Choices

After 9 months of testing my daughter has been just diagnosed with beta thalassemia. She has been receiving monthly transfusions for these past months. She is 16 months old. We are now, being told our choices are a drug called,hydroxyurea that is a oral, chemo. Or a bone marrow transfusion. Any thoughts?

Hi SamanthaDawn,

The third option would be to continue with transfusion and chelation. If you try hydroxyurea, transfusions will probably still be needed, although not as frequently, if the drug works. It does not work for all patients. There is a new drug in trials that hopefully will have a better effect at raising Hb levels than hydroxyurea.

Bone marrow transplant is a cure, but it does not work 100% of the time and there are possible dangers and side effects. The success rate continues to rise with BMTs as centers do more of them. There are some hospitals in the US with very high success rates. Where are you located?
To learn more about BMT, you can see the FAQ page of our partners at http://www.curethalassemia.org/faq/
This will answer most questions you may have about a BMT.

Hi Andy,

I didn't know much about Hydroxyurea. Could it really increase Hb levels?

Can I try to take it and see what it does?

-P.

She would get her transplant from cincinatti childrens hospital. But I was told there was no cure persay

BMT is a cure. After a successful BMT, thals no longer need transfusions. Cincinnati is highly rated for BMT.

She would get her transplant from cincinatti childrens hospital. But I was told there was no cure persay

Does your daughter have another biological sibling who you know is a perfect match for a BMT?
Nicole

The Current Standard of Care is proper transfusions (to maintain hemoglobin above 9 - 10) and proper chelation. There are regular tests (bloodwork) and annual tests (ie. Ferriscan, endocrine workups, etc). also.

If you do not have the 2012 Current Standards of Care, you can print them from the Oakland (CA) Children's Hospital or request a copy from Eileen Scott at Cooley's Anemia Foundation.
Nicole

With regards to hydroxyurea, I know this is a med. that folks with sickle cell often see results with.
I only know of one or two people who have had positive results with hydroxyurea who had thal MAJOR. By this I mean they were able to spread their transfusions out to maybe 5 weeks instead of 3. It is not a cure for thal major!
In addition, the hematologist will need to see if the child is making feta hemoglobin.
One more thing, I believe you need to be willing to allow your child's hemoglobin to fall well belong recommended ranges to see if the hydroxyurea will work. (Personally, I would not be doing this experiment on a 16 month old child who can receive regular transfusions.)  The friends of mine who did do a trial of hydroxyurea had children between 4 and 7 and needed to have their child's hemoglobin fall far below the 9-10 threshold...
Nicole

After letting her fall below a 7 and the hydroxyurea not helping 6 months later we have decided to go forward with the transplant. she has a perfect sibling match. thoughts?