Eficacia de Imiglucerasa como tratamiento sustitutivo enzimático para Enfermedad de Gaucher.

Abstract

Abstract: Gaucher disease (GD) is considered a metabolic disorder. standing out as the most common of the lysosomal storage diseases in the most cases as a hereditary lysosomal disease that, secondary to deficiency in the activity of the enzyme glucocerebrosidase (GCase) generates a accumulation in macrophages of the substrate glucocerebroside in the liver, lungs, brain and bone marrow cells, thus producing glucosylsphingosine significantly complicating the relative course of the disease under study. The disease is classified into 3 subtypes, but our study focus is directed to type 1, due to its high prevalence rate and impact on diagnosed patients. In reference to the treatment of Gaucher disease type 1, Imiglucerase is the which is currently administered and has a health registry in Ecuador, with a efficient response to the treatment of diagnosed cases. Objective: Analyze the clinical utility of Imiglucerase as an enzyme replacement treatment in Gaucher disease, to be applied in Ecuador. Methodology: The search in three databases: Pubmed, Lilacs and Science Direct, using Boolean descriptors and operators; In addition, three synonyms for each term main to improve the sensitivity of the search, resulting in Imiglucerase is enzyme replacement therapy for he and patients treatment with Gaucher disease type 1.