(8.14) Glucocorticoid Treatment Patterns in Pediatric and Adult Patients with Classic Congenital Adrenal Hyperplasia: Results from the CAHtalog Registry

Objective : Congenital adrenal hyperplasia (CAH) requires lifelong supraphysiological glucocorticoid (GC) therapy, leading to complications during pediatric and adult life stages. Treatment guidelines provide general recommendations, but real-world practices differ by practitioner and center, making it difficult to compare data over time. CAHtalog is an active patient registry of retrospective and prospective real-world data on patients with classic CAH in the United States, developed in partnership with CARES Foundation on the PicnicHealth platform. This study characterized current patterns of GC treatment in patients with CAH participating in CAHtalog.

Methods: All available medical records for adult and pediatric patients in CAHtalog were collected. Clinical data were abstracted from medical records and structured into a de-identified dataset. Patient demographics, total daily GC dose, and GC type in CAHtalog as of data download (October 2023) were described for adult and pediatric patients (³18 and < 18 years, respectively, at all observed times).

Results: Data were available for 33 adult patients (mean age: 36.3±12.8 years at data download; 70% female) and 44 pediatric patients (mean age: 11.5±6.5 years; 57% female). From the first through last medical record with relevant ages (≥18 or < 18 years), median follow-up durations were 14 years (adult) and 9 years (pediatric). Most patients (90%) were treated with mineralocorticoids. Among adults with ≥1 GC record (N=33), hydrocortisone (HC) monotherapy was the most common (67%) with a mean dose of 23.2±8.0 mg/d, followed by dexamethasone (DEX) monotherapy (42%, 0.8±0.9 mg/d) and prednisone monotherapy (24%, 5.1±2.9 mg/d); 42% of patients had ≥3 changes in treatment regimen. Among pediatric patients with ≥1 GC record (N=44), HC monotherapy was the most common (86%) with a mean dose of 15.6±15.7 mg/m²/d, followed by DEX monotherapy (11%, 0.9±0.9 mg/m²/d) and HC-DEX combination therapy (11%, 28.7±17.2 mg/m²/d HC equivalents [60x conversion factor]); 23% had ≥3 changes in treatment regimen.

Discussion/Conclusion: CAHtalog was designed to expand foundational knowledge of classic CAH. This analysis shows that HC is the most common GC prescribed in both adult and pediatric patients with CAH, and the average total daily dose tends to be at the upper end of the recommended ranges (Speiser JCEM 2018). Treatment regimens for patients with CAH are characterized by complexity with frequent changes throughout their life, emphasizing the need for comprehensive management.