Dear Katias Mummy
Yes, hydroxyurea can decrease the size of the spleen and actually, this is one of its benefits due to the increase of fetal HB. When the spleen is enlarged, a lot of ''energy'' is consumed by the body, but after using hydroxy this energy is directed to growth.
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 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?
![Huh? ???](http://www.thalassemiapatientsandfriends.com/Smileys/classic/huh.gif)
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.
As for L-carnitine, it should be taken by both thal major and intermedia for its great benefits. Find below the studies concerning the usage of L-carnitine in thalassemia. There are about 5 papers done on the usage of L- carnitine and thalassemia. They are all 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=223829Assessment of Puberty in Relation to L-carnitine and Hormonal Replacement Therapy in {beta}-thalassemic Patients.
http://lib.bioinfo.pl/auth:El%20Beshlawy,AApoptosis in Thalassemia major Reduced by a Butyrate Derivative
http://content.karger.com/ProdukteDB/produkte.asp?Doi=87890Diastolic Dysfunction and Pulmonary Hypertension in Sickle Cell Anemia: Is There a Role for L-Carnitine Treatment?
http://content.karger.com/ProdukteDB/produkte.asp?Doi=89472Pulmonary 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=249So this means it is safe
Hope this helps but please remember it must be taken only 5 days per week only. And the dose is 50mg per kilo
Hope this helps, feel free to ask what ever is not clear
![:wink](http://www.thalassemiapatientsandfriends.com/Smileys/classic/Winkk.gif)
manal