Efficacy, safety and satisfaction of using emicizumab in hemophilia A patients without factor VIII inhibitors: A systematic review

Background

Hemophilia A is a genetic disorder characterized by deficiency or dysfunction of the factor VIII clotting protein, leading to serious bleeding disorders. Conventional treatment involves the exogenous administration of factor VIII. However, this therapy faces significant challenges, including 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 using the MEDLINE, SciELO, and LILACS databases. The included studies were original articles on the use of emicizumab in hemophilia A patients without inhibitors and reviews, short communications, expert comments, and case reports were excluded. Data extraction and analysis were performed using predefined criteria.

Results

A total of 471 articles were identified, with 28 meeting the inclusion criteria. Studies demonstrated robust evidence of the efficacy of emicizumab in reducing bleeding episodes, with significant reductions in the Annualized Bleeding Rate and 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 advancement in hemophilia A treatment, offering superior 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.