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A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
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A PHASE 3 TRIAL OF SEBELIPASE ALFA IN LYSOSOMAL ACID LIPASE DEFICIENCY
STUDENT: EMILY BORGOÑO QUIROZ.
Definition
Lysosomal acid lipase is a essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolim.
Invastigators conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirhosis and severe dyslipidemia.
DEFINITION
Subtopic
Application in nursing
In this multicenter, randomized, double-blind, placebo-controlled study involving 66 patients, we evaluated the safety and effectiveness of enzyme-replacement therapy with sebelipase alfa (administered intravenously at a dose of 1 mg per kilogram of body weight every other week.
APPLICATION IN NURSING
Title
PROS-CONS
PROS:
Secondary end points included additional disease-related efficacy assessments, safety, and side-effect profile.
CONS:
Substantial disease burden at baseline included a very high level of low-density lipoprotein cholesterol (190 mg per deciliter) in 38 of 66 patients (58%) and cirrhosis in 10 of 32 patients (31%) who underwent bipsy.
PROS-CONS
Title
ISSUE APPLIED IN ECUADOR
ISSUE IN ECUADOR
The placebo-controlled phase of the study was 20 weeks long and was followed by open-label treatment for all patients.
The primary end point was normalization of the alanine aminotransferase level.
Title
CONCLUSION
Sebelipase alfa therapy resulted in a reduction in multiple disease-related hepatic and lipid abnormalities in children and adults with lysosomal acid lipase deficiency.