Risks of BMT and CBT

My understanding is that with either BMT or CBT risks include:

Mortality 5 - 20% depending on the whether it is a sibling donor, how well matched it is, health of recipient, age of     
                         recipient.

Neurological - vision, hearing, IQ, personality

Damage to hormonal glands, infertility, delayed growth and delay in reaching milestones,

Graft versus Host disease to varying degrees - the grafted cells compete with host cells - may cause mild to severe
                            problems.

Disfiguration and changed appearance - excessive weight gain, hair growth due to steroids -  I am not sure about this.  Do patient's appearances completely change after transplant?  Are these changes permanent?

I am not sure if these risks apply to all patients and to what extent they can occur.  Andy, can you help me with this?

Sharmin

Sharmin,

Everything you said and more. It is a cure with a price and for some, the price is the most extreme. I will be regularly witnessing a real life case as I watch Chuck recover, so I should have more to say as time passes. Perhaps sometime we can get Elmas to comment on what her experience has been. I also know someone whose BMT failed as a child and whose parents were told that she had no more than a day or so left to live, who is now in her 20's and working on her masters degree. I'm not sure I can say she would have been better off if the BMT worked. She's a really sweet gal so I'm happy the doctors were wrong about her survival.

For a complete analysis of the long term side effects of BMT, please see http://emedicine.medscape.com/article/989518-overview

Anyone considering a BMT should read this carefully for a realistic view of what may come. It is not for me to judge whether or not someone makes this decision, but it is up to us to provide accurate information so that people can have a clear understanding of the possibilities. I will add that the process is becoming better understood all the time and methods to counter the problems are continually being developed.

What's CBT?

CBT=Cord Blood Transplant.

Zaini.

Thanks Andy,

I wanted to bring up these points because transplant is often listed as a cure - with a 95% success rate.  People are rarely aware to the risks and side effects.  I hope that everyone will see these risks and be prepared for them before seeking transplant.  Sometimes without realizing how possible it is to live a fulfilling life with thalassemia - parents are more concerned with "eliminating" the thalassemia - without realizing that such a radical method can eliminate so much of who their child is. 

Sharmin

...parents are more concerned with "eliminating" the thalassemia - without realizing that such a radical method can eliminate so much of who their child is. 
Sharmin

These are true words of wisdom. Yes, BMT can eliminate the need for transfusions and chelation but what else is being eliminated? Thank you Sharmin, for giving us such perspective.

The same goes for Gene Therapy but who will choose not to cure himself, when time comes?
I myself, and I am sure the majority of thals, cannot imagine having another life different than the one  we have now- i am sure the "cured" me will not be the same person, a brand new life with no thal is   something totally different-like another person from the scratch--but as I said, who will withdraw himself from that possibility? Not me, that's for sure and I have spent the biggest span of my life with thal.

Lena.   

One major difference with gene therapy is that you won't have to "convince" the body to accept the bone marrow and genetic makeup that is not native to that person. You will still be the same person genetically but with enough of a properly functioning beta globin gene to eliminate transfusions. This is not to say that there won't be side effects, but we should not see the same wide range of long term effects that we do see with BMT, because rejection will not be a problem.

I am also curious on what the long term outcomes will be in cases where cord blood is used and the result is a mixed chimerism, where both the donor and recipient's bone marrow are active but peacefully co-existing. I would suspect less long term problems will occur in this situation.

Like I said, these processes are constantly being refined, so I do expect more positive outcomes as time passes.

In the Netherlands they are not doing transplants with a not familair donor. I think they only do is if there is not any other option.

Thanks you Sharmin and Andy for bringing this perspective.

It looks like the real answer lies in the success of Gene Therapy.

It's pretty funny you talk about Elmas, Andy. Last weekend Elmas and I talked about transplants.

Sometimes I believe/feel that some people don't really understand what a transplant is. It's like they think to simple and a few months after transplant releaze (sp) what it really is.

They say that CBT is the future.

I do understand what you all have put in so far...
But not all decisions are easy to make... and sometimes one learns to weigh the risks with the benefits as laid in front of them.
In our case, we completely handed ourselves in the hands of the doctor and agreed with his decision that BMT would be a better option... Like how Dore said, it is not that easy...and it is not a matter of day or two but it is a journey that we will have to embark upon...
You know honestly speaking, I wasnt too sure about it either... When we went ahead to conceive for the second time, I had decided that if the tests revealed that the second child was not a match or was a thal major then we would not go ahead with the BMT...But it did not go that way and I took it as a signal from the Almighty and are going ahead with it... Please do not think that it is not a logically and rationally thought of decision but I had just waited for a signal in the end...and we did get it!
So once we have made the decision, we are glad to be in the hands of a good doctor who is experienced with BMT and quite confident about us and the BMT. Like advances have been made in every field so too in the care of patients with BMT...so I know that though it wont be very easy, I am hoping that it wont be too tough either!
And of course I know that all your wishes will make it easier!
Madhavi

To give a PK update. There are 3 families with the wish of transplant. 1 is testing and waiting & have a date, 1 has a cb match from younger child, no date & 1 is more likely to have one, but nothing start yet.
2 in USA, 1 in Europe. So far there has been only 1 transplant by a patient with PK. (that was in Vietnam)
Children are between 1 - 5 year.
I have told about  (possible) dangers and side effects, but they don't seems to be very scared or really notice what I wrote very well.
I have asked of they wanted to talk with other parents in the same boat, but no responds yet.
As for me: my doc told me to wait 10 years - 8 to go. I dont have sliblings and I don't believe a match will be found in the rest of the family. My parents are both carriers, so ... On the other hand, better a carrier than this.
Any help what to say, and what not may be helpfull for me.

Good afternoon from Southern Germany,

referring to Gene Therapy as a hope and cure for thal majors I hat an appointment with Ma Google:

http://www.sciencedaily.com/releases/2010/07/100713171559.htm

German specialist told me that the method that is used (special bacteria to transport the gene) has still high risks of triggering cancer.

I remember in 2009 or 2010 there was a hype about a successful gene therapie to a young immigrant man from France. But  until today no more updated information about him and his going on are availabble as far I can see.

Heartly regards

Juergen

Hi all....
Reading this issue really make me scared as i also panned to do the CBT with my both sons.
God,  it is really a hard decision to make. wheter you want to try every option to cured your child or just let it be.

Im also scared behalf of my children.