A prospective study of children aged 0–8 years with cah and adrenal insufficiency treated with hydrocortisone granules
Open accessAbstract
Context: Children with congenital adrenal hyperplasia (CAH) and adrenal insufficiency (AI) require daily hydrocortisone replacement with accurate dosing. Objective: Prospective study of efficacy and safety of hydrocortisone granules in children with AI and CAH monitored by 17-OHP (17-hydroxyprogesterone) saliva profiles. Methods: Seventeen children with CAH (9 male) and 1 with hypopituitarism (male), aged from birth to 6 years, had their hydrocortisone medication changed from pharmacy compounded capsules to hydrocortisone granules. Patients were followed prospectively for 2 years. In children with CAH, the therapy was adjusted by 17-OHP salivary profiles every 3 months. The following parameters were recorded: hydrocortisone dose, height, weight, pubertal status, adverse events, and incidence of adrenal crisis. Results: The study medication was given thrice daily, and the median duration of treatment (range) was 795 (1–872) days, with 150 follow-up visits. Hydrocortisone doses were changed on 40/150 visits, with 32 based on salivary measurements and 8 on serum 17-OHP levels. The median daily mg/m2 hydrocortisone dose (range) at study entry for the different age groups 2–8 years, 1 month to 2 years, <28 days was 11.9 (7.2–15.5), 9.9 (8.6–12.2), and 12.0 (11.1–29.5), respectively, and at end of the study was 10.2 (7.0–14.4), 9.8 (8.9–13.1), and 8.6 (8.2–13.7), respectively. There were no trends for accelerated or reduced growth. No adrenal crises were observed despite 193 treatment-emergent adverse events, which were mainly common childhood illnesses. Interpretation: This first prospective study of glucocorticoid treatment in children with AI and CAH demonstrates that accurate dosing and monitoring from birth results in hydrocortisone doses at the lower end of the recommended dose range and normal growth, without occurrence of adrenal crises.
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One prospective 2-year study monitored and titrated doses of hydrocortisone based on salivary 17-OHP levels in children with congenital adrenal hyperplasia up to 8 years of age, and the outcome was a lower median daily hydrocortisone dose with no observed adrenal crises.
“One prospective 2-year study has closely monitored and titrated the doses of hydrocortisone based on salivary 17-OHP levels in children with CAH up to 8 years of age and the outcome was a lower median daily hydrocortisone dose with no observed adrenal crises (51).”
From Hypoglycaemia in adrenal insufficiency (2023) · cites this paper as [51]
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References 19 · Cited by 38
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- Authors
- Neumann, U., Braune, K., Whitaker, M. J., Wiegand, S., Krude, H., Porter, J., Digweed, D., Voet, B., Ross, R. J. M., & Blankenstein, O.
- Affiliations
- Charité – Universitätsmedizin Berlin (DE)
- Diurnal (United Kingdom) (GB)
- University of Sheffield (GB)
- Corresponding author
- Neumann, Uta
- ORCID
- Venue
- The Journal of Clinical Endocrinology & Metabolism
- Publisher
- The Endocrine Society
- Published
- 2020-09-04
- Volume
- 106
- Issue
- 3
- Pages
- e1433-e1440
- ISSN
- 0021-972X (ISSN-L)
- 1945-7197
- PMC
- PMC7947757
- OpenAlex
- W3083434612
- Cited by
- 38
- References
- 19
- Citations by year
- 2026: 5 · 2025: 6 · 2024: 6 · 2023: 9 · 2022: 7 · 2021: 5
- FWCI
- 3.96
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- The Journal of Clinical Endocrinology & Metabolism (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- White Rose Research Online (University of Leeds, The University of Sheffield, University of York) (acceptedVersion)
- Funders
- European Commission Framework 7 Programme (281654)
- Topics
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Adrenal Hormones and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Sexual Differentiation and Disorders
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Hormonal and reproductive studies
- Keywords
- congenital adrenal hyperplasia, adrenal insufficiency, glucocorticoid replacement therapy, 17-hydroxyprogesterone, adrenal crisis, growth, children, hypopituitarism, treatment-emergent adverse events, pubertal development
- MeSH terms
- Adrenal Insufficiency, Adrenal Hyperplasia, Congenital, Child, Child, Preschool, Dosage Forms, Dose-Response Relationship, Drug, Female, Follow-Up Studies, Humans, Hydrocortisone, Infant, Infant, Newborn, Male, Saliva, Cohort Studies, 17-alpha-Hydroxyprogesterone
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