Opinion
Video
Understand key factors influencing hemophilia A treatment decisions and explore the impact of prophylactic vs on-demand therapy on efficacy, safety, and patient outcomes, including joint health, bleeding episodes, and quality of life in severe cases.
This is a video synopsis/summary of an Insights involving Allison Wheeler, MD.
Wheeler delves into the multifaceted considerations when determining the appropriate treatment for hemophilia A, emphasizing the importance of patient-specific factors. She starts with evaluating baseline factor activity and disease severity, underlining the need to adapt treatment to individual needs. Early prophylactic therapy initiation, historically around 1 year of life, has been a standard for severe disease. The evolving landscape now includes nonfactor products, allowing for more flexibility in administration, catering to patient preferences.
Addressing prophylactic versus on-demand therapy for severe hemophilia A, Wheeler unequivocally advocates for prophylaxis, referencing Marilyn Manco-Johnson’s influential work showing improved joint outcomes. The standard of care involves initiating prophylactic treatment early, with advancements like factor VIII mimetic enabling earlier intervention. Wheeler emphasizes the reduction in bleeding episodes and joint damage, and improvement in quality of life with prophylaxis. Acknowledging the impact of untreated patients experiencing daily pain and restricted joint movement, she asserts prophylactic treatment as a crucial element for enhancing patient outcomes, making it a standard of care for severe hemophilia A.
Video synopsis is AI-generated and reviewed by AJMC® editorial staff.