Can Hemlibra help reduce bleeds in children with hemophilia?

Question

Hello doctor,

My six-year-old daughter was diagnosed with Hemophilia A, and her doctor recommended Hemlibra (Emicizumab) for bleeding prevention. I am wondering how this medication works compared to traditional clotting factor infusions and whether it will reduce the frequency of her bleeding episodes.

Is Hemlibra safe for children of her age, and what are the most common side effects?
Additionally, will she need to continue this treatment indefinitely, or will there be a point where her bleeding risk decreases enough to stop?

Please advise.

Thank you.

Answer 1

Hello,

Welcome to icliniq.com.

I read your query and understood your concern.

Hemlibra is an emerging medication that is not widely used yet, but it has shown promising results. It works by mimicking the function of factor VIII in hemophilia A, thereby reducing the need for traditional factor concentrates.

It has demonstrated more proven results in older patients with inhibitors. However, it is important to note that Hemlibra can sometimes cause over-clotting, so monitoring symptoms and conducting regular tests is essential.

The common side effects are primarily related to the gastrointestinal system, including nausea, vomiting, and dizziness.

Currently, it seems that this treatment will need to be continued indefinitely and cannot be stopped. However, advancements in treatment may emerge in the future.

Hemlibra is considered safe for children. Make sure to regularly get her coagulation tests and factor assays done.

I hope this helps you.

Thank you.