PCR114 The Adjunctive Therapy Preferences of Adults with Classic Congenital Adrenal Hyperplasia: A Discrete Choice Experiment

نویسندگان

چکیده

Patients with classic congenital adrenal hyperplasia (CAH) often require supraphysiologic doses of glucocorticoids to replace missing cortisol and reduce androgens. Current treatments aim control androgen-driven symptoms/impacts but can result in glucocorticoid-related side effects/complications. This study aimed understand the preferences adults CAH for benefits a potential new adjunctive therapy that could provide better androgen lower glucocorticoid via an online discrete choice experiment (DCE). Relevant data qualitative literature were reviewed eight interviewed disease treatment experience. informed development seven attributes reflected hypothetical benefits. Following input from two endocrinologists, cognitive testing three pilot launch, DCE survey collected 118 analyzed using conditional logit regression. Initial interviews (N=8, 50% female, age 19–67) revealed medication management was main daily impact CAH. Participants accustomed self-managing multiple medications/doses most willing try additional, twice-daily addressed their top five or The (N=118, 75% 19–69) following priorities (all coefficients p<0.001 vs baseline no change): complete avoidance glucocorticoid-driven weight gain (1.109); moderate decrease risk developing long-term complications: type 2 diabetes (0.540), osteopenia, osteoporosis, fractures (0.521) cardiovascular (0.502); improvement fatigue (0.439), fertility (0.437), hirsutism (females only) acne (0.410). Adults valued by twice magnitude other Reduced effects over improvements fatigue, hirsutism/acne. These preference patterns may inform where therapies have meaningful on patients.

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ژورنال

عنوان ژورنال: Value in Health

سال: 2022

ISSN: ['1098-3015', '1524-4733']

DOI: https://doi.org/10.1016/j.jval.2022.04.1457