Safety and efficacy of marstacimab in patients with hemophilia A and B: a systematic review and meta-analysis
Expert Review of Hematology, cilt.18, sa.8, ss.649-660, 2025 (SCI-Expanded, Scopus)
- Yayın Türü: Makale / Derleme
- Cilt numarası: 18 Sayı: 8
- Basım Tarihi: 2025
- Doi Numarası: 10.1080/17474086.2025.2522296
- Dergi Adı: Expert Review of Hematology
- Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Chemical Abstracts Core, EMBASE, MEDLINE
- Sayfa Sayıları: ss.649-660
- Anahtar Kelimeler: Congenital bleeding disorders, monoclonal antibody, TFPI inhibitor, annualized bleeding rate, hemostasis
- Sağlık Bilimleri Üniversitesi Adresli: Evet
Özet
Background: Hemophilia A and B are life‐threatening congenital bleeding disorders traditionally managed with frequent factor replacement therapies, often complicated by breakthrough bleeds and inhibitor development. Marstacimab, a monoclonal antibody targeting TFPI, has emerged as a novel prophylactic treatment to reduce bleeding episodes in patients without inhibitors. Methods: A systematic review was conducted following PRISMA guidelines. A comprehensive literature search was performed across multiple databases. Meta-analysis was conducted using R, applying a random-effects model. Results: Nine manuscripts were included. Marstacimab significantly reduced the annualized bleeding rate (mean difference: −16.30; 95% CI: [−18.46, −14.15], p < 0.001) and showed a favorable safety profile with most adverse events being mild or moderate, and no thrombotic events reported. The use of a prefilled pen device further highlighted the therapeutic benefits and ease-of-use of Marstacimab. Conclusions: This meta-analysis reinforces the efficacy and safety of Marstacimab in reducing bleeding rates in severe hemophilia A and B. The findings support its role as a promising, transformative alternative to conventional factor replacement therapies. Registration: 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.