ESPE2024 Poster Category 1 Adrenals and HPA Axis 3 (8 abstracts)
Negative Impact of Supraphysiological Glucocorticoid Dosing on Growth and Changes in Glucocorticoid Dose and Androstenedione Health States in Patients with Classic Congenital Adrenal Hyperplasia: Results from the CAHtalog™ Patient Registry
Oksana Lekarev 1 , Ginny Sen 2 , Mahroz Haider 3 , Fei Tang 3 , Haley Friedler 4 , Joy Chen 4 , Karen Lin-Su 1 , Dina Matos 5 , Jackie Dessibourg 2 , Henry Cheng 2 , Eiry Roberts 2 , Jean Chan 2 & George Jeha 2
1Weill Cornell Medicine, New York, USA. 2Neurocrine Biosciences, Inc., San Diego, USA. 3Cytel, Toronto, Canada. 4PicnicHealth, San Francisco, USA. 5CARES Foundation, Union, USA
Introduction: Patients with classic congenital adrenal hyperplasia (CAH) require glucocorticoid (GC) therapy to replace cortisol insufficiency and reduce excess adrenal androgens, usually necessitating supraphysiologic GC doses. CAHtalog™, developed in partnership with CARES Foundation using the PicnicHealth platform, is a real-world US patient registry that characterizes the natural history of classic CAH. This study explored changes in health states based on GC dose and androstenedione levels and negative impacts of supraphysiologic GC dose on growth in patients with classic CAH participating in CAHtalog.
Methods: Deidentified data were abstracted from medical records of participating pediatric and adult patients with classic CAH and reviewed for GC dose and androstenedione levels. GC dose (in hydrocortisone equivalents) was categorized as lower or higher: pediatric (≤11 or >11-30 mg/m2/d); adult (≤20 or >20-60 mg/d). Androstenedione was categorized as above or below the upper limit of normal (ULN) for sex and age. Health states were determined by matching an androstenedione category to the nearest GC category. Pediatric growth outcomes including body mass index, height, and bone age were analyzed by GC dose, defined as “low-medium” (≤15 mg/m2/d) or “high” (>15 mg/m2/d) based on feasible sample sizes.
Results: Among 40 pediatric and adult patients with ≥3 matched GC-androstenedione records, 37 (92.5%) had ≥1 health state transitions during the observation period (median: 6.5 years). Higher GC dose with androstenedione <ULN was the most common health state (47.5%) at the first matched record, but only 3 (7.5%) patients maintained this health state throughout the observation period. Moreover, 9 (22.5%) patients had lower GC and androstenedione <ULN at the start of observation; however, no patients sustained this health state throughout. Among 44 pediatric patients with available growth data, 12 (27.3%) received high GC doses. Patients with high GC doses had premature adiposity rebound starting at age ~2 years (earlier than the usual age of ~6 years) and early growth acceleration (attributed to advanced bone age), followed by blunted pubertal growth.
Conclusion: CAHtalog registry data provide real-world insight from patients with classic CAH on the adverse impact of supraphysiologic GC dose on pediatric growth and the challenges of achieving and maintaining lower androstenedione while on lower GC doses. The transitions in health states suggest that with existing treatment approaches, disease control today does not equal disease control over time. This highlights the need for non-GC treatment options that can reduce androgen excess without supraphysiologic GC doses in patients with classic CAH.
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