ESPE Abstracts (2024) 98 P2-17

ESPE2024 Poster Category 2 Adrenals and HPA Axis (25 abstracts)

17OHP and cortisol day curve profiles for children using hard-capsule modified release hydrocortisone as compared with immediate release hydrocortisone.

Grace Petkovic 1 , Joanne Blair 1 , Senthil Senniappan 1 & Julie Park 1,2


1Alder Hey Children's Hospital, Liverpool, United Kingdom. 2Lancashire Teaching Hospitals, Preston, United Kingdom


Background: Childhood adrenal insufficiency (AI) is generally treated with immediate release hydrocortisone (IRH), which has a short half-life, requiring multiple daily doses. Modified-release hard hydrocortisone capsules (Efmody®) (MRH) is licenced for use in children >12yrs with congenital adrenal hyperplasia (CAH).

Aims: (1) Describe the characteristics of children switched to MRH; (2) Report blood spot 17-hydroxyprogesterone (17-OHP) (CAH patients) and serum cortisol (non-CAH patients) levels during MRH treatment.

Methods: Retrospective single-centre case-series of children with primary AI [CAH or Addison’s Disease (AD)] and with secondary AI (SAI) treated with MRH.

Results: Patient characteristics are shown in Table 1. Subject 8 (with SAI) stopped MRH due to a lack of symptomatic improvement after 1 month so had no cortisol profiles. Subject 9 (with SAI) had no cortisol day curves obtained before transitioning to adult services. 17-OHP and cortisol profiles are shown in Table 2. Three patients (2 with AD and 1 with SAI) required additional afternoon doses of IRH.

Table 1: Characteristics of patients treated with MRH
Condition Number of patients (% female) Mean age starting MRH (SD) Average BMI SDS (SD) Average Height SDS (SD)
IRH MRH IRH MRH
CAH 5 (80) 13.8 (1.1) 1.50 2.32 0.012 (1.24) 0.30 (1.2)
AD 2 (0) 14.5 (2.1) -0.53 (-) -0.96 (-) -0.71 (0.46) 0.04 (0.51)
SAI 4 (75) 15.5 (3.7) -1.42 (0.29) 0.57 (1.70) -0.10 (1.04) 0.46 (1.97)
Table 2: Plasma 17OHP and cortisol concentrations (nmol/L) * previous treatment with Plenadren
Patient Preparation Time of day
00:00-0359 04:00-07:59 08:00-11:59 12:00-15:59 16:00-19:59 20:00-23:59
17-OHP concentrations (nmol/L)
1 IRH <6 79 15 53
MRH 41 <6 <6
2 IRH* <6 <6 <6
MRH 36 >200 27
3 IRH* >200 22 6 <6
MRH >200 102 28 18
4 IRH 45 <6 <6 <6
MRH
5 IRH >200 55 60
MRH >200 25 27
Plasma cortisol concentrations (nmol/L)
6 IRH* 268 231 229
MRH 908 238 811 621 268
7 IRH
MRH 239 182 254 <50 <50
10 IRH 198 125 205 290 65
MRH 640 647 <50
11 IRH
MRH 309 87 <50

Discussion: These data were collected in a clinical setting, and sampling times varied between patients and treatments. However, cortisol concentrations were high in the morning and low in the afternoon during MRH treatment and 17-OHP profiles were not significantly different. This selected group of patients may not be representative of all paediatric AI patients. Further paediatric pharmacokinetic studies are required.

Volume 98

62nd Annual ESPE (ESPE 2024)

Liverpool, UK
16 Nov 2024 - 18 Nov 2024

European Society for Paediatric Endocrinology 

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