TY - JOUR
T1 - Bone Age Maturation and Growth Outcomes in Young Children with CAH Treated with Hydrocortisone Suspension
AU - Al-Rayess, Heba
AU - Addo, O. Yaw
AU - Palzer, Elise
AU - Jaber, Mu'Taz
AU - Fleissner, Kristin
AU - Hodges, James
AU - Brundage, Richard
AU - Miller, Bradley S.
AU - Sarafoglou, Kyriakie
N1 - Publisher Copyright:
© 2021 The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society.
PY - 2022/2/1
Y1 - 2022/2/1
N2 - 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.
AB - 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.
KW - adrenal insufficiency
KW - bone age
KW - congenital adrenal hyperplasia
KW - growth
KW - hydrocortisone
KW - suspension
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U2 - 10.1210/jendso/bvab193
DO - 10.1210/jendso/bvab193
M3 - Article
C2 - 35047717
AN - SCOPUS:85126653336
SN - 2472-1972
VL - 6
JO - Journal of the Endocrine Society
JF - Journal of the Endocrine Society
IS - 2
M1 - bvab193
ER -