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Hydroxyurea Therapy in 49 Patients with Major Beta-Thalassemia

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baal:
hi

i have posatet my mutation in an earlyer post

look here:

 first tx for ur reply manal.
now i give u the results of my analysis.
compound heterozygotie ß°ß+(OMIM:141900;ICD-10:D56.1)
BUT......!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!
not only this one......
there is an other mutation too in my genes
heterozygote α+-Thalassämie(-α+/αα)  (OMIM:141800;ICD-10.D56.0)


so i have a triple mutation in my genes witch is pretty rare.....
the positiv point of view is....that the 3rd mutation seemed to be
helpfully and made the thal milder.......
andy could u tell us somethink about it?             panos

nice friend:
okiey
that mean's there is no chance that i can try hydrea , bcoze i dont know which mutation i carry's ... anywayz, thank u al , for helping me to decide . 

lots of Regards
Umair

Andy Battaglia:
Panos,

This is very difficult to answer. Studies have shown that hydroxyurea increases hemoglobin levels in thalassemics with many different mutations. However, we know that it has not been effective in all patients who have used it. Some of this is related to less than 100% compliance by the patients, so it it difficult to say whether it was lack of compliance or if it just didn't work well for them.

Using hydroxyurea in thal majors to reduce the frequency of transfusions is a relatively new idea, as opposed to the concept of completely eliminating transfusions. It is believed that this increases fetal hemoglobin and also reduces ineffective erythropoiesis (production of young red cells), which also affects the severity of thalassemia. Fewer ineffective red cells does make for a healthier individual.

Hydroxyurea also has the effect of shrinking masses caused by extramedullary haemotopoiesis, where red cells grow outside the bone marrow. This often creates serious problems as these masses can press against organs, causing pain and reducing function of the organs. Panos, because of your history, I am wondering if hydroxyurea has ever been suggested as a way of shrinking the extramedullary growths. Hydroxyurea may have a positive effect for you in more than one way. I would suggest you talk to your doctor about this and see if it seems practical for you.

opkaram:
Umair,

I agree to Andy's suggestion to talk to your doctor to try Hydrauxyurea. My daughter is taking it for last 3yrs and it is helping her to reduce the frequency. Earlier she used to get transfused every 30 days but now it has increased to 45 days since last 3 yrs.

My daughter is taking 20mg/kg. I have not seen any side effect. If anybody needs any more info, I will be happy to share.

Regards,
Om

Andy Battaglia:
http://journals.lww.com/jpho-online/Abstract/2007/11000/Efficacy_of_Hydroxyurea__HU__in_Reduction_of_Pack.5.aspx


--- Quote ---

Background: Packed red blood cell (PRC) transfusion with iron chelation is the mainstay of treatment for patients with beta-thalassemia major. Hemoglobin F augmentation is another approach to treat this hemoglobinopathy. This study evaluates the efficacy and safety of hydroxyurea (HU) in minimizing PRC transfusions in patients with beta-thalassemia major.

Method: Twenty-three patients with beta-thalassemia major received HU at a mean dose of 16 mg/kg/d. The results were analyzed at the end of 24 months. Transfusion requirement during the 6 months preceding the study was considered as the control.

Result: Twenty patients were evaluable after 24 months. The mean volume of PRC transfused was reduced from 2126.45 mL to 1489.59 mL (P<0.001). The interval between transfusions was increased by 68.7%. Grade I myelosuppression was observed in 4 patients and diarrhea in 2 patients.

Conclusions: HU was found to be safe in patients with beta-thalassemia major, and resulted in reduction in the transfusion requirements and in increase of the intervals between transfusions.


--- End quote ---

Since the focus of study shifted from replacing transfusions entirely, which is unlikely to happen with hydroxyurea in thal majors, to reducing the amount of blood being taken, we are seeing very encouraging results. The problem with the earlier research was that majors were not allowed to transfuse during the hydroxy therapy. When hydroxy did not raise Hb enough to avoid transfusions, it was thought that hydroxyurea had no place in treatment of thal major, but once hydroxy was given to patients who did not stop transfusing, the value of hydroxyurea became evident. The frequency of transfusion and the amount of blood needed both dropped. As I mentioned, the reduction in ineffective erythropoiesis also plays a role. Fewer transfusions mean less iron and less of all the bad effects of iron. I really think many majors can benefit from hydroxyurea therapy.

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