Long-term outcome of patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency

Am J Med Sci. 2012 Nov;344(5):363-73. doi: 10.1097/MAJ.0b013e31824369e4.

Abstract

Introduction: Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder affecting adrenal steroid synthesis. In this study, the authors aim to evaluate the impact of CAH due to 21-hydroxylase deficiency on final height (FH), bone health, cardiometabolic risk, fertility, neurocognition and quality of life in a hospital-based sample from Tunisia.

Methods: Twenty-six patients (11 males and 15 females; mean age: 27.4 ± 8.2 years) were recruited.

Results: Mean FH was 159.5 ± 9.7 cm. Twenty-one patients (80.7%) had a FH below the target height. Ten patients (38.4%) exhibited bone demineralization. Eight patients (30.7%) had obesity. Lipid profile alterations and carbohydrate metabolism disorders were detected in 10 (38.4%) and 5 (19.2%) patients, respectively. Seven patients (27%) had insulin resistance. Ambulatory blood pressure monitoring showed abnormalities in 6 patients (23%). Increased carotid intima-media thickness was found in 14 patients (53.8%). Inhibin B level was decreased in 4 male patients. Semen analysis showed abnormalities in 4 of 10 patients. Testicular tumors were detected in 6 of 11 patients. Anti-Müllerian hormone level was reduced in 4 female patients. Six patients showed poly-cystic ovary syndrome. Brain magnetic resonance imaging showed abnormalities in 11 patients (42.3%). Quality of life was reduced in 14 of 22 patients (63.6%). Many of the suboptimal outcomes appeared to be related to poor adherence to medication schedules, some to overtreatment.

Conclusion: CAH patients have a number of issues due to the disease or its treatment. Regular follow-up, early lifestyle interventions, bone health assessment, testicular ultrasound and psychological management are needed.

MeSH terms

  • Adrenal Hyperplasia, Congenital / physiopathology*
  • Adrenal Hyperplasia, Congenital / psychology
  • Adult
  • Body Height
  • Bone Density
  • Female
  • Fertility
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Quality of Life
  • Steroid 21-Hydroxylase / metabolism*
  • Treatment Outcome

Substances

  • Steroid 21-Hydroxylase