Patient Preference Research: Preferred Adjunctive Medication Attributes of Adult Patients with Classic Congenital Adrenal Hyperplasia

    November 2022 in “ Journal of the Endocrine Society
    Rachel Manahan, Jake Macey, Mallory Farrar, Conor O’Donoghue, Sarah Knight, Chloe Carmichael, Chris Marshall, Charles Yonan, Erik A. Imel, Maamoun Salam, Brendan Mulhern
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    TLDR Adults with classic congenital adrenal hyperplasia value medication that prevents weight gain from glucocorticoids the most.
    In a study involving 118 adults with classic congenital adrenal hyperplasia (CAH), a discrete choice experiment (DCE) was conducted to assess patient preferences for attributes of a potential new adjunctive medication. The study found that the most valued attribute was the complete avoidance of glucocorticoid-induced weight gain, with a coefficient of 1.109, which was twice as important as other attributes such as reducing the risk of health conditions like type 2 diabetes (0.540), osteopenia/osteoporosis/fractures (0.521), and cardiovascular disease (0.502), as well as improving fatigue (0.439), fertility (0.437), and excessive body hair/acne (0.410). Preferences varied among subgroups, with females and individuals with a body mass index ≥30 placing a higher importance on avoiding weight gain. Males and younger participants valued fertility improvement more, while those with excessive hair growth or energy problems had higher preferences for improvements in hirsutism/acne and fatigue, respectively. The findings suggest that a novel adjunctive therapy that mitigates glucocorticoid-induced weight gain would be highly valued by patients with classic CAH.
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