Real-World Insights and Monitoring of Non-Factor Therapies in Hemophilia

Real-world experience with non-factor rebalancing agents, including emicizumab and fitusiran, provides valuable guidance for optimizing hemophilia management. Clinicians may consider switching therapies in patients who experience breakthrough bleeding, develop inhibitors, or have lifestyle or adherence challenges that affect prophylactic efficacy. Post-marketing studies are anticipated to further clarify long-term safety, real-world effectiveness, and optimal sequencing of these agents. Evaluating patient response involves monitoring both clinical outcomes and quality-of-life measures. Key indicators of successful therapy include minimal or no spontaneous bleeding, preserved joint health, ease of administration, and improved daily functioning. Regular follow-up and individualized assessment allow providers to adjust treatment plans proactively, ensuring continued protection against bleeds while minimizing complications. These real-world insights complement clinical trial data, supporting informed decision-making and more personalized care for people with hemophilia.