Safety and efficacy of marstacimab in patients with hemophilia A and B: a systematic review and meta-analysis

Hemophilia A and B are life‐threatening congenital bleeding disorders traditionally managed with frequent factor replacement therapies, which are often complicated by breakthrough bleeds and inhibitor development. Marstacimab, a monoclonal antibody that targets TFPI, has emerged as a novel prophylactic treatment, aiming to reduce bleeding episodes in patients without inhibitors. A systematic review was conducted following PRISMA guidelines. A comprehensive literature search was carried out in multiple electronic databases. A meta-analysis of the extracted data was performed using R language and applied a random-effects model for our analysis. Nine manuscripts were included. The meta-analysis demonstrated that Marstacimab significantly reduced the annualized bleeding rate (mean difference: –16.30; 95% CI: [–18.46, –14.15], <i>p</i> &lt; 0.001) and showed a favorable safety profile with most adverse events being mild or moderate, and no thrombotic events reported. Additionally, the use of a prefilled pen injection device further highlighted the therapeutic benefits and ease-of-use of Marstacimab. This meta-analysis reinforces the efficacy and safety of Marstacimab in reducing bleeding rates in patients with severe hemophilia A and B. The findings suggest that Marstacimab provides a promising alternative to conventional factor replacement therapies, supporting its potential as a transformative option in hemophilia management. The study protocol for this systematic review was registered in the International Prospective Registry of Systematic Reviews (PROSPERO) database (www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number CRD42024620215.