PB0215 - Efficacy, effectiveness, and safety of emicizumab for the treatment of people with hemophilia A: a systematic review

Background: Hemophilia A (HA) is a rare bleeding disorder due to reduced or complete absence of the clotting activity of the factor VIII (FVIII). Replacement of FVIII to treat (episodic) or prevent (prophylaxis) bleeding is the main therapeutic recommendation for people with HA (PwHA) and a hemorrhagic phenotype. However, up to 30% of these PwHA may develop anti-FVIII alloantibodies (inhibitors), neutralizing the clotting activity of FVIII and requiring bypassing agents. Emicizumab is a monoclonal antibody recently approved for prophylaxis of both PwHA without or with inhibitors.

Aims: We are performing a systematic review to evaluate efficacy, effectiveness, and safety of emicizumab as prophylactic therapy of PwHA.

Methods: The protocol for this systematic review was published on PROSPERO (CRD42021282088) and conducted following PRISMA 2020 statement. Electronic bibliographic databases were used to search publications, followed by validation. No language nor publication period were used as restrictions. We included randomized controlled trials and observational studies (prospective cohort, retrospective cohort, and cross-sectional) comparing emicizumab with FVIII or bypassing agents as prophylaxis. Two independent reviewers will select the articles and extract the data using a standardized form. Risk of bias assessments and grading the quality of evidence will be performed for the evaluation (image 1).

Results: Our search identified 1,436 publications from the databases. After removal of 411 duplicates and 924 articles by title/abstract screening, 101 publications will be evaluated as full-text for selection (image 2). We hope this systematic review will be complete by the ISTH congress.

Conclusion(s): It is expected that this systematic review will help to understand the aspects of efficacy, effectiveness, and safety regarding the treatment of PwHA with emicizumab.