Loading ...

TOPLINE:

Adjunct treatment with anastrozole in children with congenital adrenal hyperplasia (CAH) delays the rate of bone maturation and increases the predicted adult height over 6 years, with no significant increase in hydrocortisone doses.

METHODOLOGY:

• Aromatase inhibitors, such as anastrozole, have been used off-label to increase height in children with short stature; however, few studies have investigated the use of aromatase inhibitors in children with CAH.
• Researchers conducted a retrospective chart review to examine the effect of anastrozole as an adjunctive therapy in children with CAH and advanced bone age, providing long-term longitudinal growth and bone maturation outcomes.
• They included 60 children (mean age at initiation of anastrozole, 7.7 years; 43.3% girls) who received 1 mg anastrozole daily in addition to their regular dose of hydrocortisone, with or without fludrocortisone depending on their salt-wasting status. Fifty-two patients were diagnosed with classic CAH and eight with non-classic CAH.
• Anastrozole treatment lasted for an average of 4.9 years. It was discontinued when the patient’s bone age reached 14 years in girls and 16 years in boys.
• Z-scores of bone age, predicted adult height, and height corrected for bone age were measured 2 years prior to initiation of treatment and over 6 years of treatment with anastrozole.

TAKEAWAY:

• Anastrozole treatment significantly decreased the mean bone age Z-score from 4.2 to 1.3 over 6 years (P < .001).
• The mean predicted adult height Z-score increased from −2.1 at the start of therapy to 0.18 at 6 years of treatment (P < .001).
• Predicted adult height increased by 13 cm in girls (149 cm to 162 cm) and 17 cm in boys (162 cm to 179 cm) over 6 years.
• Height Z-scores adjusted for bone age also gradually increased from −1.7 to 0.18 over 6 years of treatment (P < .001).
• No significant difference was observed in the average total daily hydrocortisone dose used before or during treatment.

IN PRACTICE:

“Anastrozole can be an effective adjunctive therapy in children with CAH and advanced bone age to optimize their growth. Nonetheless, a longitudinal randomized controlled clinical trial is needed to assess long-term outcomes,” the authors wrote.

SOURCE:

The study, led by Heba Al-Rayess, MD, Division of Pediatric Endocrinology, and Rebecca Wiersma, MD, Department of Pediatrics, University of Minnesota Medical School, Minneapolis, was published online in The Journal of Clinical Endocrinology & Metabolism.

LIMITATIONS:

No limitations were provided for this study. 

DISCLOSURES:

The study did not receive any funding. One author reported receiving research support from various sources and serving as a consultant for Neurocrine Biosciences and Spruce Biosciences.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.