Can gene therapy reduce the treatments for hemophilia A?

Question

Hello doctor,

I have hemophilia A and have been getting factor VIII infusions since I was a child. I heard about gene therapy and want to know if it can help me need fewer treatments. Is it already approved or still being tested in trials? Also, are there risks like immune reactions or liver problems? I am just tired of always worrying about every little injury.

Kindly help.

Thank you.

Answer 1

Hello,

Welcome to icliniq.com.

I understand your concern.

Gene therapy for hemophilia A is now approved in some countries. One approved treatment is called Valoctocogene roxaparvovec (Roctavian). It is a one-time intravenous treatment that helps your body produce its own factor VIII, which could reduce or even eliminate the need for regular infusions.

However, there are a few important things to consider. Not everyone is eligible for this therapy. If you have certain liver problems, antibodies to the virus used in the treatment, or specific past treatments, it might not be suitable for you. Some people may also have an immune reaction, hemoglobinopathies, so doctors may prescribe steroids for a short time to protect the liver and help the therapy work better. In some cases, patients have experienced elevated liver enzyme levels after treatment, so regular liver monitoring is necessary.

While gene therapy does not cure hemophilia A, it can greatly reduce the number of bleeding episodes and make daily life easier. It is a good idea to speak with your hematologist or a specialized hemophilia treatment center to find out if this option is right for you. With today’s medical advances, there is real hope for living with less fear and more freedom.

I hope you are satisfied with my answer.

For further queries, kindly follow up.

Thank you.