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