Reply from Singapore general Hospital regarding Cord Blood

I emailed SGH about Cord Blood transplants for foreign patients like me and they replied with the message below

Thank you for you for your enquiry of hematopoietic (in particular, cord blood) stem cell transplantation for thalassemia.  We are sorry to learn of the illness you have had to deal with. I apologise for my late reply as I had been away and I have wanted to take the time to craft a proper reply to you.
Thalassemia is a disorder, which leads to the production of abnormal hemoglobin and consequent lack of production of red blood cells as well as the inability to utilise ingested iron for red blood cell production.  The excess iron leads to iron deposition in the heart, liver and endocrine organs and resultant failure of the respective organs. The medical drug treatment of beta thalassemia major has advanced dramatically through the years. This involves life-long transfusion and intensive iron chelation therapy, which are expensive, disruptive and that compliance is difficult. A review of modern drug therapy revealed that 68% of patients with beta-thalassemia are alive at the age of 35 (Cao A. Haematologica. 2004;89:1157-9). However, despite modern drugs and transfusions, many patients have succumbed to heart failure, infections, and thrombosis.  Some patients survive these complications beyond the age of 35, although the health-related issues tend to persist and pose problems in later life with heart disease and multiple hormonal failures.  Thus, even with optimal medical drug management a normal life span has not been achieved.  Also, in many developing countries, the safety of the blood supply is questionable.

The first successful BMT for thalassaemia major was performed in 1982, and now over 1500 transplants have been performed worldwide.  Thalassemia-free survival varies on the status of the liver (classified according to the degree of iron overload and resultant liver damage) of the patient going for transplantation, 91% for those with class I, 84% for those with class II and 58% for those with class III. For those with class I and II liver involvement the survival of these patients at the age of 35 is anytime better than the 68% with drug therapy. Furthermore, with improvements in transplant preparative regimens, it was found that a survival rate of >90% could be achieved for patients with class III liver involvement (Lucarelli G, Andreani M, Angelucci E. Blood Rev 2002; 16:81-85; Sodani P, et al.  Blood 2004;104:1201-3.). Thus, if a fully matched sibling or parent is available for transplantation, we would strongly recommend (the authors deemed this as ?mandatory?) this as a primary form of therapy in those thalassemic patients in class 1 and class 2 and in those of class 3 aged less than 17 years.

If a matched sibling or parent is not available, unrelated donors (bone marrow or peripheral blood stem cells or cord blood should be explored.  In a study of unrelated donor bone marrow transplantation (BMT) in 11 children with severe thalassaemia, all 11 patients are alive without thalassaemia after a median follow-up time of 397 days (range; 171-814 days).  (Bone Marrow Transplant. 2004;33:377-9).

In another publication by us we report two successful cases of unrelated hematopoietic stem cell transplants (HSCTs) in thalassemia major, one of which was performed using umbilical cord blood. The patient who received cord blood had a large liver and spleen but liver function tests were normal and liver biopsy did not show any peri-portal fibrosis. A cord blood sample fully matched in the direction of rejection but two-antigen mismatched in the direction of graft-versus-host was used. At the point of publication he was 2 years posttransplant and was well with a good hemoglobin level without receiving blood transfusions since the transplant (currently he is 4 years post-transplant and continues to do well).  A subsequent paper also details the results of twelve children who received a cord blood transplant for thalassemia.  They have all done very well.
In summary, where there is a matched sibling available, a cord blood or bone marrow transplant from that sibling is strongly encouraged.  If that is not available, an unrelated donor cord blood transplant may be considered if a well matched cord with a good cell dose is found.  This is especially so if there is difficulty in access to intensive iron chelation and a safe blood transfusion supply.

Mr Sajid Rauf, I would caution in your case, however, being already 24 years old, the risk of the transplant would be higher and success rate would be lower given the multiple previous transfusions and iron overload that you must already have.

But if you still wanted to explore a transplant then the first thing you need to do is to get something called HLA typing. This is preferably done with your self and your siblings. If these are not available then you need high resolution HLA typing at HLA-A, B and DRB1 in order to search for an unrelated donor. When you do have the HLA type, which you may be able to do locally or via other agencies closer to you, then you may send the results to me via email and I can help you search for a donor worldwide. The chances of an unrelated donor are not high for you, but there could be a chance of a suitably matched cord blood unit for you.

The transplant costs (including medication costs for the first 3 months post transplant) usually varies between US$50,000 to US$100,000. This excludes the cost of the cord blood unit which could be between US$15,000 to US$22,000 depending on the bank it is from. The cost of each HLA typing varies.

I hope the information is helpful and please feel free to write if you still have any queries.


Dr William YK Hwang
MBBS, MRCP (UK), MMed (Int Med), FAMS (Haematology)
Consultant Haematologist and Transplant Physician, Singapore General Hospital
Clinician Scientist, SingHealth Research Facilities
Medical Director, Singapore Cord Blood Bank

Thanks Sajid for following up on this one.

It seems there is a way to get unrelated transplants but as the doctor mentioned it is quite risky if we are old. I hope there is some other cure which could get the risk down.

-Narendra

Hi sajid

I hv also done this check around and tht time they had said BMT not good for thal etc . I hv lost faith in S'pore Medical system. They are very good in terms of technology but miss the human touch tht is the reason in this article he is giving the statistics abt the age of thal n stuff.

As for BMT things are stil not 100 % sure about thal, lets see.

Puja

Hi

Would you like to try other hospitals? SGH is a prominent hospital but they do not deal with thals and thier information may not be up to date. Although it may be true, I do not like the way he describes the life of a thal. Don't get me wrong. Dr William Huang is a good doctor and is well respected especially in the area of cord blood research.

I would suggest trying other hospitals which deals more with Thalassaemia patients like National Universty Hospital (NUH) or KK Women's & Children's Hospital (KKH). In NUH, you can look for Dr Tan Poh Lin. She's the BMT specialist and has given many talks about BMT for our thal society. She has encouraged me before to do testings for BMT although I don't know why I never. Her email is paev6@nus.edu.sg. In KKH, look for Dr Tan Ah Moi. Sorry but I do not have her contact. Can try searching for her at www.kkh.com.sg

Sajid, what is your decision on Cord Blood transplant now?

Hi Eponine!

I'm afraid it is out now

With my age and Iron overload, it's too risky

Dear sajid
            That true the high risk will be with old PpL and as Proffesor Lucaralli said in his lecture in kuwait on 24th nov. 2007 he said if any one have a matching donor and his age is small the chance of succesful is high .. Also he tlalk a bout the BMT from the mother even if she is not matching with her child ... and when i ask him a bout cord blood he replay
if we have the donnor why we need the cord blood BUT if there is no donnor in this case we have to look in the cord blood bank and also he don't prefare to do BMT for adult PpL ...
                 
                                       khalifa
                                state of kuwait
                          one for all and all for one

sigh... sorry to hear that. Yes that's why i kinda regretted not doing test for BMT when i was younger. But then again, I didnt know better when I was younger....
Well, God's will as you say.

Hi Kim

Happy New Year

Have you heard abt anythign happening on biomolecular therapy in S'pore(pls refer to Manals' post . She is getting it done for Ahmad).

Thanks
Puja

Hi All
The statistics given by the doctor from SGH regarding BMT and cord blood transplant might sound inhumane or less of humane touch.. but he is stating facts and the reality..
BMT is best done when young and when a child has had fewer transfusions and has all parameters like SF levels, heart, spleen etc are in good condition.
BMT is most successsful, when there is a matching sibling donor and below 5 years of age.
The technology has improved vastly with almost 100% success rate.
That is why I keep posting, that if your child is young, please refrain from alternative methods of treatment.. medicines which are still in the experimental stages.
But to keep your child healthy with proper transfuions.. and to go in for another child at the earliest, so that you can at least try and give your affected child a chance.
With CVS sampling, one can know if your second will be affected or not.
I dont think BMT will work after the child has received multiple transfusions.. because by then the child will have developed so many antibodies which will be resistent to the BMT drugs..

I am not an expert.. but this is my common sense, and from my experiences and readings on this topic.

Hi Puja

No I've not heard any biomolecular therapy at all. What is happening with that?