Do All Patients with Congenital Adrenal Hyperplasia Need to Be on Hydrocortisone Three Times a Day in Order to Have Normal Growth?

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.
(© 2022 S. Karger AG, Basel.)