Thalassemia Patients and Friends
Discussion Forums => Working Towards a Cure => Topic started by: zahra on September 20, 2010, 05:06:38 PM
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Hi
I think my son will be started on hydroxy urea after his next appointment on Wednesday. Any guidance on what to expect and what to look out for would be appreciated. Manal? Priya? or anyone else with experience.
From what I understood was that transfusions would have to be stopped so that there would be bone marrow activity earlier but now that they are using it in major also is that still applicable?
Wish us luck
Zahra
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Zahra,
When you are under hydroxyurea, you should be monitering three things
1- The counts of white blood cells and hydroxurea should be stopped if they become below 3500
2- Kidney functions
3- Liver functions
These tests should be made monthly until you are sure that the body is tolerating the hydroxy then you start doing these tests every 3 months and then every 6 months
The good thing about hydroxy is that if for any reason there are side effects, they are completly reversed when the drug is stiooed
For some patients, they may feel dizzy, or had a stomach ache, i advise that the medicine is taken just before bed time
Hydroxy can be taken with or without food and even when you are on antibiotics
It is very safe taking the dose from 10mg per kg until 20mg per kg, but in some sickle cell cases it is taken until 35mg per kilo
The exact dose should be fine tuned according to each patient, i mean that there is no exact dose some react positivly at lower doses and other patints at higher ones so we have to make a trail and error
Also you should know that some patients react positivly from the first month others don't and may take more time, that is why it is agreed that you can take it up to one year until you can decide wheather it is useful or not
Hydroxy helps to reduce:
1- Size of the spleen and therefore the body is able to redirect its energy to growth
2- Decrease the retix count and therefore reduce risk of bone deformity
3- The activity of the child increase and the child ''somehow'' eats better
4- Reduces ferritin levels
Hydroxy is best taken with L-carnitine (5 days per week) and magnesium
When taken with transfusions, in some patients, it can increase gap between transfuions and in some cases can eliminate transfuions
Wish you all the luck and please keep us updated
manal
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Dear Manal,
Aoa. Thank you sooooooooooo much for writing such a comprehensive answer. I hope it works and that we see results soon. I wish to ask how much L-carnitine would be advised for a 13 kg two year old? Also is the magnesium in osteocare sufficient or is extra required? I hope he doesnt get more stomachache. We are still hoping the one caused by exjade will go away with time.
Thank you for your wishes. I will keep you informed inshallah.
Zahra
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Zahra,
Best of luck with Hydrea :goodluck .
Zaini.
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Good luck Zahra. Please keep us posted.
Sharmin
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You are welcome Zahra :biggrin
As for L-carnitine it is taken from 50mg to 100 mg per kilo. But all studiies made concerning the usage of L-carnitine with thal were on the basis of 50mg per kilo. So according to your son's weight, he will be taking 650mg per day divided into 2 or three doses ( 5 days in a week)
As for magnesium, i think what is in Osteocare is fine. In addition that most multivitamin already contain magnesium
Manal
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Dear Manal and Sharmin,
The doctor did prescribe the hydroxy urea but I havent gotten it yet. As expected he said that it isnt really possible to tell who it will help but they do have 9 patients who have become transfusion independant . Two of these have needed spleenectomies but he said not to worry about that as my son is too young at the moment. :-\
On the other hand some patients dont respond at all . Like they had been given a placebo. The dose we will start at is 20mg/kg . He said at this dose we dont see the side effects seen when it is used as a chemo. No fertility issues, no nausea, vomiting , hairloss, etc. Some patients do have a drop in white blood cell counts but usually it is controlled by lowering the dose or you could stop the medicine also to control it but he hasnt needed to do that i n any patient yet. Also kidney and liver functions willl be watched.
The target Hb is now 8.5. Lower than that and it will be back to transfusions. Initially they hope to space out transfusions. Like the next appointment is in 4 weeks but if Hb is around 10 they will not transfuse but wait another week. If its still above 9 wait another week and see how it goes. when it is below 8.5 they will transfuse.
I asked .how to know if wbc's are down. He said they will be watching it at appointments but if low there will be an infection and fever also.
Zahra
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Hi,
I asked the doctor about L-carnitine but he said they tried it and found it didnt really help unless the patient had cardiomyeopathy ( I hope I got that right) . I asked about the NO involved in the mechanism somehow being made more available by taking L-carnitine. He said that that mechanism is more in sickle cell patients where the disease is controlled by fetal Hb preventing the sickling . In thalassemia its a different mechanism. Something about younger RBC's being released by the bone marrow into the blood stream so that they have a longer life span. He said thalasemia patients already have only fetal Hb.
Can anyone explain this to me. I'm pretty confused. ??? ??? ??? ???
Zahra
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Hi Zahra,
I don't agree with your doctor aout the usage of L-carnitine, because you will find below studies showing the importance of using l-carnitine in thalassemia both on protective/treatment levels and you can print them and show them to him. In addition that there are no side effects from using it
All the studies are published in scientific journals.
Improvement of Cardiac Function in Thalassemia major Treated with L-Carnitine
http://content.karger.com/ProdukteDB/produkte.asp?Aktion=ShowAbstract&ArtikelNr=76522&Ausgabe=229926&ProduktNr=223829
Assessment of Puberty in Relation to L-carnitine and Hormonal Replacement Therapy in {beta}-thalassemic Patients.
http://lib.bioinfo.pl/auth:El%20Beshlawy,A
Apoptosis in Thalassemia major Reduced by a Butyrate Derivative
http://content.karger.com/ProdukteDB/produkte.asp?Doi=87890
Diastolic Dysfunction and Pulmonary Hypertension in Sickle Cell Anemia: Is There a Role for L-Carnitine Treatment?
http://content.karger.com/ProdukteDB/produkte.asp?Doi=89472
Pulmonary hypertension in beta-thalassemia major and the role of L-carnitine therapy.
http://www.ncbi.nlm.nih.gov/pubmed/19065439?dopt=Abstract
L-carnitine is generally a safe substance that is found in every cell in our bodies, you can even find it in baby food like in this link
..........L-Carnitine, Taurine, Ascorbic Acid, Lo Han Extract, Vitamin D, Stevia Extract, Rutin, Vitamin K, d-alpha -tocopherolsuccinate, SunActive FE ® Iron as Ferrous Pyrophosphate, Niacinamide, Vitamin B5, Boron Chelate, Magnesium Chelate, Vitamin B6, Copper Chelate, Vitamin B2, Vitamin B1, Vitamin B12 as........
http://www.babyorganic.com/cart.php?target=product&product_id=16190&category_id=249
So this means it is safe
Actually i don't get what the doctor explained to you. But in some other post, i explained the relation between fetal HB and hydrea, you will find it below, may be things would be clearer after you read it
http://www.thalassemiapatientsandfriends.com/index.php?action=profile;u=280;sa=showPosts;start=45
Concerning Hb f and its relation to thal or hydroxyurea, you will find below an explanation for this. First F HB refers to Fetal HB and it is one of the kinds of HB. When we were babies inside the wombs of our mothers, all our HB was Fetal Hb and is produced by the gamma genes and after the baby is delivered this F HB is still being produced until the baby is around 6 months old. From 6 months to 12 months, babies are supposed to start producing Adult Hemoglobin (HB A) which is produced from the combination of both alpha and beta chains. At the same time the gamma genes which produce the HB F start to switch off as Adult HB is enough and it is needed more by the body. Therefore by the age of maximum 2 years you will not detect the presence of HB F in the human body cause the body has replaced it with the adult HB. So this is the normal cycle for any person.
In thalassemia patients, the story is different because the body can not produce adult HB (because there are no beta chains to combine with the alpha chains to produce the Adult HB). At this point, the body wants to defend itself and want to help itself so the body ''remembers''that one day it had gamma genes that produced HB F, so the body starts switching on the gamma genes to produce this fetal HB to help the body to survive. That is how thal is diagnosed by the presence of HB F. Because in people that don't have thal, there is 0% of Fetal HB but in thal patients Fetal Hb is present and the test that diagnose it is called blood electrophoresis test OR HPLC. These tests detect the presence of every kind of HB in the body and its percentage in the blood.
Again in order to produce adult HB you need 4 alpha genes and two beta genes. In Beta thalassemia the two beta genes are both muted and therefore can not produce the beta chains. What differentiate thal major from thal intemedia? Both thal major and intermedia have two muted beta genes but the difference is that, in intermedia the two muted genes are not severely muted so this means they still can produce ''little good hemoglobin'' that enables their bodies to function for a while without the need for transfusions ( that is why some thal intermedia transfuse later in life because you had some ''good'' HB produced) While in major there is no Hb produced at all cause the genes are severly muted and that is why major starts transfusing when they are babies or they would die.
Role of hydroxy in intermedias:
Though it is used with cancer patients, it was found by coincidence that hydroxyurea switched on the gamma genes and this resulted in the increase of Fetal HB in patients so they started giving it in smaller amount s to thalassemia intermedia patients and found that it can help them (NOW it is also given to thal major to increase gaps beween transfusions and sometimes eliminate them) It helped them by increasing the percentage of HB f and this resulted in the increase of the total HB and therefore eliminating transfusions. Humans can live, function and grow with Fetal HB. Although Adult HB is the best choice, Fetal HB will do the job too. So this is the relation between Fetal HB and hydroxyurea.
Usually The reaction to this medicine differs from a person to the other, some react positively from the first month of trying it and others takes a while that is why it is approved to give it a try up to 9 months in order to judge whether it will help the patient or not. Some patients don't react to it at all. The usage of hydroxy is safe in thalassemia patients because it is used in very small amounts compared to cancer patients and by the way, many trails are going on these days to produce another drug which will do the same thing but not a chemotherapy.
Theoretically, in order to know whether hydroxurea will benefit you or not, a test called Xmn polymorphism is made. If the result is positive,it means you will benefit and if it is negative this means you won't, but practically doctors found that some times patients with the negative result react very good to hydroxurea and their hb rises to the extent that they eliminate transfusion and there explanation to this is that sometimes there are other unknown factors that help the body to react positively and this resulted in that many doctors try hydroxurea any way because each patient is completely a different case.
Hydroxy too helps to suppress the bone marrow and therefore eliminate pressure on the bones resulting in no deformity and pains.
The most important thing to do when you start it, is monitor the kidney and liver fuction in addition the WBCs count in order to make sure that you won't have any side effects. One of the good things about hydroxy is that if it happenes that you get any side effect by chance, it will vainish as soon as you stop it.
Also find below an important study about the role of hydroxy in eliminating transfusions in thal major patients done on 49 patients. Please show it to the doctors, hope they get convienced.
http://www.ams.ac.ir/AIM/09123/0013.pdf
Please feel free to ask anything
manal
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Great post, Manal. I agree that for hydroxyurea to give maximum results, L-carnitine and magnesium should both be taken along with the drug. Research backs this up.
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Thank you Andy :wink
manal
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Hi Manal,
This is a link to the research I found on use of L-carnitine and Magnesium with hydroxy urea. I will show it to my doctor at the next appointment.
http://www.ncbi.nlm.nih.gov/pubmed/19799627
I have got the hydroxy urea now and will start it tomorrow,inshallah L-carnitine I still need to get . Is there any brand you would recommend?
Thanks once again for your great guidance.
Zahra
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Hello Zahra
Personally i use a local brand for L-carnitine (Syrup), but i know that the GNC brand is also very good. But the GNC are capsules, so may be you can disolve them in a juice or water and give them to your son though i prefer you use syrup cause he is still young.
You can check in your pharmacy, may be you find something good. You may also check for the one i am using because i know that they export. The comany that produces the L-carnitine is called MEPACO.
I really wish you Zahra all the best and hope that it will really work for your son, but don't forget that it needs patience,keep updating
manal
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Hi Zahra
GNC also has liquid L-carnitine. Its available in GNC in two forms. One with 500mg pantothenic acid for every 1000mg of L-carnitine (white coloured bottle) and the other 15mg pantothenic acid for every 1000mg of L-carnitine (black coloured bottle). I use the black coloured bottle. You will be able to get it in Abu dhabi as my sister got it for me this time from the same place. (150 DHS).
maha
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I'm really keen on hydroxy urea, but the last I checked with Adel's hemotologist in Spore (NUH) she said it doesn't work on Beta-Thal Major. She said they found that it works well with Thal Intermediaries only. I'm going to speak to her again next month on it. Can hydroxy urea taken with XJade?
Adel started XJade 3 weeks ago, at a low dose of 125mg, he is 10.5kg (3 years old), he's tolerating ok. His liver/spleen is noticeably smaller. We are very pleased with his recent progress after given him Vit E, D, Calcium & Magnesium (started about 2 months ago). He's much stronger and his bone density has improved since. These days he is even walking confidently around without oxygen. Something we haven't done for the last 2 years!
As we have a chelation plan now, we are keeping closer watch on his HB/Liver/kidneys/Ferritin. Today HB is 9 and we transfused him (currently half way through transfusion). Last transfusion was 3 weeks. Before XJade, we were a little reluctant to transfuse him at 3 weeks. Normally wait till 3+ or 4 weeks.
After 1 year old, his spleen & liver were getting progressively bigger, we had to go for embolisation of splenic artery to reduce his spleen when he was 2 years old. He was so sick then he need transfusion every week, even 3 days then. The trend of spleen becoming big and frequent transfusion came back early this year, and we went for a 2nd splenic embolisation.
Adel's case is really tricky since he has congential heart disease + chronic lungs. I guess I had overlooked his nutritional aspects, chelation (I was under the impression that chelation starts when ferritin is over 2000 or something). We were just not informed. The doctors do not say very much and I can't rely on them to be pro-active. I'm really glad I had found a forum where I can express my thoughts and get 1st hand information. Thanks Thapal!
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Hi Syaida,
Welcome on the forum,we have a member Cherrianne who is a thal major and she uses hydroxyurea,it didn't make her transfusions stop totally but it helped her to prolong intervals between her transfusions,i believe she goes for transfusion every six weeks and she is a grown woman .
Just though you would like to know :)
Zaini.
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Zaini,
Thanks for your note. Where is Cherrianne from? My doctor just replied to me after reading some of the articles that I "linked" her on Hydroxyurea and she said it depends on the mutation. It may work on middle eastern mutation of beta-globin gene but in her experience the south east asian patients that she has doesn't respond to it. However, we are exploring it, I understand my son, adel's inherits a copy from his father which is usually by Mediteranean descent and one from me, which is from South East Asian descent. She is also worried about the long term side effects (after 10 years) which is unknown, apparently cancer causing? What do you say?
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Hi Syaida Lee
Our member is from Newzeland.
As for the usage of hydroxy according to mutation. This is correct and there is a test called Xmn polymorphism is made. If the result is positive,it means you will benefit and if it is negative this means you won't, BUT practically doctors found that some times patients with the negative result react very good to hydroxurea and their hb rises to the extent that they eliminate transfusion and their explanation to this is that sometimes there are other unknown factors that help the body to react positively and this resulted in that many doctors try hydroxurea any way because each patient is completely a different case.
As for the safty of the drug, there is a study done by an Iranian group that proved the drug safty on a long term usage on low doses which is between 10mg per kg until 20mg per kg.
And in my opinion other drugs will definetly be available for patients before those 10 years that would do the same jo as hydroxy (hopefully)
Please make sure from the doctor that Adel's issues concerning his lungs does not contradict with the usage of hydroxy
Take care
manal
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Hi everyone,
Yesterday on the 8th day of hydroxy urea 20mg/kg my son got a slight fever. This morning he was ok but now he seems feverish again. He is sleeping so I haven't seen how much exactly. The doctor had said if W.B.C. go low there will be fever and infection but not what to do if there is a fever and what kind of fever needs action. Should I wait and see ................or go to the ER?????
Advice wanted.
Zahra
PS: Others in the family have a cold and he did complain of throat pain yesterday.
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Zahra,
Most probably he is having a cold or an infection specially that others in the family complain too. Take him to the doctor and check especially if there are other symptoms like runny nose,cough,...
But i must also note that i know a family whose son's body had a reacton with hyrea that was continous high fever (almost reached 41 c and throat infection and after several ons and offs and lowering the dose, it was discovered that his body does not tolerate hydrea and it was stopped but this is very very rare, even the doctor said that he is the only case during her whole medical life)
But in the case of your son, having other members in the family ill makes more sense that this does not have to do with hydrea. Keep monitering and give him th suitable medication according to his infection and keep us informed please
manal
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Dear Manal,
thank you so much for your prompt reply. It means the world. My sons fever was gone by the time he woke up so I didnt take him to the doctor. I hope it is over but if it comes again I will take him to the doctor. The fever was very low and he has stayed active and happy throughout.
Zahra
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You are welcome Zahra and i am sure he will be fine :hugfriend
Please keep in mind that we can not avoid the common colds or infections that our children are exposed to like any other children. Hydrea can be questioned if only things don't go the way it was and the frequency of infections increased, fevers with no reason...thiings that you are not used to or not common. Also bare in mind that these days the weather is changing and all of us are prone to infections
take care
manal
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Hi everyone,
4 weeks after starting hydroxy urea my sons Hb was 9.2 but he was not transfused. Next visit will be in a weeks time. The prescribing doctor had recommended letting the Hb drop to 8.5 before transfusing to give the hydroxyurea a chance to work but the 4 weeks after doctor said they will let it go down even below 7 if he is active and has no spleen or bone issues. Which is the right way? I couldnt see any real changes in his blood reports yet.
Zahra
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Zahra
I can not get your question.... but if you mean when to transfuse him, i think that since he is on hydrea, you should not transfuse him as long as he is active and doing and eating well. You should be monitering his spleen and body activity
Did you check his hb after using hydroxy or not yet?? Also did you check his fetal Hb before starting hydrea??
manal
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Dear Manal,
I understand your point of not transfusing now that he is on hydroxy urea so that the hydroxy urea would get a chance to work but the primary doctor looking after my son here had told me that initially they wont be trying for transfusion independence but spacing out the transfusions. He has been on regular transfusions a long time now so it is scary all over again to watch him get pale and weak. I really hope to get to transfusion independance but perhaps it will take quite a while to get there. The last time his fetal Hb was checked was before his first transfusion at age 6 months which is about two years ago. Since then he has been living on donated blood. Maybe they will check again once the last tranfused blood is ending its life cycle. I dont know how long that is but I suspect its more than the 4 weeks at the last check up.Till then I doubt there would be much fetal Hb. I worry also about the idea in some of the posts here that once the bone marrow has been surpressed so long via transfusions it wont start producing RBC's again. I hope that is wrong as hydroxy urea has been shown to benefit even Thal major patients sometimes and they must have been transfused long term.
His Hb after 4 weeks on hydroxy urea was just a little less than it usually is 4 weeks post transfusion at 9.2 but that was to be expected b/c of the fever he had anyway so no information there yet either. I guess its really just pray and wait for now.
Zahra
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Dear Zahra
I totally understand your fear about the idea of not transfusing, but all what i meant is to monitor very closely in a way that will not make him reach a stage of ''weakness and palness''. The purpose for this is to know how long hydrea will keep him away from transfusion.
It is as if you were transfusing for the first time, definetly you kept monitering until you knew for example that he transfuses every 4 weeks.
If you just followed your normal schedule of transfusion, how will you know if you will benefit from hydrea in increasing the gaps between transfuions, hope you get my point :wink
As for the fetal HB, there is no fetal Hb in transfused blood so any fetal Hb measured in his body will be only his. Usually measuring fetal Hb is done before starting hydrea in order to compare before and after usage to see if hydrea has caused any increase or not
Wish you all the luck :biggrin
manal
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Hi Zahra,
As Manal mentioned, you should have a baseline HbF reading for later comparison. If the HbF rises while using hydroxyurea, it is a sign that the hydroxyurea has a better chance of working. Don't worry about the bone marrow not being able to start producing again. Every thal can tell you about bone pain when the Hb is low, a sign that the bone marrow regularly does try to start to produce red blood cells when the Hb gets low. Of course, it is mostly useless activity in majors, but it does readily occur as soon as the Hb drops.
I suggest patience, as it does take 1-2 years to adequately assess if hydroxyurea is having a real impact. Hopefully, you will see some change in the percentage of HbF present.
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Dear Manal,
Thank you for your support. You are right that it is like starting all over again. There was a sense of calm in the knowledge that his Hb will stay in the safe range for 4 weeks at least after transfusion. Less worry I mean. But of course the danger of transfusiona nd iron overload is greater that way. If hydroxy urea works it is the best way I guess b/c it will eliminate transfusion risk, reduce iron overload and reduce the clutter of unused excess alpha chains. That is why we must try.
Andy ,
Thanks for your post. It makes me wonder if the body pain my son has been complaining about is from his cold or from the resumption of bone marrow activity. He especially complains of pain in his hands. ??? As you mentioned we need a baseline fetal Hb to compare with but I was just thinking that when tranfused there is no bone marrow activity and hence no fetal Hb and that will come into the picture when transfusion is stopped and bone marrow is active again. Maybe I am wrong and need to be corrected. Is there high fetal Hb in a transfused thal patient or is it normal as long as transfused? What I mean is when do do the test to get his fetal Hb before the effect of hydroxy urea but after he starts making his own blood again so there is a baseline Hb. If we test while he transfused wont it give a false impression of increased fetal Hb due to hydroxy urea when it is actually due to going back to his own pattern of high fetal Hb due to thal. I hope I am making sense. Also is the fetal Hb at 6 months of any value in making this assessment of baseline Hb F?
Thank you both again.
Zahra
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Zahra,
The 6 month fetal Hb percentage isn't good for a baseline, but it might give a hint at his ability to produce HbF by what its percentage was at that time. It doesn't matter how much HbF is currently present, but the level now gives a reference for future measurements. What you hope to see is even if the Hb level doesn't rise significantly, but remains somewhat stable, that the percentage of this Hb that is HbF will rise. When this happens, we know that the bad red blood cells are being suppressed while the red cells with higher amounts of fetal hemoglobin are becoming more prominent. This is far more important than it sounds because it means there is less bad hemoglobin produced, so fewer defective red cells and less "debris" in the bloodstream.
One thing to remember with hydroxyurea is the seeming paradox where bone marrow is suppressed to reduce the amount of defective beta globin produced, but the drug also can basically turn back on the gene that produced the gamma globin found in HbF. I really think this needs to be emphasized, as the benefits of hydroxyurea are more than a stabilization or rise in Hb. Reducing the amount of defective globins produced is one of the main goals of treatment for thalassemia. This is somewhat accomplished by keeping the Hb high through transfusions, and it is also accomplished by use of hydroxyurea. Both reduce bone marrow activity. Use of hydroxyurea in transfusing patients has worked in trials and some patients have become transfusion independent, while others have reduced their transfusion frequency. Every patient is unique, but the majority of patients in trails have found some benefit from hydroxyurea use. I also want to add that better drugs are in trials, which will make this approach to treatment much more productive.
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Hi Andy,
Thank you. I will ask for a Hb electrophoresis but I remembered on looking for his 6 month report that this lab doesnt give fetal Hb amount. just greater than 40%. I will ask but will probably have to take him elsewhere to find the correct amount of fetal Hb . That will be sometime after his appointment tomorrow then.
Zahra
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Can anyone suggest where I can do the xmn polymorphism test? The last I checked with my son's doctor that it is not available in Singapore. I was told by her the nearest could be Thailand for me. Can anyone link me to a hospital or doctor contact so I can air flown Adel's blood overseas from Singapore/Malaysia?
I need to have a back-up plan should we need to start HU. Thanks in advance.
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hiiii all ,
Today after reading all the posts on this topic i was very disturbed thinking that some of u are so lucky that atleast ur doctors guide u in using l-carnitine/hydroxyurea/magnesium.....but here in my case m hearing all these names for the first time even after being wth thal from past 7 years
I know that the doctors will never be proactive and let u knpowbut here i had asked ma doctor about this and he told me not to give attention to all these things and he is a renowed haemat..........its been high time that i think we should change the dr who also is concerned wth the patients and not jst wth the earnings........m really dissapointed and angry at the same trime as even we are not finanacially tht good that we could meet wth all the expenses but atleast if we know that there are ways to help our child may be we can atleast try for that and if possible
can anyone pls suggest me good haemat in mumbai........................pls i really need one for my daughter m literally in tears ...some of u may think that i m so negative and rude in my remarks but the kind of approach i ve got is horrible just cant say nything else here...............so pls help me out
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Hi pleasance
I really can understand what you feel and i definetly agree that this is annoying to any parent. But unfortunatly not all doctors are fully aware of the management of thal and to some extent in chronic disease parents have to search and search for any thing that can be of a benefit. But the good thing is that it is never too late :biggrin
Definetly you will see anyof our friends who live in mumbai will guide you to where to go
Wish you all the luck
manal
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Dear Pleasance,
I can understand your pain and frustration. To be honest, most doctors, even here in Canada are not aware of these things. We also had to research these things on our own in order to give them to our son. My son was 8 or 9 when we began giving him these supplements. To be honest, thalpal is where I found out about most of them. Were it not for Andy and our great friends here I don't know where we would be now. 4 years ago when I came to this site my son's antibodies were out of control, we were not on any of these supplements and his iron levels were beginning to increase.
With Andy's advice, we took him to Oakland, had him assessed and began treating his antibodies and reducing his iron levels. Thankfully, his iron is now well below 1000 and he is on many different supplements. You have come to the right place, we will all help you here and we will make sure your daughter does well regardless of what your doctor is educated about.
Don't worry Manit, as Manal has said - it's not too late. Your little girl is precious to all of us - and we are here to support you.
Sharmin
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i thank all of u here m so lucky to be a prt of this site and if there is any one in mumbai who is on all these supplements pls lemme know tht as tht wld be of gr8 help
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Hi,
Today it is 9 weeks since starting hydroxy urea and I am feeling depressed. At yesterdays appointment the doctor suggested stopping it. My sons Hb was 7.5 yesterday at 4 weeks post transfusion and was 8.2 four weeks ago. These are pretty much the lowest values since he started transfusing. I would have said it wasnt fair b/c he had been ill in a period starting a week before and lasting at least a week after the 8.2 and thus affecting the HB both times but he had more reasons. He said twice the urea levels had been high so it seems it is affecting his kidney functions. The first time they had thought it may be b/c he had been refusing to eat and seemed dehydrated and the urea went down from 8.8 to 6.4 with IV fluids. Yesterday the urea was 8.3 again. In the end we decided to give it one more month with great care towards keeping him hydrated.
I do have questions after looking at the old reports. His Urea was 9 in July which was before they even started hydroxy urea??? Could it be the exjade that is causing this. Perhaps this is why the doctor said to stick with a lower dose of exjade for now. How concerned should I be? Am I doing the right thing going on with hydroxy urea?
Zahra
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Hi Zahra,
Do you have electrophoresis tests before and after starting hydroxyurea? This is far more important for determining whether or not any benefit is being derived from hydroxyurea than Hb alone. What is hoped is that the percentage of HbF is rising even if the hemoglobin level is staying in the same range. If hydroxyurea has little or no effect on HbF, there is much less chance of it working, but if there is a transition to more HbF, this is a good sign and shows the intended changes are happening. Hydroxyurea will somewhat suppress the bone marrow, so the amount of bad hemoglobin being produced will drop, while the HbF level will rise. If this occurs but the hemoglobin level doesn't move much, it is still a positive gain, so then physical observations may become more important in deciding whether or not to continue hydroxyurea.
I would also say,it hasn't been long enough to make a determination, as it takes one to two years to see the total effect. Also, since he was sick, this can affect everything, so patience is required.
Hydrate, hydrate, hydrate. All thals should listen to this. Chelators affect the kidneys and liver and you need to stay hydrated at all times, especially when using oral chelators.
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Hi Andy,
Thank you for replying so quickly. You are amazing. I am feeling so guilty about not getting the elctrophoresis done. When they didnt do it at his hospitak I took him to see our regular family paediatrician and asked there but they said their machine was out of order and to call back in a few days. I still cant believe I forgot . Would it still be useful if I got it done now? do you think the elevated urea is b/c of exjade? He does show symptoms of dehydration like chronic constipation. I'm working harder to get him to drink water now. If the urea is elevated b/c of hydroxy urea what would be the level of urea at which discontinuation should be considered. I have to admit I was shocked by the Hb of 7.5 b/c he was behaving normally and his appetite was improving.
Zahra
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Hi Zahra,
Yes, get an electrophoresis done. Even now, it is important to note the HbF level. As time goes by, you want to see this percentage of HbF be a significant portion of the total Hb and hopefully rise. You need some point of reference, so it is not too late.
The high urea is undoubtedly connected to being dehydrated. Encourage him to drink plenty of water daily. As you have already seen when his urea dropped to 6.4. he gets within an acceptable level once properly hydrated. With Exjade, hydration becomes even more important. Correcting this is an area where the parent makes much more difference than the doctor can. Establish good hydration habits when he is still young.
The physical description you gave of your son shows what is most important. Normal behavior and a good appetite. What we have seen with Manal's son is that his Hb has not risen, but is fairly stable. However, his HbF percentage rose over time and his growth and activity level are in normal ranges. I feel that this is more important than an elevation in total Hb. If the child's Hb stays fairly level and his health and energy are good, and he shows some ability to produce HbF, then I feel hydroxyurea should be continued.
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The physical description you gave of your son shows what is most important. Normal behavior and a good appetite. What we have seen with Manal's son is that his Hb has not risen, but is fairly stable. However, his HbF percentage rose over time and his growth and activity level are in normal ranges. I feel that this is more important than an elevation in total Hb. If the child's Hb stays fairly level and his health and energy are good, and he shows some ability to produce HbF, then I feel hydroxyurea should be continued.
I totally agree Zahra.
From my expierence, hydrea started giving some rise to my son's hb after the fifth month so it is too early to decide especially that he was sick as you mentioned.
One more thing is the tuning of the dose as it may affect the result.
When you get the result of the HPLC, you should do another one after 3 month to compare the percentage of fetal Hb
Good luck
manal
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Hi Andy and Manal,
I will get the electrophoresis done as soon as the weekend is over. I had some success getting him to drink extra water by pretended it was petrol for his tricycle so he kept coming back for refills. I hope it continues to work. I have tothink of how to convince the doctor to let us continue with hydroxy urea. Perhaps if the urea decreased by hydrating I wont need to. Wish me luck.
Zahra
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Hi Andy
My son has been on hydroxy urea for almost four months now and the doctors are saying it is time to decide if it is working or not. They think it isnt. He has had quite low Hb durng this time despite being transfused more frequently. Even after 2 or 3 weeks his Hb would be around 8. Last time they increased the amount of blood transfused from 250 to 280 ml (weight 13.9 kg). This seems to have helped as his Hb after 3 weeks was 10.2. The plan that they have outlined now is to let his Hb drop to even 7 before transfusing. They will have him come in weekly to keep tabs on where his Hb is during this time. The doctor said that if his Hb does drop below 8 it means hydroxy urea isnt really helping him and should be discontinued. He said hydroxy urea has potential side effects even if they arent visible at the moment and the child should not be exposed to those risks if it is not helping him to increase transfusion intervals. I dont know how to convince him to try for longer as I have read on the forum that it can take upto a year for hydroxy urea to take effect.
I was really hoping hydroxy urea would help and am feeling depressed that it isnt. What do I look forward to now?
On the flip side he gained in both height and weight (0.4 kg in 3 weeks) once they got the Hb up. He hadn't really been gaining weight for a long time now. Is it ok to let him be on such a low Hb that he cant grow properly?
I am so confused about what to do .
I had the Hb electrophoresis done a little late . His fetal Hb in it was 2.1 % (not very hopeful is it?) and Hb 10.7 on 29 November. Would it be useful to do a repeat test with less than 2 months gap?
How can i convince the doctor to allow us to try hydroxy urea for a longer time? Also should I try to convince him of this?????
Looking to you for help again.
Zahra
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I think another electrophoresis should be done before stopping hydroxyurea, just to double check that there is no real movement in the HbF. The percentage is so low that I am not optimistic, but you don't want it lingering in your mind about whether or not there was any sign of improvement. If the HbF remains low, I would have to agree that the hydroxyurea is having little if any effect.
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thank you Andy Your advice is invaluable as usual. I will try to get them to give me some time at tomorrows appointment while I get the Hb electrophoresis done elsewhere.
Zahra
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Hi Andy,
I have got the Hb electrophoresis report. On 29/11/2010 Hb was 10.7 HbF was 2.1%. This comes to 0.2247g/100ml of HbF(2.1% of 10.7).
On 31/01/2011 Hb was 12.7 and HbF was 3.7%. This comes to 0.4669 g/100ml of Hb F (3.7% of 12.7).
This is almost double but its still a very small amount. Do you think it warrants a request for trying hydroxyurea for longer?
This would mean restarting it as it had been discontinued on 26th Jan. which is about 3 weeks now.
The doctor at that appointment told us we should get serious about a bone marrow transplant when he turns 3. My other two children, my husband and I all are only 50% matches but he said sometimes you can get matches from extended family. Is there anyway to decide who to test? He has 14 first cousins and 7 aunts and uncles. Someone suggested to first try those with the same blood group but I am doubtful b/c they tested me and my bloodgroup is different. The doctor also mentioned a bone marrow registry in the US. Is it likely to find matches in strangers?
Tomorrow is his next appointment. I have so many questions and am so confused.
Zahra
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Hi Zahra,
In two months the Hb jumped two points while on hydroxyurea. The HbF also rose. I would say you should start again. Hydroxurea reduces ineffective erythropoiesis, which means there are fewer bad red blood cells floating around in the blood, which in turn leads to less destruction of the good blood cells, and then the good red cells live longer, causing an increase in Hb, along with the increased HbF. I would really go six months more and then evaluate. I know parents worry about the side effects of the drug, but there is a silent danger within the blood of thalassemics, and it is caused by a combination of bad red blood cells and unmatched alpha globin chains, that are in excess due to the deficient beta globin production. We should not underestimate the damage that this does to thals. Even thals with no iron load suffer from a shrinking pituitary gland that eventually disappears altogether. It is not just iron that is the danger. The same thing goes for the heart and pulmonary artery. The damage is not solely iron related. When you consider this, hydroxyurea becomes a much safer option in relative terms because of this ability to reduce the ineffective erythropoiesis. Results are also seen in reduced spleen size, lower bone marrow activity, which means less stress on the bones from expansion, and more normal growth. I have yet to see a study that did not show a positive effect on the majority of thals who used hydroxyurea. In fact, I have often found a tone of surprise in some reports, because they really did not expect it to work so well. Even with a low increase in Hb, these other things are taking place. As I have advised with Manal's son and found that Dr Marwaha concurred, we have to look at the child. If growth is in the normal range and the child is healthy and has normal activity, then we have to keep doing what we are doing. If you see noticeable changes in activity level, increased lethargy and worsened health, then transfusion may be necessary, but as long as a child is healthy, transfusion isn't an ideal choice.
Doctors are really pushing BMT these days because of increased success rates. You may want to investigate if a mother to child transplant is possible. I don't think there's a good way to screen. Only siblings would have a good chance of being a perfect match. I doubt there is any better chance within a set of relatives than there is in the general public. I would suggest contacting Eileen at CAF to see if she can give you information on the bone marrow registry.
Patient Services Manager
Eileen Scott
eileen.s@cooleysanemia.org
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Hi Andy,
thank you as always for your expert advice. I should have made it clearer that my son has been continually transfused during this time. More often than before which was 4 weeks.During use of hydroxy urea it was once at two weeks and the rest at 3 weeks. The doctors stopped the hydroxy urea b/c of no increase in transfusion intervals. Even with more frequent transfusions his HB was less until they upped the amount transfused from 250 to 280 ml. This last report of Hb 12.7 is from 5 days after transfusing 280ml of blood. So the only positive thing is the slight increase in HbF. Hb has not gone up b/c of hydroxyurea and neither have transfusion intervals increased at least not yet. Is it still your opinion that hydroxy urea should be restarted?
Thanks again.
Zahra
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Hi Zahra,
I feel that the trial has to be six months minimum for any patient on hydroxyurea. The full effect is often not see for two years or more, so it definitely is a gradual change. What I would look at now is was there any change in retic count or bilirubin levels, as positive changes in these would indicate that hydroxyurea is cutting down the amount of ineffective erythropoiesis. I don't know how much change you might see after a short time on hydroxyurea, but any lowering of either value would have to be considered as a positive development.
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Hi Andy,
i feel really lucky to get two answers in one day. They havent done any retic tests since he started transfusing. Ther are bilirubin results though. Before starting hydroxyurea on sept 23
bilirubin total 19 and bilirubin direct 2.6 Sept 21
BT 23 and BD 3.0 Oct 20
BT 27 and BD 3.0 Oct 27
BT 29 and BD 2.0 Nov 23
BT 21 and BD 4.1 Dec 8
and I dont have any later reports back from insurance yet....................
Will try to get them tomorrow and post them then.
Thank you for all your help once again.
Zahra
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Hi
i spoke to the head doctor this visit and he said the change in fetal Hb really isnt useful unless transfusion intervals increase. He also said bone marrow transplant is not recommended for thalassemia patients unless they have a 100% matched sibling.This is to limit the chances for rejection so it is of no use to look for a match in further relatives or bone marrow registry.
The only option since both of my other kids don't match is to go for PGD-HLA. That is such a loaded option. So many things to wrestle with.
I wish I could ask what to do but I know no one really would have the answer to this one.
The doctors parting words think about it and pray and see what you are comfortable with. May Allah guide us correctly.
Zahra