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Decreased transfusion needs with hydroxyurea in thalassemia major or intermedia

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Andy Battaglia:
I just ran across this article of interest about trials of hydroxyurea on both thalassemia intermedia and major. While we have heard much about the use of hydroxyurea in intermedia, we don't hear much about its use in major. Apparently, many majors could benefit from hydroxyurea therapy and this study suggests it as a potential therpay for thal majors especially in countries without adequate blood supplies.

From http://www.blackwell-synergy.com/doi/abs/10.1111/j.1537-2995.2007.01399.x

Credits

--- Quote ---Mohamed Bradai, Serge Pissard, Mohand Tayeb Abad, Agnes Dechartres, Jean-Antoine Ribeil, Paul Landais, Mariane de Montalembert
Decreased transfusion needs associated with hydroxyurea therapy in Algerian patients with thalassemia major or intermedia
Transfusion (OnlineEarly Articles).
doi:10.1111/j.1537-2995.2007.01399.x
--- End quote ---


--- Quote ---Hydroxyurea treatment was initiated in 9 patients with thalassemia intermedia (TI) and 45 with thalassemia major (TM). Patients received transfusions when their hemoglobin (Hb) levels dropped below 6 g per dL. A decrease in annual transfusion requirements greater than 70 percent defined a good response, between 40 and 70 percent a partial response, and smaller than 40 percent no response.  RESULTS:The response was good in 8 (90%) patients with TI and 20 (44.5%) with TM, partial in 9 (20%) patients with TM, and absent in 1 (10%) with TI and 16 (35.5%) with TM. In TM patients, transfusion needs decreased by 56 percent over the first year of hydroxyurea treatment. By univariate analysis, a better response to hydroxyurea was associated with older age at the first transfusion (p = 0.02), higher prehydroxyurea Hb (p = 0.0004), codon 6(–A) mutation (p = 0.002), TI (p = 0.03), and history of splenectomy (p = 0.05). Xmn1–/– was associated with a worse response (p = 0.0001). By multivariate analysis, a better response was associated with the Xmn1 polymorphism (p = 0.008).

CONCLUSION:Hydroxyurea may be an alternative to transfusions for TI patients as well as for TM patients in countries that have limited blood supplies.
--- End quote ---
 

This is the second Algerian study I have seen regarding hydroxyurea. In light of the findings of the following study concerning the safety of hydroxyurea, I think the time has come to take a more serious look at using it with thal major patients.

From http://bloodjournal.hematologylibrary.org/cgi/content/abstract/103/6/2039


--- Quote ---HbF induction has been sustained for up to 8 years without adverse effects on growth or increased numbers of acquired DNA mutations. Long-term hydroxyurea therapy at MTD is well tolerated by pediatric patients with SCD and has sustained hematologic efficacy with apparent long-term safety.
--- End quote ---

This study was done using sickle cell patients, but there is no reason to believe that there would be anymore expectation of adverse affects on thal patients than there is in sickle cell patients.

Canadian_Family:
Thanks for the article Andy, We are a strong believer in using body's own capability to produce Hbf or Hba. The current regimen to transfuse blood is an artificial way of keeping the Hemoglobin levels high and is causing complications as we see it. The use of drugs like Hydroxuurea or gene therapy is the only viable solution for the treatment of thalassemia.

Hydroxyurea is a step forward in this respect and must be explored further, however, we think gene therapy is the ultimate cure for thalassemia.

priya:
Dear Mr. Andy

I have heard that hydroxyurea and Blood Transfusion both can’t be given together because they are contradictory to one another.

With Great Regards

Dimple

Andy Battaglia:
Hi Dimple,

That is true, because for hydroxyurea to be effective, there must be bone marrow activity, and this activity is greatly diminished by transfusions. In fact, this has been a barrier to testing hydroxy on majors, as very few majors are willing to stop transfusing, which would be required by the trial. As better fetal Hb inducers are tested, I believe more majors will be willing to stop transfusing so that they can take part in trials. A trial of a drug that has the potential to raise Hb by 4 points would be much more attractive than one that raises Hb only 1 to 2 points.

Is Priya still using caroa syrup? Have you noticed any improvements in Hb or overall health and energy?

Manal:
Hi Dimple

Actually they can be given togather as in the case of a 4 years girl whom i know that developed extramedullary hematopoesis ( spelling..) in the lungs although she get regular transfusion every month. So the hema put her on hydra togather with transfusion but every 21 days instead of 30

By the way this is a rare case

Manal

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