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A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

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.

SCHOOL UNIVERSITY CATHOLIC

NAME: JIMENA CHAVEZ.

FACEBOOK: JIMENA CHAVE

Name: Jimena Chávez Tarambis

Teacher : Jhon Moscoso

Facebook : Jimena Chávez

Twitter: @Jimena1979Rocio

Correo: jimena1979@hotmail.es

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 biopsy.

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.

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.

Definition

Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.

jimena chavez

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