Neurocrine (NBIX) presented a systematic literature review illustrating the relationship between higher glucocorticoid dose and adverse clinical outcomes, including decreased bone mineral density, increased insulin resistance and higher body mass index, in patients with congenital adrenal hyperplasia. This study was presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research Annual Meeting in Montreal. Congenital adrenal hyperplasia, or CAH, is a rare, lifelong genetic condition linked to overproduction of adrenal androgens and cortisol deficiency. CAH is traditionally treated with lifelong high-dose glucocorticoids or GCs, which increase the risk of complications across multiple aspects of health. This systematic literature review is the first to capture the impact of higher GC dose on the incidence and severity of all relevant adverse clinical outcomes in patients with CAH. More than half of the publications found statistically significant associations between GC dose and clinical outcomes.
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