Efficacy/effectiveness and safety of emicizumab prophylaxis of people with hemophilia A: a systematic review and meta-analysis

ABSTRACT

Background

Emicizumab is a monoclonal antibody approved for prophylaxis against bleeds for people with hemophilia A (PwHA). A systematic review was conducted evaluating the efficacy/effectiveness and the safety of emicizumab as prophylaxis for PwHA compared to prophylaxis with factor VIII (FVIII) or bypassing agents (BPA), respectively in patients without and with inhibitors.

Research design and methods

Database-directed search strategies were performed in Aug/26/2022 and updated in Mar/16/2023. Studies evaluating the prophylaxis with emicizumab versus prophylaxis with FVIII or BPA in PwHA without or with inhibitors, respectively, were selected by two independent reviewers. Data were extracted by two independent reviewers. Annualized bleeding rates for total treated bleeding events (ABR-all) were evaluated by meta-analysis. The quality of studies and certainty of evidence were assessed.

Results

A total of 11 studies were included. The standard mean differences for ABR-all were −0.6 (95%CI −1.0 to −0.2, p-value = 0.0002), among PwHA without inhibitors, and −1.7 (95%CI −2.4 to −0.9, p-value <0.00001), among PwHA with inhibitors. However, there was moderate heterogeneity in both meta-analyses. The most frequent adverse event was injection site reaction.

Conclusions

Emicizumab prophylaxis was superior in reducing the ABR-all when compared with prophylaxis with FVIII or BPA.

KEYWORDS:

• Bleeding rate
• bypassing agents
• emicizumab
• factor VIII
• hemophilia A
• prophylaxis

Declaration of interest

RM Camelo received speaker fees from Bayer, Novo Nordisk, Hoffman-La Roche, and Takeda; consultancy fees from Hoffman-La Roche and Takeda, and scientific event grants from Bayer, Novo Nordisk, Hoffman-La Roche, and Takeda. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Author contributions

RL Muniz Jr and RM Camelo contributed to the study conception and design, collection and assembly of data, analysis and interpretation of the data, drafting of the article, critical revision of the article, and final approval of the article. MM Barbosa and MS Araujo contributed to the study conception and design, collection and assembly of data, analysis and interpretation of the data, critical revision of the article, and final approval of the article. AA Guerra Jr, FA Acurcio, J Alvares-Teodoro contributed to the study conception and design, critical revision of the article, and final approval of the article.

Acknowledgments

Partial results of this study were published at the following scientific meetings: ISTH congress, 2023; ISPOR, 2023; EAHAD, 2023; HEMO, 2022; and ISTH congress, 2022.

Supplemental data

Supplemental data for this article can be accessed online at https://doi.org/10.1080/17474086.2023.2293096.

Additional information

Funding

This paper was not funded.