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Use of Hydroxyurea in Thal Major

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Andy Battaglia:
I've often heard that many thalassemia intermedias use hydroxyurea to boost hemoglobin levels, but does anyone know of it being used in majors? I just ran across this article from 2003 and majors were treated with hydroxyurea and some were able to completely discontinue transfusions after being treated.

Has anyone heard more about this being tried in majors? Is there any reason it isn't being done?


http://www.scienceblog.com/community/older/2003/C/20031073.html


--- Quote ---In the study, seven transfusion-dependent beta-thalassemic children in Algeria were treated daily with hydroxyurea at the maximum levels...The total fetal hemoglobin level increased in all patients, as early as the first month of treatment. Mean hemoglobin levels increased from 6.5 to 10.5 g/dl in two children with thalassemia intermedia, and from 4.5 ±0.9 to 7.0 ±0.8 g/dl in the five children with thalassemia major. The increase enabled five patients to stop undergoing transfusions.
--- End quote ---

§ãJ¡Ð ساجد:
I'd love to try this one out. Where can I get this stuff and in form is it available?

Poirot:
Andy,

If I remember correctly some of the stuff that I have read on this topic, hydroxy urea only works on a small proportion of people by stimulating an increase in Hb F. But, it has to be administered as a drip intravenously, so you are just substituting one drip for another, and it has its own side effects.

Let me see if I can dig up more data on this.

Cheers

Poirot

Bharat:
Andy

My wife was on Hydroxy Urea in India and in the United States. She had good results with it. Because it is a also a chemotherapy drug, the side effects were her nails started to turn bluish. But it helped with the hemoglobin and also helped to reduce her  platelets. My wife is Thal Intermedia.

Bharat

Hallu:
Poirot,
Based on the limited knowledge that I have, Hydroxyurea treatment is administered by taking few tablets orally. One possible side effect of the treatment is reduction in total WBC count. 
It's kind of slow chemotherapy, but the amount of dose is very small so it doesn't hurt that much(in terms of side effects) if it suits you. Also once started, patient has to be on it lifelong.

It does not work for all patients. Not sure why exactly.

--Hallu

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