학술논문
Prophylaxis with emicizumab in children under 12 years old with haemophilia A without inhibitors in Uruguay: National experience.
Document Type
Article
Author
Source
Subject
*EMICIZUMAB
*BISPECIFIC antibodies
*BLOOD coagulation factor VIII antibodies
*HEMOPHILIA
*ABO blood group system
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Language
ISSN
1351-8216
Abstract
This article discusses the use of emicizumab, a medication used to treat haemophilia A (HA), in children under 12 years old in Uruguay. HA is a genetic disorder characterized by a deficiency of coagulation factor VIII. The standard treatment for severe HA involves regular prophylaxis with factor VIII concentrates, but this can still leave patients at risk of bleeding. Emicizumab is a monoclonal antibody that mimics the function of factor VIII and has shown efficacy in preventing bleeding in adult and teenage patients. This study aimed to assess the efficacy and safety of emicizumab in a pediatric population in Uruguay. The results showed that emicizumab was effective in reducing bleeding episodes, with a low rate of adverse events. This study provides valuable insights into the treatment of HA in Uruguay and contributes to the broader understanding of managing this condition. [Extracted from the article]