A Case of Lysosomal Acid Lipase Deficiency Confirmed by Response to Sebelipase Alfa Therapy

J Pediatr Gastroenterol Nutr. 2020 Dec;71(6):726-730. doi: 10.1097/MPG.0000000000002870.

Abstract

Lysosomal acid lipase (LAL) deficiency, or cholesterol ester storage disease, is a disorder affecting the breakdown of cholesterol esters and triglycerides within lysosomes. Clinical findings include hepatomegaly, hepatic dysfunction, and dyslipidemia with a wide range of phenotypic variability and age of onset. The available clinical and molecular information of the patient presented herein was consistent with a diagnosis of LAL deficiency, but her LAL activity assay repeatedly showed normal or borderline low results. Her response to enzyme replacement therapy and demonstrable deficiency on a newer specific enzymatic assay ultimately confirmed her diagnosis of LAL deficiency.

Publication types

  • Case Reports

MeSH terms

  • Cholesterol Ester Storage Disease* / diagnosis
  • Cholesterol Ester Storage Disease* / drug therapy
  • Cholesterol Ester Storage Disease* / genetics
  • Female
  • Humans
  • Sterol Esterase* / genetics
  • Sterol Esterase* / therapeutic use
  • Wolman Disease* / diagnosis
  • Wolman Disease* / drug therapy
  • Wolman Disease* / genetics

Substances

  • Sterol Esterase
  • Sebelipase alfa