Prophylaxis for inherited factor X deficiency: a systematic review

BACKGROUND: Hereditary factor X (FX) deficiency (FXD) leads to bleeds. Therapy is based on treating (episodic) or preventing (prophylaxis) bleeds with procoagulants. We aimed to evaluate the efficacy/effectiveness and safety of prophylaxis in people with FXD (PwFXD) compared to on-demand treatment or prophylaxis with a different procoagulant. RESEARCH DESIGN AND METHODS: A systematic review was conducted based on database searches on 6 May 2024, and 11 August 2025. Two reviewers independently selected publications according to the research question. Additionally, prophylaxis-referenced citations from included studies and excluded reviews since 2014 were screened. Efficacy/effectiveness was evaluated using bleeding-related outcomes. Study quality and risk of bias were assessed using Joanna Briggs Institute checklists. RESULTS: Fifteen publications involving 78 PwFXD were included, of whom 47 (60%) received prophylaxis. Most studies were case reports from Europe or North America. Prophylaxis regimens included prothrombin complex concentrate and plasma-derived FX, with follow-up from 22 weeks to 15 years. Control groups received on-demand treatment. Individual annualized bleeding rates ranged from 0-2.2 during prophylaxis versus 2.3-136.8 during on-demand periods. Study quality was mostly low (31%) to intermediate (56%). CONCLUSION: Prophylaxis appears more effective than on-demand treatment; however, better-designed studies are needed to confirm this evidence. PROSPERO REGISTRATION: CRD42024535021.