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.
منابع مشابه
Classic congenital adrenal hyperplasia.
Congenital adrenal hyperplasia is attributed to inherited enzyme defects in the adrenal cortex. The classical form results in reduced production of cortisol and aldosterone, accompanied by an increase in production of adrenal cortical androgens. This causes virilisation in girls, adrenocortical failure and early puberty in both sexes. This article describes the genetics, clinical picture, diagn...
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Congenital adrenal hyperplasia (CAH) is a rare congenital disorder, which in cases of female genotype may result in virilization. Specific enzyme deficiencies in adrenocorticoid hormones biosynthetic pathway lead to excess androgen production causing virilization. Classic type presents early in infant life as salt losing or simple virilizing type, whereas non classic form presents late at puber...
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Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive disorders resulting from deficiency of one of the five enzymes required for synthesis of cortisol in the adrenal cortex. The most common form of the disease is classic 21-hydroxylase deficiency, which is characterized by decreased synthesis of glucocorticoids and often mineralocorticoids, adrenal hyperandrogenism and impaire...
متن کاملCongenital Adrenal Hyperplasia in Adults
Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder affecting adrenal steroid synthesis. More than 95% of CAH cases are caused by reduced 21hydroxylase function leading to variable extent of cortisol and aldosterone deficiency in addition to androgen excess. The foundation of CAH treatment is the use of glucocorticoids. However, overtreatment leads to Cushing’s syndrome and ...
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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