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Decreased transfusion needs with hydroxyurea in thalassemia major or intermedia

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Andy Battaglia:
When hydroxyurea is used in cases of extramedullary hematopoiesis, it is used as an alternative to traditional chemotherapy to stop the extramedullary growth. This is mainly used in cases of thal intermedia, where between the hydroxy and transfusions, the Hb can be brought up to a level that hopefully eliminates the growth of red blood cells outside the bone marrow. In these cases, the use of hydroxy enables the transfusions frequency to be cut.

From http://www.springerlink.com/content/d52yvagplldlq12v/


--- Quote ---Hydroxyurea, stimulating fetal hemoglobin synthesis, may represent an alternative therapeutic approach...Hypertransfusion therapy and splenectomy were followed by regular transfusion (baseline hemoglobin 10 g/dl) and chelation with desferrioxamine. With this treatment, clinical symptoms disappeared, paravertebral hematopoietic masses did not progress, but severe hemosiderosis developed within a few years. Hydroxyurea therapy was initiated to increase the efficacy of erythropoiesis, thereby reducing the required transfusion volume but suppressing concomitantly further expansion of extramedullary hematopoiesis, and finally leading to a reduction of transfusional iron load. Treatment was started with 4 mg/kg per day and stepwise increased to 12.5 mg/kg per day. The fetal hemoglobin concentration increased from 4.5 to 5.5 g/dl after 1 year and to 9.9 g/dl after 2 years of treatment. The yearly transfusion volume was halved during the 1st year of treatment. At present, after 26 months of treatment, the patient has been transfusion-independent for 10 months. Serum ferritin levels decreased from 2844 to 1335 ng/ml. Size and shape of paravertebral hematopoietic pseudotumors remained stable. No side effects of hydroxyurea have been observed. In thalassemia intermedia patients with extramedullary hematopoiesis, hydroxyurea may lead to independence from regular transfusion therapy without further expansion of ectopic hematopoietic tissue.
--- End quote ---

I don't know how much this can help true majors who are completely transfusion dependent, as their Hb levels are so low without transfusions. The goal is to get enough bone marrow activity so that the body does not create these masses outside the bone marrow. In the west, most studies involving hydroxyurea have taken place with intermedias. Part of the reason is that with sufficient blood supplies, few western thals have the low Hb level that leads to the extramedullary growths. However, in countries where keeping Hb to adequate levels can be a real challenge, doctors have been more willing to try various methods that aren't commonly used in the west. I salute these doctors who are willing to be pioneers and lead us into new therapies. An Algerian study using hydroxyurea with majors, resulted in 7 of 11 patients becoming transfusion free. I feel that not enough is being done in the field of hemoglbin inducers, as this is truly one of the most promising therapies for treating thal.

I hope you can keep us updated on the 4 year old, including if there is any change in hydroxy dosage or in transfusion interval.

priya:
Thanks a lot Mr. Andy for the explanation.
We couldn't able to continued with Carao syrup for Priya cause she vomits may be due to its smell.

Thanks a lot  Manal,

With lots of Love and Regards

Dimple

cherieann:
I just want to comment on the use of Hydroxyurea.
I have been taking it for the past 4 years at a slow rate of 3 tabs a week. now I have been moved to 1 a day as my last bloods showed a fluctuating level.
I don't have much marrow activity, but my haematologist decided to give it a try.
At times I feel it is working as I have managed to extend my tranfusions from 4 weeks to 11 weeks. My Hb at that stage was 67.
I must admit I didn't feel great when I hit the 10 week mark. My poor specialist was freaked by my attempts that the longest I will go now is 8 weeks. But then you never know how things go down the line... My motto is I know my limits, but I will always try new ideas at least once.

Ekala74:
My son has 5 years old with two spleen sequestrations, 1 episode of acute chest syndrome. He is being on monthly blood transfusions since April 2008(to prevent another acute chest-better know as pneumonia). He is sickle cell beta plus thalassemia and the Dr now is recommending to give him hydroxyurea since we don't want to remove his spleen :(. I want to know if anyone has a child taking this medication and what side effects has. Do you think is recommended to take it in my son condition  :huh.

cherieann:
Hi Ekala74
Athough I'm not a child, I take it and have found no side effects.
The only thing my haematologist told me is that the dose must be right to get the effectiveness.
This may take awhile or not. It depends on the individuals.
There are times when I feel it is working and when it is not.
Sometimes the only answer to your questions is either to ask more questions or to trial it.
For me its hit and miss but I don't mind as it has no side effects for me.
I hope I have helped answer one of your questions

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