Patients With Aldolase B Deficiency Are Characterized by Increased Intrahepatic Triglyceride Content

J Clin Endocrinol Metab. 2019 Nov 1;104(11):5056-5064. doi: 10.1210/jc.2018-02795.

Abstract

Context: There is an ongoing debate about whether and how fructose is involved in the pathogenesis of nonalcoholic fatty liver disease (NAFLD). A recent experimental study showed an increased intrahepatic triglyceride (IHTG) content in mice deficient for aldolase B (aldo B-/-), the enzyme that converts fructose-1-phosphate to triose phosphates.

Objective: To translate these experimental findings to the human situation.

Design: Case-control study.

Setting: Outpatient clinic for inborn errors of metabolism.

Patients or other participants: Patients with hereditary fructose intolerance, a rare inborn error of metabolism caused by a defect in aldolase B (n = 15), and healthy persons matched for age, sex, and body mass index (BMI) (n =15).

Main outcome measure: IHTG content, assessed by proton magnetic resonance spectroscopy.

Results: IHTG content was higher in aldo B-/- patients than controls (2.5% vs 0.6%; P = 0.001) on a background of lean body mass (median BMI, 20.4 and 21.8 kg/m2, respectively). Glucose excursions during an oral glucose load were higher in aldo B-/- patients (P = 0.043). Hypoglycosylated transferrin, a surrogate marker for hepatic fructose-1-phosphate concentrations, was more abundant in aldo B-/- patients than in controls (P < 0.001). Finally, plasma β-hydroxybutyrate, a biomarker of hepatic β-oxidation, was lower in aldo B-/- patients than controls (P = 0.009).

Conclusions: This study extends previous experimental findings by demonstrating that aldolase B deficiency also results in IHTG accumulation in humans. It suggests that the accumulation of fructose-1-phosphate and impairment of β-oxidation are involved in the pathogenesis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • 3-Hydroxybutyric Acid / blood
  • Adult
  • Blood Glucose / metabolism
  • Body Composition
  • Body Mass Index
  • Case-Control Studies
  • Diet
  • Female
  • Fructose Intolerance / diagnostic imaging
  • Fructose Intolerance / metabolism*
  • Fructose-Bisphosphate Aldolase / deficiency*
  • Glucose / metabolism
  • Humans
  • Liver / diagnostic imaging
  • Liver / metabolism*
  • Magnetic Resonance Imaging
  • Male
  • Metabolism, Inborn Errors / metabolism
  • Middle Aged
  • Transferrin / analysis
  • Triglycerides / metabolism*
  • Young Adult

Substances

  • Blood Glucose
  • Transferrin
  • Triglycerides
  • Fructose-Bisphosphate Aldolase
  • Glucose
  • 3-Hydroxybutyric Acid