PSAT082 A Survey of Patients Living with CAH Assessing Their Perceptions on Glucocorticoid Regimen and Glucocorticoid-Associated Side Effects

BACKGROUND: Congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD) is an autosomal recessive disorder characterized by insufficient cortisol production resulting in excess adrenal androgen production. The standard of care to replace the insufficient cortisol production is the lifelong use of glucocorticoids (GCs), usually at supraphysiologic levels to control hyperandrogenemia. Chronic use of supraphysiologic GCs is associated with long-term comorbidities. The degree of patient awareness of GC associated comorbidities is unknown and of critical importance to focus patient education. METHODS: An online survey was conducted in the US and promoted through a nationally known patient advocacy organization and through social media. Survey questions assessed the perceptions of patients living with CAH regarding their GC regimens, most common side effects associated with GCs, patients understanding of the risk and benefit of GC use and patients’ attitude about adjusting their GC regimen. RESULTS: A total of 113 patients living with CAH completed the online survey. Over 97.5% reported taking GCs at the time of the survey, with 75% being satisfied and 85% rating their regimen effective or very effective. Commonly reported side-effects associated with GC use included increased weight gain and appetite, fatigue, "moon face," thin skin that bruises easily, and mood changes or depression. Less common side effects included osteoporosis, muscle weakness, high blood pressure and increased infections. Approximately half reported discussing the long-term risks of GC treatment with their healthcare provider, with the remaining half reported not having this discussion or were unsure. Approximately 43% of respondents reported being in situations where they were not given enough information about the risk of supraphysiologic GC use to make an informed decision on treatment. A majority of respondents (67%) reported a willingness to change their current regimen if there was a safe and effective way to reduce their current GC dose closer to physiologic levels. CONCLUSIONS: While a majority of patients living with CAH are satisfied with their current GC regimen, there is a general lack of patient awareness of the long-term side effects associated with supraphysiologic GCs. Patients may benefit from more patient education to make informed decisions with their healthcare providers. Presentation: Saturday, June 11, 2022 1:00 p.m. - 3:00 p.m.