Treatment Thalassemia Major

Hi
My son is suffering from Thal Major. He is 2 yrs old and is on regular blood transfusion. Can anyone help me to know what are the possible ways of his permanent cure. I have heard about the bonemarrow transplantation. But here we have dependency that BM marrow should match 100% and the possibility of getting BM matched is in siblings only and thats also 25%.
This is our 1st baby so in case siblings happens it will take time and so many other factors also there that it would be matched or not.
Is there any stem cell or gene therapy available in this case?

Regards
Sumit

Hi Sumit,

Welcome on the forum,gene therapy is in trials right now and not available as a cure,stem cell transplant will also need a donor just like BMT.

How much do you know about BMT? To tell you the truth BMT itself is a very risky process,there are a couple of our members who opted for BMT and they can tell you what they went through and how hard it was,our member Khalifa's son's BMT failed first time.

Now a days thalassemia is a very manageable disorder,with proper knowledge of how to deal with things,with good supplementation,on time transfusions and regular chelation one can live a fairly normal life,we have members who are in their 40's and living a healthy life,one of them just had her second baby .

I know picture painted by the doctors is very grim,i have a thal daughter she is on regular transfusions,i remember when she was diagnosed i was obsessed with a cure,i just felt like my daughter can not live with thal,she needs a cure,and ofcourse cure was BMT,we had a son after her but he was not genetically matched.Then i calmed down and then i found this site,which changed my whole perspective towards thal,i have learn a lot from here and you'll find that true for your self if you keep reading the posts,i consider my daughter a very healthy child Alhumdulillah,you can see her in the gallery and tell me what do you think .

Still if you want to go for BMT,i'd advise to opt for IVF and PGD and have a thal free and genetically matched baby and then go for a transplant rather then opting for an unmatched BMT.

Good luck .

Zaini.

Hi Sumit ,
  ,

Best Regards
Umair

Hi
   if you have any question pls send for me an e-mail i will answer what do you want
                                  khalifa
                            state of kuwait