Abstract
Introduction
Hemophilia A is a genetic disorder characterized by deficiency or dysfunction of the coagulation protein factor VIII, leading to severe bleeding disorders. Conventional treatment consists of exogenous administration of factor VIII. However, this therapy presents significant challenges, such as the development of inhibitors and the need for frequent intravenous administration. Emicizumab, a recombinant bispecific monoclonal antibody that can be administered subcutaneously, offers a novel therapeutic alternative by mimicking the action of factor VIII.
Methods
This systematic review evaluates the efficacy, safety, and patient satisfaction with emicizumab in patients with hemophilia A without inhibitors. A comprehensive literature search was conducted in the MEDLINE, SciELO, and LILACS databases. The included studies were original articles on the use of emicizumab in patients with hemophilia A without inhibitors, and reviews, brief communications, expert comments, and case reports were excluded. Data extraction and analysis were performed according to predefined criteria.
Results
A total of 471 articles were identified, of which 28 met the inclusion criteria. The studies demonstrated strong evidence of the efficacy of emicizumab in reducing bleeding episodes, with significant reductions in the annualized bleeding rate and the annualized joint bleeding rate. Safety profiles were favorable, with mainly minor adverse events reported. High patient satisfaction scores highlighted improvements in quality of life and treatment adherence.
Conclusion
Emicizumab represents a significant advance in the treatment of hemophilia A, offering greater efficacy, safety, and patient satisfaction compared to traditional therapies. Future research should focus on long-term outcomes and specific subpopulations to further validate these findings.
Isabela de Oliveira Araujo, Lucas Fernandes Suassuna a, Isabela Lima dos Santos b, Daniela de Oliveira Werneck Rodrigues c
a Federal University of Juiz de Fora (UFJF), Juiz de Fora, Minas Gerais, Brazil
b Faculty of Medical and Health Sciences of Juiz de Fora (FCMS/JF), Juiz de Fora, Minas Gerais, Brazil
c Hemominas Foundation, Juiz de Fora, Minas Gerais, Brazil
To download the complete research, click below:
https://www.htct.com.br/pt-efficacy-safety-satisfaction-using-emicizumab-articulo-S2531137925001178
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