Neurocrine Biosciences, Inc. presented a systematic literature review illustrating the relationship between higher glucocorticoid (GC) dose and adverse clinical outcomes in patients with congenital adrenal hyperplasia (CAH). This study was presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Annual Meeting.
The review, the first to capture the impact of higher GC dose on the incidence and severity of all relevant adverse clinical outcomes in CAH patients, identified 105 publications. More than half (62%) found statistically significant associations between GC dose and clinical outcomes, including decreased bone mineral density, increased insulin resistance, and higher body mass index.
This highlights the significant clinical burden of long-term, high-dose glucocorticoid use in CAH patients and the challenges clinicians face in optimizing treatment. The emergence of new therapeutic options, such as CRENESSITY, offers hope for potentially reducing reliance on high-dose glucocorticoids, representing an important advancement in CAH management.
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