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Do All Patients with Congenital Adrenal Hyperplasia Need to Be on Hydrocortisone Three Times a Day in Order to Have
Lauren A Logan1, Todd D Nebesio1, George J Eckert2
1Division of Pediatric Endocrinology, Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana, USA.
Insights
Optimal treatment for congenital adrenal hyperplasia (CAH) in children may vary. This study found no significant differences in growth or disease control between children on three-times-daily hydrocortisone (HC) and those on other glucocorticoid regimens.
Area of Science:
- Pediatric Endocrinology
- Genetics and Genetic Diseases
- Pharmacology and Therapeutics
Background:
- Current guidelines recommend three-times-daily hydrocortisone (HC) for managing congenital adrenal hyperplasia (CAH) in pediatric patients.
- However, diverse glucocorticoid treatment regimens are utilized in clinical practice, prompting investigation into their comparative efficacy.
Discussion:
- This retrospective study analyzed medical records of 104 children with classic CAH over ten years.
- Key variables included glucocorticoid type, dosage, frequency, and growth parameters (height z-score, BMI z-score, growth velocity z-score).
Key Insights:
- Less than half of the CAH patients received the recommended three-times-daily HC regimen.
- No significant differences were observed in height z-score, BMI z-score, or growth velocity between patients on TID HC and alternate regimens.
- Patients on TID HC were younger and received lower glucocorticoid doses compared to those on other regimens.
Outlook:
- The findings suggest that various glucocorticoid treatment strategies may be equally effective for managing CAH in children.
- Further research could explore long-term outcomes and patient-reported quality of life across different regimens.
Background:
Three times daily (TID) hydrocortisone (HC) is recommended as the optimal glucocorticoid regimen in growing children with congenital adrenal hyperplasia (CAH). However, a variety of other treatment schemes are used in the clinical setting.
Objective:
The aim of this study was to determine whether there are clinical differences between children being treated with TID HC versus those receiving other glucocorticoid regimens. Furthermore, we sought to determine whether there was evidence of a deleterious effect on growth in children receiving treatment with alternate regimens.
Methods:
Medical records of children followed in our pediatric endocrinology outpatient clinic for classic CAH secondary to 21-hydroxylase deficiency during the last 10 years were reviewed. Variables analyzed included sex, age at the most recent visit, glucocorticoid type, frequency and dose (mg/m2/day), height z-score, BMI z-score, ethnicity, most recent bone age, growth velocity z-score, and provider's impression of compliance (good or poor).
Results:
Of 104 children (51% boys) with CAH, 50 (48%) were on TID HC, 43 (41%) were on prednisone or prednisolone, and 5 (5%) were on dexamethasone. An additional 6 (6%) were on HC administered either 2 or 4 times daily. No differences were seen between TID HC and alternate regimen groups with respect to sex, height z-score, BMI z-score, ethnicity, provider assessment of compliance, ratio of bone age to chronologic age, or growth velocity. The average height z-score was -0.40 ± 1.31 in the TID HC group compared to -0.87 ± 1.33 in the alternate regimen group (p = 0.075). Patients receiving TID HC were younger (p = 0.027) and on a lower glucocorticoid dose (p = 0.001) than those on alternate regimens.
Conclusions:
Less than half of our patients with CAH were receiving TID HC. Reassuringly, growth parameters and other indices of disease control were equivalent between patients on conventional HC dosing and other therapeutic approaches. These results suggest that a range of glucocorticoid treatment regimens may be equally viable in children with CAH.
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