Can newer therapies help manage hemophilia A effectively?

Question

Hello doctor,

I am a 27-year-old male with severe hemophilia A, which I have managed well with prophylactic factor VIII infusions. Recently, I have been having more frequent breakthrough bleeds, especially in my right knee. I am worried about potential joint damage. Should we consider adjusting my treatment plan? I am interested in exploring newer, longer-acting factor products or other innovative therapies that could enhance my quality of life. What are your thoughts on this?

Please suggest.

Answer 1

Hello,

Welcome to icliniq.com.

I went through your query and can understand your concern.

Long-term treatment with factor replacement can sometimes lead to the development of inhibitors, which may result in bleeding episodes. You should get screened for inhibitors, and if they are present, treatment options include using high-dose factor therapy for a period or steroids.

Additionally, seeing a physiotherapist for your knee and engaging in regular exercises to improve joint mobility is important. Newer treatment modalities exist, such as gene therapy, which is still experimental and available in some countries. This therapy aims to repair genes and potentially cure hemophilia.

Please get an inhibitor screening done and share the results with me. I look forward to your follow-up.

Take care.

Thank you.