Bone Age Maturation and Growth Outcomes in Young Children with CAH Treated with Hydrocortisone Suspension

Abstract

Background Young children with CAH require small doses (0.1-1.25 mg) of hydrocortisone (HC) to control excess androgen production and avoid the negative effects of overtreatment. The smallest commercially available HC formulation, before the recent FDA approval of HC granules, was a scored 5 mg tablet. The options to achieve small doses were limited to using a pharmacy-compounded suspension, which the CAH Clinical Practice Guidelines recommended against, or splitting tablets into quarters or eighths, or dissolving tablets into water. Methods Cross sectional chart review of 130 children with classic CAH treated with tablets versus a pharmacy-compounded alcohol-free hydrocortisone suspension to compare growth, weight, skeletal maturation, total daily HC dose and exposure over the first four years of life. Results No significant differences were found in height, weight, or BMI z-scores at 4 years, and in predicted adult height, before or after adjusting for age at diagnosis and sex. Bone age z-scores averaged 2.8 SDs lower for patients on HC suspension compared to HC tablets (p<0.001) after adjusting for age at diagnosis and sex. The suspension group received 30.4% lower (p>0.001) average cumulative HC doses by their 4th birthday. Conclusions Our data indicates that treatment with alcohol-free HC suspension decreased androgen exposure as shown by lower bone age z-scores, allowed lower average and cumulative daily HC dose compared to HC tablets, and generated no significant differences in SDS in growth parameters in children with CAH at 4 years of age. Longitudinal studies of treating with smaller HC doses during childhood are needed.

Practice Variation among Pediatric Endocrinologists in the Dosing of Glucocorticoids in Young Children with Congenital Adrenal Hyperplasia

“…A more recent study compared cortisol exposure and pharmacodynamic adrenal steroid responses to a pharmacy-compounded alcohol-free HC suspension versus commercially available HC tablets and found no significant differences in cortisol pharmacokinetic/pharmacodynamic parameters between the two formulations [6]. In another study comparing epiphyseal (bone age) maturation and growth outcomes, treatment with HC suspension decreased androgen exposure, as shown by lower bone-age z-scores, and allowed a lower average and cumulative daily HC dose compared to HC tablets [7]. More than half of our survey respondents would use HC suspension if it were commercially available, although about one-third of the participants were unsure if they would.…”