Accceptable mortality risk for BMT?

Dear all,

I come from Vietnam and I have a 7 years old son with thalassemia intermedia/major. He has been on regular blood transfusion every 4-6 weeks since 3 1/2 years old. He has a 2 years old brother who is 10/10 match. We have just gone to Taiwan for a thorough examination for BMT possibility. The result showed moderate iron deposit in liver and mild inflammation of liver (He was unluckily infected by HCV and has finished 6 months of HCV treatment). Another issue is that he has been transfused blood without leukocyte filter, which would increase the GVHD risk. The doctor said that in this case success rate of BMT is 80% with 10% mortality risk.

We are very confused and don't know what to do now, proceed with BMT or waiting for less risky treatment? Could you please give us an advice?

Thank you very much and wish you all the best
Lan Anh

Dear Andy,

Please kindly help us, the doctor in Taiwan is waiting for us and we have to make decision soon.

Thank you very much
With best regards,
Lan Anh

Dear Lan Anh,

my suggestion if you want look for other opinion, better go to bangkok, find Dr. Suradej Hongeng. My daughter done the bmt with him. if u want to know our experience, can send message to my inbox.

Lan Anh,

That seems like a high failure rate and high mortality risk.  You may wish to consult another clinic or wait for a safer alternative.  Keep your child's ferritin low.

Best,

Sharmin

I don't think you can even consider it if the mortality rate is so high.

Dear Gabriellavanessa, Sharmin and Andy,

Thank you very much for your valuable advices. We are now confident to make decision not to go for BMT in Taiwan.

Dear Gabriellavanessa, we also think of going to Bangkok and would be very grateful to know your experiences there. Please kindly check your inbox and advise us.

Dear Sharmin and Andy, if Thailand doctor also confirms such high mortality risk, we will not consider BMT anymore. In that case, could you please kindly introduce any doctor who has expertise in thalassemia treatment that we can consult to find the best long term solution for my son at an economical cost?

Thanks a lot once again for your great help and looking forward to hearing from you.
With best regards,
Lan Anh

Quite a few people have gone to Thailand for BMT. If their success rate is acceptable, that may be your best option in terms of cost, also.

Dear Andy,

Perhaps we will not consider BMT anymore. Could you please tell us if you know any doctor good at thalassemia treatment in general (not BMT) so that we can take my son there to consult the doctor about the most suitable medicine, diet,...for him?

Thank you very much for your kindness

I do not know of doctors in Vietnam.

I do not understand why the doctor would say the risk of mortality is so high with the BMT. 10% is a very high rate. I would not suggest totally giving up on the idea of a BMT, until you have talked to more centers. You might want to get another opinion at the hospital in Bagkok. http://med.mahidol.ac.th/en

Dear Andy,

Thanks a lot for your advice. But besides mortality risks, we have known that there are many serious long-term effects of BMT such as: infection, GVHD, secondary cancer, organs damage, so we are very scared. We also heard that Luspatercept is on phase 3 trial and seems to be a very promising drug. Therefore, we think we should wait for it instead of doing risky BMT.
We also wish to try hydroxyurea while waiting for Luspatercept but no doctors in Vietnam prescribe it. So could you please suggest any good thalassemia center near our location so that we could go there to consult?

With many thanks and best regards,

Hmm, maybe it's me but I don't think 10% is high. Many doctors in India says so too. You have to see there's 80% chance of success, 10% stands nowhere.

@Andy how does one die in a BMT operation? Marrow being rejected? I never understood it. If it's a failure, won't it simply reject?

That's a 10% death rate. It's ridiculous. Not acceptable at all. GVHD is usually what will cause death after a BMT. I have never recommended a BMT in India.

Lan Anh, I have no information on centers that treat thal in Vietnam at all.


The only mention I find is this  https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4563497/

In Vietnam, we have a thalassemia centre at the National Institute of Hematology and Blood Transfusion and several outpatient clinics at National Hospital of Pediatrics, Children No. 1 Hospital, Blood Transfusion and Hematology Hospital Ho Chi Minh city, Central Hue Hospital. In provincial hospitals, we have transfusion service but very variance.

Dear Andy,

Thanks a lot for your help, we will contact them

Wish you all the best

Dear Gabriellavanessa, Sharmin and Andy,

Thank you very much for your valuable advices. We are now confident to make decision not to go for BMT in Taiwan.

Dear Gabriellavanessa, we also think of going to Bangkok and would be very grateful to know your experiences there. Please kindly check your inbox and advise us.

Dear Sharmin and Andy, if Thailand doctor also confirms such high mortality risk, we will not consider BMT anymore. In that case, could you please kindly introduce any doctor who has expertise in thalassemia treatment that we can consult to find the best long term solution for my son at an economical cost?

Thanks a lot once again for your great help and looking forward to hearing from you.
With best regards,
Lan Anh

Lan Anh,

Hi. I'm liveing in Bangkok and I know a lot of best Thal doctor. I can give your advice for long-term treatment. BTW, in Thaniland, we quite advance for BMT and stem-cell therapy and also now we also have stem-cell bank in order to search matched donor.
Thailand's top 3 hospitals of Hematology & Thalassemia

1. Ramathibodi Hospital (public hospital)
Most of doctors in hospital likely progressive style, they're adopt new knowledge, technique and researched therapy for their patients. Dortor Suradej Hongeng (Division of Hematology) was the member of trail Lentiglobin Gene Therapy, he was experienced in BTM & gene therapy. You can make him an appointment to consult. Rama has highest successful rate in BMT. Thay have premium department for patients who don't want to wait a long doctor appointment. I recommend Dr.Suradej Hongeng if you interesting for BMT or stem-cell. 

2. Siriraj Hospital (public hospital)
One of the biggest hospital in Thailand. They have a lot of Thal patients so they have a lot of Thalassemia knowledge base. They have a fully medical facilitates. Siriraj was the highest number of BMT in Thailand, success rate is very good. The drawback of Siriraj is a lot of crowded patients so physical examination time may short (15-20min) and you may not met the senior and experience doctor. If you need to examination with experience doctor and have premium service you can go "Siriraj Hospital Piyamaharajkarun" premium services with premium price.   

3. Chula-longkorn Hospital (public hospital)
One of the biggest hospital in Thailand. I have doctor in this hospital. In my opinion, most of doctors in Chula hospital quite conservative style. BMT and stem-cell were not so expert as two hospital above but if you need for long-term treatment this hospital is quite good due to new building (convenient as private hospital and patient not so crowned) and fully of medical facilities. Chula hospital is advantage in blood bank because the fully co-operate with National Thailand red-cross so you won't worry for rare blood typing.
   
if you need more detail, just post and i will answer you.