PSAT097 Patient Preference Research: Preferred Adjunctive Medication Attributes of Adult Patients with Classic Congenital Adrenal Hyperplasia

Manahan, R; Macey, J; Farrar, M; O’Donoghue, C; Knight, S; Carmichael, C; Marshall, C; Yonan, C; Imel, E; Salam, M; Mulhern, B

PSAT097 Patient Preference Research: Preferred Adjunctive Medication Attributes of Adult Patients with Classic Congenital Adrenal Hyperplasia

Manahan, R Macey, J Farrar, M O’Donoghue, C Knight, S Carmichael, C Marshall, C Yonan, C Imel, E Salam, M Mulhern, B

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Publisher:: The Endocrine Society
Publication Type:: Journal Article
Citation:: Journal of the Endocrine Society, 2022, 6, (Suppl 1), pp. a118-a118
Issue Date:: 2022-11-01

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Field	Value	Language
dc.contributor.author	Manahan, R
dc.contributor.author	Macey, J
dc.contributor.author	Farrar, M
dc.contributor.author	O’Donoghue, C
dc.contributor.author	Knight, S
dc.contributor.author	Carmichael, C
dc.contributor.author	Marshall, C
dc.contributor.author	Yonan, C
dc.contributor.author	Imel, E
dc.contributor.author	Salam, M
dc.contributor.author	Mulhern, B https://orcid.org/0000-0003-3656-8063
dc.date.accessioned	2023-03-02T02:54:59Z
dc.date.available	2023-03-02T02:54:59Z
dc.date.issued	2022-11-01
dc.identifier.citation	Journal of the Endocrine Society, 2022, 6, (Suppl 1), pp. a118-a118
dc.identifier.issn	2472-1972
dc.identifier.issn	2472-1972
dc.identifier.uri	http://hdl.handle.net/10453/166620
dc.description.abstract	<jats:title>Abstract</jats:title> <jats:sec> <jats:title>Background</jats:title> <jats:p>People with classic congenital adrenal hyperplasia (CAH) often require supraphysiologic doses of glucocorticoids (e.g., hydrocortisone, dexamethasone) to treat cortisol deficiency and excess androgen production. Healthcare providers and patients continually try to balance androgen control with side effects from supraphysiologic glucocorticoids. This study aimed to understand the preferences of adults with classic CAH regarding benefits of a potential new adjunctive medication that may provide better androgen control and allow for lower glucocorticoid doses.</jats:p> </jats:sec> <jats:sec> <jats:title>Methods</jats:title> <jats:p>Discrete choice experiment (DCE) methodology was used to calculate the relative stated preferences for the additional therapy's hypothetical benefits (attributes). Seven attributes were developed for the DCE after review of relevant data and qualitative literature, interviews with eight adults with classic CAH and input from two endocrinologists. The online DCE survey was piloted via interviews with three adults with classic CAH and a soft launch, which confirmed survey content and functioning. Preference data and subgroup differences were analyzed using conditional logit and scale assessment modelling, respectively.</jats:p> </jats:sec> <jats:sec> <jats:title>Results</jats:title> <jats:p>US adults with classic CAH (N=118, 75% female, age 19–69) valued avoidance of glucocorticoid-induced weight gain by twice the magnitude of other treatment attributes (all coefficients p&lt;0.001 compared to baseline levels of no avoidance/decrease/improvement): complete avoidance of weight gain from glucocorticoids (1.109); moderate decrease in risk of developing health conditions from long-term glucocorticoids: type 2 diabetes (0.540), osteopenia, osteoporosis, fractures (0.521) and cardiovascular disease (0.502); moderate improvement in: fatigue (0.439), fertility (0.437) and excessive body hair (females only) and acne (0.410). Avoidance of glucocorticoid-induced weight gain remained the most preferred attribute across all subgroup analyses. Avoidance of glucocorticoid-induced weight gain was relatively much more important to females (n=89) than males (n=28) and participants with a body mass index ≥30 (n=70) than &lt;30 (n=48). Improvement in fertility was more important to males than females, was valued by participants aged 18–45 (n=82) but was not a significant preference in participants &gt;45 (n=36) and was relatively more important to participants with fertility problems (n=31) than without (n=87). Improvement in hirsutism and acne was highly valued by participants with excessive hair growth (n=38) but had the lowest preference among those without (n=80). Improvement in fatigue was a significant preference for participants with energy problems (n=73) but was not significant for those without (n=45).</jats:p> </jats:sec> <jats:sec> <jats:title>Conclusions</jats:title> <jats:p>The strong and consistent preference for avoiding glucocorticoid-induced weight gain suggests that patients with classic CAH would most value a novel adjunctive therapy that helps mitigate glucocorticoid-induced weight gain. In relation to other potential adjunctive therapy benefits, reduced risk of glucocorticoid-driven side effects may be valued more than improvement in fatigue or androgen-driven adverse events (fertility and hirsutism/acne). However, subgroup analyses indicated that individuals’ characteristics impact their treatment preferences and priorities, after avoidance of glucocorticoid-induced weight gain.</jats:p> <jats:p>Presentation: Saturday, June 11, 2022 1:00 p.m. - 3:00 p.m.</jats:p> </jats:sec>
dc.language	en
dc.publisher	The Endocrine Society
dc.relation.ispartof	Journal of the Endocrine Society
dc.relation.isbasedon	10.1210/jendso/bvac150.240
dc.rights	info:eu-repo/semantics/closedAccess
dc.title	PSAT097 Patient Preference Research: Preferred Adjunctive Medication Attributes of Adult Patients with Classic Congenital Adrenal Hyperplasia
dc.type	Journal Article
utslib.citation.volume	6
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Health
pubs.organisational-group	/University of Technology Sydney/Strength - CHERE - Centre for Health Economics Research and Evaluation
pubs.organisational-group	/University of Technology Sydney/Faculty of Health/Centre for Health Economics Research and Evaluation
utslib.copyright.status	closed_access	*
dc.date.updated	2023-03-02T02:54:58Z
pubs.issue	Suppl 1
pubs.publication-status	Published
pubs.volume	6
utslib.citation.issue	Suppl 1

Abstract:

Abstract Background People with classic congenital adrenal hyperplasia (CAH) often require supraphysiologic doses of glucocorticoids (e.g., hydrocortisone, dexamethasone) to treat cortisol deficiency and excess androgen production. Healthcare providers and patients continually try to balance androgen control with side effects from supraphysiologic glucocorticoids. This study aimed to understand the preferences of adults with classic CAH regarding benefits of a potential new adjunctive medication that may provide better androgen control and allow for lower glucocorticoid doses. Methods Discrete choice experiment (DCE) methodology was used to calculate the relative stated preferences for the additional therapy's hypothetical benefits (attributes). Seven attributes were developed for the DCE after review of relevant data and qualitative literature, interviews with eight adults with classic CAH and input from two endocrinologists. The online DCE survey was piloted via interviews with three adults with classic CAH and a soft launch, which confirmed survey content and functioning. Preference data and subgroup differences were analyzed using conditional logit and scale assessment modelling, respectively. Results US adults with classic CAH (N=118, 75% female, age 19–69) valued avoidance of glucocorticoid-induced weight gain by twice the magnitude of other treatment attributes (all coefficients p<0.001 compared to baseline levels of no avoidance/decrease/improvement): complete avoidance of weight gain from glucocorticoids (1.109); moderate decrease in risk of developing health conditions from long-term glucocorticoids: type 2 diabetes (0.540), osteopenia, osteoporosis, fractures (0.521) and cardiovascular disease (0.502); moderate improvement in: fatigue (0.439), fertility (0.437) and excessive body hair (females only) and acne (0.410). Avoidance of glucocorticoid-induced weight gain remained the most preferred attribute across all subgroup analyses. Avoidance of glucocorticoid-induced weight gain was relatively much more important to females (n=89) than males (n=28) and participants with a body mass index ≥30 (n=70) than <30 (n=48). Improvement in fertility was more important to males than females, was valued by participants aged 18–45 (n=82) but was not a significant preference in participants >45 (n=36) and was relatively more important to participants with fertility problems (n=31) than without (n=87). Improvement in hirsutism and acne was highly valued by participants with excessive hair growth (n=38) but had the lowest preference among those without (n=80). Improvement in fatigue was a significant preference for participants with energy problems (n=73) but was not significant for those without (n=45). Conclusions The strong and consistent preference for avoiding glucocorticoid-induced weight gain suggests that patients with classic CAH would most value a novel adjunctive therapy that helps mitigate glucocorticoid-induced weight gain. In relation to other potential adjunctive therapy benefits, reduced risk of glucocorticoid-driven side effects may be valued more than improvement in fatigue or androgen-driven adverse events (fertility and hirsutism/acne). However, subgroup analyses indicated that individuals’ characteristics impact their treatment preferences and priorities, after avoidance of glucocorticoid-induced weight gain. Presentation: Saturday, June 11, 2022 1:00 p.m. - 3:00 p.m.

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