Review of Published Data Affirms Emicizumab Safe, Effective in Hemophilia A

Real world published data firmly back a clinical view of emicizumab as a well-tolerated and effective treatment for congenital hemophilia A. This was the key takeaway from a scoping review recently published in Research and Practice in Thrombosis and Haemostasis.

The review authors wrote that their findings underscore that, for patients with hemophilia A, “emicizumab has an acceptable safety profile, is effective and efficacious in bleed prevention, and is associated with improvements in [quality of life].”

The investigators consulted three citation databases, as well as abstract books, for original data published on emicizumab use in congenital hemophilia A. They then evaluated these data for safety, efficacy, and quality of life (QoL) improvement associated with the agent. The data were not differentiated by patient age, disease severity, or factor VIII inhibitor status.

The review encompassed a total of 97 publications published in English between December 2014 and August 2022.

Of the total cohort comprised of the data from these publications, the median percentage of patients who had zero treated bleeds was 66.7%. Across the publications’ separate cohorts, the calculated range of mean treated annualized bleeding rates (ABRs) was low, at between 0.7 and 1.3. The calculated range of median treated ABRs was similarly low, at between zero and 1.4.

Across the publications’ separate cohorts, the proportion of patients who experienced treatment-related adverse events ranged from zero to 60.0%. Most of these events were injection site reactions. The authors noted that “[d]ata from well-established tools show QoL benefits with emicizumab.”

37 of the publications in the review had cohorts that were larger than 2,300 participants. Among the patients represented by their combined data, there were only 11 thrombotic events and four thrombotic microangiopathies reported.

Reference

Young G, Pipe SW, Kenet G, et al. Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review. Res Pract Thromb Haemost. 2024;8(4):102415. doi:10.1016/j.rpth.2024.102415