC than conventional GC replacement regimen. This was further reflected in a subjective improvement in both physical and mental health status.