Hydrocortisone Suspension Provides Similar Growth Outcomes as Hydrocortisone Tablets in Young Children With Congenital Adrenal Hyperplasia: A Cross Sectional Study

Young children with CAH require small doses (0.1–1.25mg) and incremental adjustments of hydrocortisone (HC) to control excess androgen production and avoid the negative effects of overtreatment. A recent 6 hour pharmacokinetic/pharmacodynamic study reported that alcohol-free HC suspension provides similar cortisol exposure to tablets (1), but more data is needed to assess its clinical efficacy. We performed a chart review to determine the effect of the alcohol-free HC suspension compared to tablets on height, weight, BMI, bone age z-scores and corrected height z-scores to target height z-scores in children aged 2 yrs and 4 yrs in a cohort with classic CAH. Independent 2-sample t-tests examined cumulative and average HC dose at 2 and 4 yrs. Triple logistic modeling of longitudinal heights were used to calculate predicted near-adult height. Adjusted linear regression models assessed the effect of HC suspension compared to tablets on final adult height. Charts of 130 children (70 females, 100 salt wasting and 30 simple virilizing) were reviewed. At 2 yrs, 97 were treated with tablets and 33 with suspension (17 previously switched from tablets). At 4 yrs, 89 were treated with tablets and 41 with suspension (25 switched). No significant differences in height or BMI z-scores at both 2 and 4 yrs, before or after adjusting for age at diagnosis and sex were found. Bone age z-scores averaged 7.2 SDs lower for patients treated with HC suspension only compared to patients on HC tablets at age 4 (p<0.001), and 5.93 SDs lower for patients switched from tablets to suspension compared to tablets (p<0.001). The suspension group received 16% lower (p=0.055) and 25% lower (p=0.002) cumulative HC doses by the ages of 2 yrs and 4 yrs respectively. Average daily HC dose was lower by 3.44 and by 4.46 mg/m2/d over the first 2 and 4 yrs of life respectively. No significant differences were found between patients treated with tablets and suspension in the predicted final adult height, its z-score or its corrected z-score to target height after adjusting for age at diagnosis, sex and diagnosis. Our data indicates that treatment with alcohol-free HC suspension decreased androgen exposure as shown by lower bone age z-scores, generated no significant differences in SDS in observed height, BMI or predicted near-adult height, and allowed for lower average and cumulative daily HC dose compared to HC tablets in children with CAH. Reference: (1) Sarafoglou et al., J Clin Pharmacol.2015;55(4):452–7.