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Bone Age Maturation and Growth Outcomes in Young Children with CAH Treated with Hydrocortisone Suspension
Heba Al-Rayess1, O Yaw Addo2, Elise Palzer3
1Division of Endocrinology, Department of Pediatrics, University of Minnesota Medical School, Minneapolis, MN 55454, USA.
Insights
Hydrocortisone suspension for children with congenital adrenal hyperplasia (CAH) led to lower doses and reduced androgen exposure, without impacting growth compared to tablets. Further studies are needed for long-term outcomes.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Genetics
Background:
- Children with congenital adrenal hyperplasia (CAH) require precise, low-dose hydrocortisone (HC) to manage androgen excess.
- Limited availability of low-dose HC formulations necessitated compounding or tablet manipulation.
- Pharmacy-compounded suspensions were previously discouraged by clinical guidelines.
Purpose of the Study:
- To compare the efficacy and safety of hydrocortisone (HC) suspension versus tablets in young children with classic CAH.
- To evaluate differences in growth, skeletal maturation, and HC dosage over the first four years of life.
Main Methods:
- A cross-sectional chart review included 130 children with classic CAH.
- Patients were treated with either standard HC tablets or a pharmacy-compounded alcohol-free HC suspension.
- Key outcomes measured included growth parameters, bone age, and total daily HC dose.
Main Results:
- No significant differences in height, weight, or BMI z-scores at age 4 were observed between groups.
- Bone age z-scores were significantly lower in the HC suspension group, indicating reduced androgen exposure.
- The HC suspension group received 30.4% lower cumulative HC doses by age 4.
Conclusions:
- Alcohol-free HC suspension effectively reduced androgen exposure and allowed lower HC dosing in children with CAH.
- Growth parameters remained comparable between suspension and tablet formulations at 4 years.
- Longitudinal studies are recommended to assess the long-term effects of low-dose HC treatment in childhood.
Background:
Young children with congenital adrenal hyperplasia (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 US Food and Drug Administration 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 vs a pharmacy-compounded alcohol-free hydrocortisone suspension to compare growth, weight, skeletal maturation, total daily HC dose, and exposure over the first 4 years of life.
Results:
No significant differences were found in height, weight, or body mass index 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 with 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 fourth birthday.
Conclusions:
Our data indicate 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.
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