The benefits of sustaining high factor VIII levels in people with hemophilia A.

Abstract

Hemophilia A is a bleeding disorder caused by a deficiency in clotting factor VIII (FVIII), leading to recurrent joint bleeds, musculoskeletal damage, and chronic pain. The World Federation of Hemophilia (WFH) recommends prophylactic FVIII replacement therapy to reduce bleeding risk, yet joint deterioration and pain persist. Maintaining high FVIII levels provides clinical benefits but requires awareness of best practices and managed care considerations. This publication examines the clinical and economic impact of hemophilia A, treatment goals, FDA-approved therapies, and managed care factors. People with hemophilia experience lower bone mineral density, increased osteoporosis risk, and significant effects on mental health, mobility, and quality of life. Treatment options-including standard and extended half-life FVIII therapies, non-factor therapies, and gene therapy-vary in sustaining FVIII levels and preventing bleeds. The high cost of care burdens patients and health systems, though prophylaxis reduces emergency visits and hospitalizations. Adherence challenges arise as patients transition to self-infusion, and insurance restrictions often limit access to comprehensive care. The WFH supports individualized, patient-centered management with pharmacokinetic-guided dosing, multidisciplinary care, and shared decision-making. Maximizing FVIII levels, rather than maintaining minimal thresholds, may improve long-term health. A holistic approach-combining early intervention, personalized prophylaxis, and strategies to address treatment barriers-is essential to better outcomes and achieving the WFH goal of zero bleeds.