Poor Response to Substitution Therapy with Cortisone Acetate in Patients with Congenital Adrenal Hyperplasia

Ishiguro, Hiroshi; Imamura, Toshihiro; Nakajima, Ryoichi; Yamano, Tsunekazu

doi:10.1297/cpe.13.11

Cited by 3 publications

(2 citation statements)

References 12 publications

(10 reference statements)

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“…While we do not have information regarding the clinical reasoning that led to these changes, we can speculate that the timing is related to the completion of linear growth. Using cortisone acetate in children with 21OHD is in line with international recommendations; however, previous publications described its variable bioactivity in relation to the dependency on 11ß-hydroxysteroid dehydrogenase type 1, suggesting higher variability of therapeutic efficiency (23,24). We observed a number of children visits reporting the use of dexamethasone and prednisolone.…”

Section: Discussionsupporting

confidence: 79%

International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: data from the I-CAH registry

Bacila

Freeman

Daniel

et al. 2021

European Journal of Endocrinology

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Objective: Despite published guidelines no unified approach to hormone replacement in congenital adrenal hyperplasia (CAH) exists. We aimed to explore geographical and temporal variations in the treatment with glucocorticoids and mineralocorticoids in CAH. Design: This retrospective multi-center study, including 31 centers (16 countries), analyzed data from the International-CAH Registry. Methods: Data was collected from 461 patients aged 0-18 years with classic 21-hydroxylase deficiency (54.9% females) under follow-up between 1982 – 2018. Type, dose and timing of glucocorticoid and mineralocorticoid replacement was analyzed from 4174 patient visits. Results: The most frequently used glucocorticoid was hydrocortisone (87.6%). Overall, there were significant differences between age groups with regards to daily hydrocortisone-equivalent dose for body surface, with the lowest dose (median with interquartile range) of 12.0 (10.0 – 14.5) mg/ m2/ day at age 1 - 8 years and the highest dose of 14.0 (11.6 - 17.4) mg/ m2/ day at age 12-18 years. Glucocorticoid doses decreased after 2010 in patients 0-8 years (p<0.001) and remained unchanged in patients aged 8-18 years. Fludrocortisone was used in 92% of patients, with relative doses decreasing with age. A wide variation was observed among countries with regards to all aspects of steroid hormone replacement. Conclusions: Data from the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency to treatment with high doses in children.

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Section: Discussionsupporting

confidence: 79%

International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: data from the I-CAH registry

Bacila

Freeman

Daniel

et al. 2021

European Journal of Endocrinology

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“…Cortisone acetate is not recommended in the treatment of 21OHD, because of its lower availability compared to HC and the need to be converted to cortisol by 11-beta hydroxysteroid dehydrogenase [ 32 ].…”

Section: Conventional Treatment In Different Age Groupsmentioning

confidence: 99%

Novel treatments for congenital adrenal hyperplasia

Schröder

Grinten²

2022

Rev Endocr Metab Disord

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Patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21OHD) need life-long medical treatment to replace the lacking glucocorticoids and potentially lacking mineralocorticoids and to lower elevated adrenal androgens. Long-term complications are common, including gonadal dysfunction, infertility, and cardiovascular and metabolic co-morbidity with reduced quality of life. These complications can be attributed to the exposure of supraphysiological dosages of glucocorticoids and the longstanding exposure to elevated adrenal androgens. Development of novel therapies is necessary to address the chronic glucocorticoid overexposure, lack of circadian rhythm in glucocorticoid replacement, and inefficient glucocorticoid delivery with concomitant periods of hyperandrogenism. In this review we aim to give an overview about the current treatment regimens and its limitations and describe novel therapies especially evaluated for 21OHD patients.

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Biomarkers in congenital adrenal hyperplasia

Bacila,

Lawrence,

Badrinath

et al. 2023

Clinical Endocrinology

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Monitoring of hormone replacement therapy represents a major challenge in the management of congenital adrenal hyperplasia (CAH). In the absence of clear guidance and standardised monitoring strategies, there is no consensus among clinicians regarding the relevance of various biochemical markers used in practice, leading to wide variability in their application and interpretation. In this review, we summarise the published evidence on biochemical monitoring of CAH. We discuss temporal variations of the most commonly measured biomarkers throughout the day, the interrelationship between different biomarkers, as well as their relationship with different glucocorticoid and mineralocorticoid treatment regimens and clinical outcomes. Our review highlights significant heterogeneity across studies in both aims and methodology. However, we identified key messages for the management of patients with CAH. The approach to hormone replacement therapy should be individualised, based on the individual hormonal profile throughout the day in relation to medication. There are limitations to using 17‐hydroxyprogesterone, androstenedione and testosterone, and the role of additional biomarkers such 11‐oxygenated androgens which are more disease specific should be further established. Noninvasive monitoring via salivary and urinary steroid measurements is becoming increasingly available and should be considered, especially in the management of children with CAH. Additionally, this review indicates the need for large scale longitudinal studies analysing the interrelation between different monitoring strategies used in clinical practice and health outcomes in children and adults with CAH.

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Poor Response to Substitution Therapy with Cortisone Acetate in Patients with Congenital Adrenal Hyperplasia

Cited by 3 publications

References 12 publications

International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: data from the I-CAH registry

International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: data from the I-CAH registry

Novel treatments for congenital adrenal hyperplasia

Biomarkers in congenital adrenal hyperplasia

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