The Endocrine and Metabolic Characteristics of a Large Bardet-Biedl Syndrome Clinic Population

J Clin Endocrinol Metab. 2018 May 1;103(5):1834-1841. doi: 10.1210/jc.2017-01459.

Abstract

Context: Bardet-Biedl syndrome (BBS) is a rare autosomal recessive disorder in which previous reports have described obesity and a metabolic syndrome.

Objective: We describe the endocrine and metabolic characteristics of a large BBS population compared with matched control subjects.

Design: We performed a case-control study.

Setting: This study was performed at a hospital clinic.

Patients: Study patients had a clinical or genetic diagnosis of BBS.

Main outcome measurements: Our study determined the prevalence of a metabolic syndrome in our cohort.

Results: A total of 152 subjects were studied. Eighty-four (55.3%) were male. Mean (± standard deviation) age was 33.2 ± 1.0 years. Compared with age-, sex-, and body mass index-matched control subjects, fasting glucose and insulin levels were significantly higher in subjects with BBS (glucose: BBS, 5.2 ± 1.2 mmol/L vs control, 4.9 ± 0.9 mmol/L, P = 0.04; insulin: BBS, 24.2 ± 17.0 pmol/L vs control, 14.2 ± 14.8 pmol/L, P < 0.001). Serum triglycerides were significantly higher in subjects with BBS (2.0 ± 1.2 mmol/L) compared with control subjects (1.3 ± 0.8 mmol/L; P < 0.001), but total cholesterol, high-density lipoprotein, and low-density lipoprotein were similar in both groups. Systolic blood pressure was higher in the BBS group (BBS, 135 ± 18 mm Hg vs control subjects, 129 ± 16 mm Hg; P = 0.02). Alanine transaminase was raised in 34 (26.8%) subjects with BBS, compared with five (8.9%) control subjects (P = 0.01). The rate of metabolic syndrome, determined using International Diabetes Federation criteria, was significantly higher in the BBS group (54.3%) compared with control subjects (26% P < 0.001). Twenty-six (19.5%) of male subjects with BBS were hypogonadal (serum testosterone, 9.9 ± 5.3 mmol/L), but significant pituitary abnormalities were uncommon. Subclinical hypothyroidism was present in 24 of 125 (19.4%) patients with BBS, compared with 3 of 65 (4.6%) control subjects (P = 0.01).

Conclusions: Insulin resistance and the metabolic syndrome are increased in adult patients with BBS compared with matched control subjects. Increased subclinical hypothyroidism in the BBS cohort needs further investigation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Bardet-Biedl Syndrome / complications
  • Bardet-Biedl Syndrome / epidemiology*
  • Bardet-Biedl Syndrome / genetics
  • Bardet-Biedl Syndrome / metabolism*
  • Body Mass Index
  • Case-Control Studies
  • Female
  • Hospitals
  • Humans
  • Insulin Resistance / genetics
  • Male
  • Metabolic Syndrome / complications
  • Metabolic Syndrome / epidemiology*
  • Metabolic Syndrome / genetics
  • Middle Aged
  • Obesity / complications
  • Obesity / epidemiology
  • Obesity / genetics
  • Prevalence
  • Sample Size
  • Young Adult