Height Velocity Defined Metabolic Control in Children With Congenital Adrenal Hyperplasia Using Urinary Steroid GC-MS Analysis
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BACKGROUND: Treatment of children with classic congenital adrenal hyperplasia (CAH) with glucocorticoids is a difficult balance between hypercortisolism and hyperandrogenism. Biochemical monitoring of treatment is not well defined. Achievement of a normal growth rate is the most important therapeutic goal. METHODS: We retrospectively evaluated 123 24-hour gas chromatography-mass spectrometry urinary steroid metabolome analyses together with their corresponding 1-year height velocity (HV) z scores in 63 prepubertal children aged 7.2 ± 1.6 years with classic CAH due to 21-hydroxylase deficiency treated with hydrocortisone and fludrocortisone. RESULTS: Multivariate linear mixed effects model analysis revealed a positive influence of CAH-specific z scores of summed urinary androgen metabolites (B = 0.97 ± 0.20, t = 4.87, P < 0.0001) and a negative influence of the cortisol metabolite tetrahydrocortisol (B = -1.75 ± 0.79, t = -2.20, P = 0.03) on HV z scores. Receiver operating characteristic analysis demonstrated that adrenal androgen excess, defined as HV >1.5 z, was best determined by a z score of all urinary androgen metabolites of >0.512 [accuracy, 66.2%; sensitivity, 57.1%; specificity, 74.4%; positive prediction value (PPV), 66.7%; negative prediction value (NPV), 65.9%]. Tetrahydrocortisol excretion >1480 µg/m2 BSA/d in conjunction with suppressed urinary androgen metabolites <0.163 z indicated overtreatment, defined as HV < -1.5 z (accuracy, 79.6%; sensitivity, 40.0%; specificity, 94.9%; PPV, 75.0%; NPV, 80.4%). CONCLUSION: We established target values for urinary steroid metabolite excretions in children with CAH based on their growth rate. Urinary steroid metabolome analysis represents a highly suitable method for monitoring metabolic control in children with CAH.
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Timed samples (e.g. 24-hr urine) provide additional information on hormonal production rates via determination of steroid excretion rates.
“Timed samples (e.g.24-hr urine) provide additional information on hormonal production rates via determination of steroid excretion rates (296,297).”
From Congenital Adrenal Hyperplasia—Current Insights in Pathophysiology, Diagnostics, and Management (2022) · cites this paper as [297]
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Self-citation
- Authors
- Kamrath, C., Wettstaedt, L., Hartmann, M. F., & Wudy, S. A.
- Affiliations
- Justus-Liebig-Universität Gießen (DE)
- Corresponding author
- Wudy, Stefan A
- Venue
- The Journal of Clinical Endocrinology & Metabolism
- Publisher
- The Endocrine Society
- Published
- 2019-05-21
- Volume
- 104
- Issue
- 9
- Pages
- 4214-4224
- ISSN
- 0021-972X (ISSN-L)
- 1945-7197
- OpenAlex
- W2945645978
- Cited by
- 18
- References
- 34
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 3 · 2023: 3 · 2022: 4 · 2021: 2 · 2020: 3 · 2019: 1
- FWCI
- 0.83
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- The Journal of Clinical Endocrinology & Metabolism (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Sexual Differentiation and Disorders
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Adrenal Hormones and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Keywords
- classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, height velocity, adrenal androgen excess, tetrahydrocortisol, cortisol, fludrocortisone, overtreatment, growth rate, children
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