Pediatric and Adolescent Endocrinology

SAT-208 - Significant Heterogeneity in Hormonal Control in Classical Congenital Adrenal Hyperplasia Highlights the Limitations of Standard Glucocorticoid Dosing.

Saturday, June 13, 2026

9:30 AM - 4:30 PM CT

Location: ENDOExpo; Poster Floor

Abstract Poster Presenter(s)

JM

Joshua Matthew, Medical student MS3

Medical student MS3
Rutgers Robert Wood Johnson Medical School, New Brunswick, NJ, US
New Brunswick, New Jersey, United States

Co-Author(s)

EL

Erica Lupiano, BS

Short Hills, NJ, United States
AB

Amrit Bhangoo, MD (he/him/his)

Pediatric Endocrinologist
Children Hospital of Orange County
Orange, California, United States
DH

Deboragh Horlacher, APN

APN
Cooperman Barnabas and Newark Beth Israel Medical Center, RWJBarnabas Health, NJ, US
Livingston, New Jersey, United States
HC

Hope-Elizabeth Clennon, DO

Pediatric Endocrinologist
Cooperman Barnabas and Newark Beth Israel Medical Center, RWJBarnabas Health, NJ, US
Livingston, New Jersey, United States
FB

Frank Barrows, DO

Pediatric Endocrinologist
Unterberg Children’s Hospital Monmouth Medical Center, RWJBarnabas Health
Long Branch, New Jersey, United States
LN

Laura Novello, MD

Pediatric Endocrinologist
Unterberg Children’s Hospital Monmouth Medical Center, RWJBarnabas Health
Long Branch, New Jersey, United States
IM

Irene Mamkin, MD

Pediatric Endocrinologist
Cooperman Barnabas and Newark Beth Israel Medical Center, RWJBarnabas Health
Livingston, New Jersey, United States
MJ

Marianne Jacob, DO

Pediatric Endocrinologist
Cooperman Barnabas and Newark Beth Israel Medical Center, RWJBarnabas Health
Livingston, New Jersey, United States
DB

Dennis Brenner, MD

Pediatric Endocrinologist
Cooperman Barnabas and Newark Beth Israel Medical Center, RWJBarnabas Health
West Orange, New Jersey, United States
MG

Mariam Gangat, MD

Pediatric Endocrinologist
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
IM

Ian Marshall, MD

Pediatric Endocrinologist
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
AK

Ahmed Khattab, MD

Pediatric Endocrinologist
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States

Disclosure(s):

Dennis Brenner, MD: No relevant disclosure to display

Svetlana Ten, MD: BioMarin: speaker ()

Background
Congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency requires glucocorticoid therapy. However, patients show marked variability in ACTH-dependent adrenal steroids (17OHP and Androstenedione (A4) response, suggesting differences in glucocorticoid sensitivity or glucocorticoid metabolism.
Methods
We retrospectively reviewed the records of 50 patients, including 49 children and one 48-year-old adult male, all diagnosed with classical CAH (35 with salt-wasting form and 15 with simple virilizing). They were stratified by 17-hydroxyprogesterone (17-OHP) levels, and glucocorticoid doses were compared across these groups.
Results
Patients were divided into four groups based on the degree of hormonal control.
Group 1. Good control, 21 patents (42%), 8 ± 5.6 yrs, 7 females, 17 OHP less than 2000 ng/dl
17-OHP of 596 ± 584 ng/dL and A4 of 48 ± 65 ng/dL
Group 2. Satisfactory, 9 patients (18%), 11.8 ± 6.2 yrs, 3 females, 17 OHP less than 4000 ng/dl
17-OHP of 3200 ± 592 ng/dL and A4 of 260 ± 376 ng/dL
Group 3. Fair, 8 patients (16%), 14.6 ± 8.1 yrs, 1 female, 17 OHP less than 8000 ng/dl
17-OHP of 5462 ± 941 ng/dL and A4 of 449 ± 343 ng/dL
Group 4. Poorly controlled, 12 patients (24%), 14.9 ±5.6 yrs, 4 females, 17 OHP more than 8000 ng/dl,
(17-OHP of 16424 ± 9557 ng/dL and A4 of 1021 ± 555 ng/dL)
Despite similar glucocorticoid doses across groups (12-16 mg/m2/day; P > 0.05), differences in hormonal control were significant both clinically and statistically (P < 0.01). Notably, 24% of patients (Group 4) demonstrated severely elevated adrenal steroid levels, with a mean 17-OHP of 16424 ± 9557 ng/dL and A4 of 1021 ± 555 ng/dL. An additional 14% (Group 3) exhibited moderately elevated 17-OHP and A4. Together, this indicates that approximately 40% of patients had suboptimal hormonal control despite receiving comparable glucocorticoid doses. In Group 4, 10 out of the 12 patients received steroid doses more than 12 mg/m2 (ranging from 13 to 30.5 mg/m2/day)

Conclusion
A significant proportion of patients with classical CAH fail to achieve adequate biochemical control despite high glucocorticoid doses, supporting variability in glucocorticoid sensitivity. Approximately 40% exhibited moderate to severe hormonal dysregulation, including 24% with markedly elevated androgen levels. These findings support the importance of individualizing treatment, not only by dose escalation. Non-glucocorticoid therapy such as Crinecerfont can be an effective adjunctive approach by decreasing ACTH-mediated androgen production.

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