Managing Lysosomal Acid Lipase Deficiency (LAL-D)

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Lipid-lowering medications (LLMs) are typically used to treat dyslipidemia—a common complication in patients with LAL-D.¹ However, LLMs do not address the underlying cause of LAL-D—accumulation of cholesteryl esters and triglycerides within the lysosome.^1,3 Additionally, they do not prevent liver disease progression, a common complication of LAL-D.^1,2

In an observational study of 48 patients with LAL-D⁴

81%

[39/48]

had reported LLM use

88%

[270/306]

of available LDL-c measurements ≥2.6 mmol/L

It is important to monitor disease progression in patients with LAL-D who are taking LLMs. In a case review of 27 patients with LAL-D on statins:¹

56%

[15/27]

had liver biopsy findings showing fibrosis or cirrhosis

44%

[12/27]

underwent repeat biopsies, demonstrating progressive liver disease; of these, 50% (6/12) progressed to liver transplant or death

Liver transplant does not address the underlying cause of the disease—the deficiency of the vital LAL enzyme¹—and does not halt disease progression in extrahepatic organs, including the heart and kidneys.¹Approximately 33% (4/12) of patients who received liver transplant experienced transplant rejection, congestive heart failure, renal failure, or death.¹

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