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Author Topic: Decreased transfusion needs with hydroxyurea in thalassemia major or intermedia  (Read 23725 times)
Andy Battaglia
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« on: September 07, 2007, 09:55:35 PM »

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

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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

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.
 

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.

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.
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Canadian_Family
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« Reply #1 on: September 09, 2007, 10:01:43 AM »

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.

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« Reply #2 on: February 15, 2008, 06:24:17 PM »

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
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Andy Battaglia
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« Reply #3 on: February 15, 2008, 07:02:54 PM »

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?
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Manal
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« Reply #4 on: February 15, 2008, 07:20:25 PM »

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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Andy Battaglia
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« Reply #5 on: February 15, 2008, 07:44:27 PM »

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.

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.
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« Reply #6 on: February 16, 2008, 12:09:46 AM »

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
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« Reply #7 on: June 18, 2008, 03:14:54 AM »

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.
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« Reply #8 on: August 23, 2008, 10:41:59 PM »

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  .
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« Reply #9 on: August 24, 2008, 03:59:13 AM »

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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« Reply #10 on: November 13, 2008, 04:33:59 AM »

Hi All,

I am a thalassemia major, 32 years old, and I got lately 2 times back bone vertebral fracture in 15 months. The spinal MRI showed abnormal bone marrow activity and changing different signals from the bone marrow, as well as some extra cells and something like edema or tumor.

The doctor decided to put me on hydroxyurea and regular transfusion to be taken every 18 days 2 units of blood. I started hydroxyurea with 10mg/kilo/day and currently it is 20/kilo/day

Does anyone of you has any beneficial information for me in this issue as I’m confused about how critical is my case?

Thanks for everyone.
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Manal
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« Reply #11 on: November 13, 2008, 04:47:39 AM »

Hi Daffodils and welcome to the site

I think that you are in the right track ( regular short transfusion and the hydroxyurea ), but please keep us updated.


Quote
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

As for this young girl, using hydroxyurea has helped her and this mass had shrinked in size. That what i was told from her mother

good luck

manal

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LoriM
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« Reply #12 on: April 17, 2009, 08:57:22 PM »

I have a question.  Wei's (age 3) doctor started her on this and after several months I took her off.  I just was not thrilled with my 3 y/o being on a chemo drug for the rest of her life.  The doctor told me there weren't any side effects, but the drug pamplet said differently. 

Do you think I made a mistake?

Lori
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Lori
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« Reply #13 on: April 18, 2009, 03:41:11 AM »

we had been on hydra also for sometime well we had seen some imporovment in increase in hb but my son pediactric was against hydrea so we cant continue the therepy in peace and we cancel that for that time. but there we had seen many  kids on hydrea and met one of thal. major taking hydera and he is  not taking transfuison now for alteast about 1.5 year. and hb is between 8-9 . as they check it. rest it surely increaes the gap of blood trasfusion what i had seen cauz even my son  hb drop from 9.5 to 7.8 in 2 months gap not less.

rest it depends of some  ++ and + -   facotr as i think mutation of parents and my doctor said its more likely to work with ++  mutation. + -   have 30% chance to work  and  - -  dont have any.

regs
micky
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