Haploidentical transplant

Hi everyone,

On my recent  trip to the US asked for a repeat search for a match for my son. The doctor  told me about the good success  rate (70%) of haploidentical  (half matched) transplant  now.This means all parents and most siblings can be a match. They are using  reduced intensity conditioning so side effects  like infertility  are  minimized. They are hopeful to increase success rate to 90 % by adding low dose radiation  to the  conditioning. Of the 35 patients  at this centre two have mild skin GVHD which requires  only topical treatment and one had a more severe but still  treatable GVHD. This doctor didn't  favor cord blood though he has previously  done many b/c if it doesn't  engraft you can't get more and because  it's not reduced intensity  conditioning  you've  already wiped out the patients  bone marrow. Because of  RIC in their haploidentical  procedure if the new marrow doesn't  engraft the old one grows back (so patient survives albeit with thal major) because  the  donor is an adult relative ( usually ) you  can  always  get more donated marrow if needed.
He said gene therapy so far is also using high intensity  conditioning so has same problems  except chances of GVHD are nil.
What do you think?
Zahra

Hi again,
I am surprised at the encouragement  for this haploidentical transplant.
Earlier I was told that as my son is doing well on transfusions and exjade we shouldn't  consider  a transplant  that isn't a  full match and even if full match should  consider  carefully if its not a sibling full match.

Now the same doctors are encouraging  a half match. Now they say even though he is  doing well  now compliance is a huge issue as kids hit their late teens and twenties. Also one doctor said even with exjade life span will be reduced . They will reach 50's at most.

How big of an issue is compliance?  Any older patients/ parents experience?

What changed? Very confused.

Zahra

Agree with Zahra, I am seeing more push from medical community for haplo-identical, even Open ADs from large hospitals for haplo match BMT.

Like Zahra, I am too very confused on what has changed and why is haplo identical is getting more attention.

warm regards,
CatchR

Hi everyone,
Hope everybody is doing fine. I am new to this community. My five years old grandson is a thalassemia major. He got first transfussion at his five months age. As he has no siblig we exlored the way of unrelated BMT and 10point high resolution HLAtyping was done, but no suitable donor is yet available. Dr has advised us that if suitable donor is not available then we may try BMT from mother to child. Few days ago this Dr. did not want to go for Haplo BMT and many other docttors in our locality are against Haploed BMT. They are reluctant only in case of hopeless blood cancer. Andiy, I request you to kindly guide us properly.
With regards,
MC

hi MC,

I am agreed with doctors, blood transfusion is best option rather going for BMT if not fully matched. I was also explored this in past but now convinced with blood transfusion.

I can only recommend haploidentical under Dr Sodani of Italy at this time. I know of no other doctor with his experience in this type of BMT. I would suggest you continue to look for an unrelated donor in the bone marrow registries.

Hi everyone,
Hope everybody is doing fine. I am new to this community. My five years old grandson is a thalassemia major. He got first transfussion at his five months age. As he has no siblig we exlored the way of unrelated BMT and 10point high resolution HLAtyping was done, but no suitable donor is yet available. Dr has advised us that if suitable donor is not available then we may try BMT from mother to child. Few days ago this Dr. did not want to go for Haplo BMT and many other docttors in our locality are against Haploed BMT. They are reluctant only in case of hopeless blood cancer. Andiy, I request you to kindly guide us properly.
With regards,
MC

Have you explored transplant through Cord Blood. Cord Blood Transplant can be done even with partially matched HLA type? The results of CBT are comparable with fully matched unrelated BMT.

Andy,

What are your thoughts on this procedure for lil A?  He is 17 years old now and his sister is a half match.  Alternative, my husband and I can be donors because we would be half matches. 

Would the risks be lower than they would for regular BMT?  Should we seek a consult from Dr. Sodani?

Thank you,

Sharmin

Sharmin,

Dr Sodani will not do haploidentical beyond age 15. He has had his best success with young patients. The mortality rate for patients older than 15 has not been acceptable.

Thank you Andy,

In that case I will hold out for luspatercept and gene therapy.  I think it is just a matter of time before these treatments become successful.

Sharmin

I agree that there are better options for A and not far off in the future. He may be able to get into a trial that will hasten things, as well. He is a perfect candidate.

Well said Andy and Sharmin. Hope for the best

Parin2005,

The present treatment for thalassemia is far superior to what it was a decade ago, and the future will be much brighter - with mutlitple options for a cure clearly in sight now.  Hoping for the best for everyone here.