Should I consider gene therapy for hemophilia at 38?

Question

Hello doctor,

I have had haemophilia since childhood and have been receiving regular factor infusions for years now. Physically, it is exhausting, and mentally, it is starting to wear me down more as I get older. Gene therapy was recently mentioned by my haematologist, and it honestly shook me a bit. I have read that some people stop needing factor infusions, but I keep wondering how long the effects really last outside clinical trials.

Should I consider gene therapy for haemophilia at 38?
What if the factor levels drop after a few years, or my body reacts later?

My other concern is liver issues or long-term problems that no one can predict yet. It is like having to choose between a steady but exhausting thing and a hopeful but risky one. I do not want to chase a cure and then regret losing the control I have right now.

Kindly advise.

Answer 1

Hello,

Welcome to icliniq.com.

I have read your query and understand your concern.

Gene therapy for hemophilia can reduce or sometimes even stop the need for regular factor infusions in some people, which is why many patients are interested in it. However, it is not considered a guaranteed permanent cure yet, and long-term results are still being studied.

Some people maintain good factor levels for years after treatment, while others slowly lose some benefit over time and may eventually need factor treatment again. Results in hemophilia B have generally appeared more stable so far than in hemophilia A.

Because the treatment works through the liver, doctors closely monitor liver enzymes and immune reactions after therapy. Some patients need temporary steroids (corticosteroids) to protect the treatment effect. Serious problems have been uncommon, but there are still unanswered questions because these therapies are relatively new and do not yet have decades of follow-up data.

There is no need to rush this decision. Continuing regular factor therapy is still a completely valid option if predictability and control are preferred. It is reasonable to ask the hematologist detailed questions about expected benefits, monitoring, long-term plans if factor levels fall, and how much experience the treatment center has with gene therapy before making a decision.

I hope this is clear, and if you have any questions at any time, please feel free to ask.

Thank you.