Nonmalignant Adrenocorticotrophic Hormone-Independent Cushing’s Syndrome in Pediatric Patients: A Retrospective Observational Cohort Study
Authors' conclusion
ACTH-independent CS is rare but can lead to significant burden in children. Early diagnosis and management are essential. New drugs targeting adrenal steroid synthesis are awaited.
Study context
- Population
- Patients aged 0–18 years diagnosed with adrenocorticotrophic hormone (ACTH)-independent Cushing's syndrome (CS) 1992–2022, three Paris pediatric hospitals1 with no etiology found
- Participants
- 15 patients
- Exposure
- McCune-Albright syndrome (MCAS) vs primary pigmented nodular adrenocortical disease (PPNAD)/CNC etiology of ACTH-independent CS
- MCASn = 7
- CNCn = 7reference
- Outcome
- Growth retardation at diagnosis
Methodological detailsobservational, multicentre
- Observational
- Multicentre
Main finding
As reported in the abstract
MCAS
−4.75
SDS
CNC
−1
SDS
P = 0.006
Observational data: associations, not effects.
Further findings
Age at CS diagnosis
| Outcome | MCAS | CNC |
|---|---|---|
| Learning disabilities | 7 events | 2 events |
| Treatment | ||
| GH therapy | 0 | 4 |
| Growth at end of follow-up | −1.5 SDS | −0.5 SDS |
- Intrauterine growth retardation and liver dysfunction at diagnosis · higher in MCAS
- P = 0.01
- Intrauterine growth retardation and liver dysfunction at diagnosis · higher in MCAS
- P = 0.04
- Treatment — Bilateral adrenalectomy
- 12 out of 15
Growth at end of follow-up
- Growth recovered in both groups, less in MCAS, in which liver dysfunction often persisted
Original publication
Nonmalignant Adrenocorticotrophic Hormone-Independent Cushing’s Syndrome in Pediatric Patients: A Retrospective Observational Cohort Study. Horm Res Paediatr. Published 25 March 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →