Assessment of central adrenal insufficiency in children and adolescents with Prader-Willi syndrome

Clin Endocrinol (Oxf). 2012 Jun;76(6):843-50. doi: 10.1111/j.1365-2265.2011.04313.x.

Abstract

Objective: A recent study evidenced by metyrapone test a central adrenal insufficiency (CAI) in 60% of Prader-Willi syndrome (PWS) children. These results were not confirmed in investigations with low [Low-Dose Tetracosactrin Stimulation Test (LDTST), 1 μg] or standard-dose tetracosactrin stimulation tests. We extended the research by LDTST in paediatric patients with PWS.

Design: Cross-sectional evaluation of adrenal stress response to LDTST in a PWS cohort of a tertiary care referral centre.

Patients: Eighty-four children with PWS.

Measurements: Assessment of adrenal response by morning cortisol and ACTH dosage, and 1-μg tetracosactrin test. Response was considered appropriate when cortisol reached 500 nm; below this threshold, patients were submitted to a second test. Responses were correlated with the patients' clinical and molecular characteristics to assess genotype-phenotype correlation.

Results: Pathological cortisol peak responses to the LDTST were registered in 12 patients (14.3%) who had reduced basal (169.4 ± 83.3 nm) and stimulated (428.1 ± 69.6 nm) cortisol levels compared to patients with normal responses (367.1 ± 170.6 and 775.9 ± 191.3 nm, P < 0.001). Body mass index standard deviation score was negatively correlated with basal and peak cortisol levels (both P < 0.001), and the patients' ages (P < 0.001). In patients with deletion on chromosome 15, the cortisol peak was significantly lower than that in uniparental disomy (UPD) cases (P = 0.030). At multiple regression analysis, the predictors of peak response were basal cortisol, age, and UPD subclass (r(2) = 0.353, P < 0.001). Standard-dose (250 μg) tetracosactrin test confirmed CAI in 4/12 patients (4.8% of the cohort).

Conclusions: Our results support the hypothesis that, albeit rare, CAI may be part of the PWS in childhood.

MeSH terms

  • Adolescent
  • Adrenal Insufficiency / blood
  • Adrenal Insufficiency / physiopathology*
  • Adrenocorticotropic Hormone / blood
  • Body Mass Index
  • Child
  • Child, Preschool
  • Cross-Sectional Studies
  • Female
  • Humans
  • Hydrocortisone / blood
  • Infant
  • Infant, Newborn
  • Male
  • Prader-Willi Syndrome / blood
  • Prader-Willi Syndrome / physiopathology*
  • Regression Analysis

Substances

  • Adrenocorticotropic Hormone
  • Hydrocortisone