Comprehensive Insights into Hemophilia A: Prevalence, Clinical Impact, and Economic Considerations

EP: 1.Management Insights for Patients With Hemophilia

EP: 2.Coverage Decisions for Expensive Hemophilia Treatments

EP: 3.Impact of ICER Reports on Hemophilia Treatment Decisions

EP: 4.Enhancing Provider Access and Expertise for Rare Disease Care

EP: 5.Factors Impacting Hemophilia A Care Costs

EP: 6.Collaborative Strategies in Hemophilia A Care Cost Management

EP: 7.Strategies to Manage Medication Costs and Waste

EP: 8.Adapting to Novel Hemophilia A Treatments

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EP: 9.Comprehensive Insights into Hemophilia A: Prevalence, Clinical Impact, and Economic Considerations

EP: 10.Evolution of the Hemophilia A Treatment Landscape: Innovations and Guidelines

EP: 11.Hemophilia A Treatment Factors and Outcomes

EP: 12.Prophylactic Treatment Challenges and Considerations for Hemophilia A

EP: 13.Consequences of Nonadherence in Hemophilia A

EP: 14.Advancements in Hemophilia A Therapies

EP: 15.Comparing Hemophilia A Therapies: Efficacy and Safety Insights

EP: 16.Real-World Insights: Factor vs Emicizumab in Hemophilia A

EP: 17.Emicizumab Dosing: Prophylaxis and Bleeding Management

EP: 18.Optimizing Hemophilia Treatment: Strategies and Adherence

EP: 19.Advancements in Hemophilia A Therapies: Altuviiio and Roctavian

EP: 20.Advancements in Hemophilia A Treatment: Fitusiran and the Evolving Landscape

This is a video synopsis/summary of an Insights involving Allison Wheeler, MD.

In the United States, hemophilia A occurs in about 1 in 5000 live male births, with about 400 babies born with it each year. Estimates suggest there are about 33,000 male patients living with the disorder, though some mild cases in males and females may be undiagnosed.

The clinical impact varies based on disease severity, categorized by clotting factor VIII levels: less than 1% is severe, 1% to 5% is moderate, and 6% to 50% is mild. Those with severe disease have spontaneous bleeding and bleeding with minor trauma, significantly impacting quality of life. Moderate disease (1%-5% factor VIII) has variable impacts. Those with mild disease (6%-50%) mostly bleed with major trauma or surgery.

Economic costs also vary by severity. Adults with severe disease are recommended prophylactic therapy to prevent bleeds, costing $500,000 to $750,000 yearly. Additional factor VIII is needed to manage breakthrough bleeds. Hospitalizations for bleeding or surgery mean lost work/school days. Patients who can self-infuse factor VIII at home may avoid hospitalization for minor bleeds. Those with moderate/mild disease self-infuse less often, so they need hospitalization more often when bleeding occurs. In summary, hemophilia A has variable but often greatly impactful clinical and economic consequences.

Video synopsis is AI-generated and reviewed by AJMC^® editorial staff.