Thalassemia Patients and Friends

Discussion Forums => Curing Thalassemia. Bone Marrow & Cord Blood Transplant => Topic started by: zahra on September 15, 2015, 06:16:18 AM



Title: Haploidentical transplant
Post by: zahra on September 15, 2015, 06:16:18 AM
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


Title: Re: Haploidentical transplant
Post by: zahra on October 12, 2015, 02:02:08 AM
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


Title: Re: Haploidentical transplant
Post by: catchR on October 29, 2015, 08:42:17 AM
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


Title: Re: Haploidentical transplant
Post by: MC on February 22, 2016, 09:29:05 PM
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


Title: Re: Haploidentical transplant
Post by: Parin on February 22, 2016, 11:21:29 PM
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.


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on February 23, 2016, 09:58:15 PM
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.


Title: Re: Haploidentical transplant
Post by: Himanshu Kumar on February 24, 2016, 08:38:16 AM
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.


Title: Re: Haploidentical transplant
Post by: Sharmin on February 24, 2016, 01:31:57 PM
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


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on February 28, 2016, 08:52:22 PM
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.


Title: Re: Haploidentical transplant
Post by: Sharmin on February 28, 2016, 09:06:44 PM
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


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on February 28, 2016, 09:12:14 PM
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.


Title: Re: Haploidentical transplant
Post by: Parin on February 28, 2016, 11:35:30 PM
Well said Andy and Sharmin. Hope for the best


Title: Re: Haploidentical transplant
Post by: Sharmin on February 29, 2016, 01:20:25 PM
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. 



Title: Re: Haploidentical transplant
Post by: zahra on March 06, 2016, 12:02:16 AM
http://m.hopkinsmedicine.org/kimmel_cancer_center/centers/bone_marrow_transplant/haploidentical_transplantation.html


Title: Re: Haploidentical transplant
Post by: zahra on March 06, 2016, 12:33:37 AM
http://m.hopkinsmedicine.org/news/media/releases/half_matched_transplants_widen_pool_of_donors


Title: Re: Haploidentical transplant
Post by: Baby_boy on March 08, 2016, 07:34:59 PM
Hello Andy,

I signed the petition a few days ago. My son was diagnosed with beta thalassemia major since he was 8 months old. He is now 3 1/2 years old. Apart from regular transfusions and chelation, my wife and I have never stopped working towards getting him cured.  We decided to have another child with the hope that a match would result.  Prenatal testing at 12 weeks showed the baby did not have thalassemia major nor the trait. We contacted cryocell to also save the cord blood and tissue. This was successfully done. Both mother and baby girl are fine. However,  the hla test came back with bad news. They did not match. We wanted to conduct a unrelated donor search, but the cost to do that is very high. Also my ancestors are from India but my wife family on her mother's side are indigenous to the Caribbean  (Trinidad and Tobago ). Our hematologist said that it may be difficult to get a unrelated match and the process can take very long to complete. We were told that a haploidentical mother to child bmt procedure is an option and of course Dr. Sodani would be the one to oversee /do it. We want to do it but in light of the current situation at IME we are not sure if Dr. Sodani and his team are in a position to do so. Before we decided to try for our second child I did have a couple of email conversations with Dr.  Sodani....FYI.  I  decided to post this because I know that all parents out there would want a chance to cure their child and I myself would like to know what is my next step. Should I contact Dr.  Sodani like before or professor lucarli. Not sure what to do.

Kind regards,
Alicia and Shastri.


Title: Re: Haploidentical transplant
Post by: Bostonian_04 on March 09, 2016, 03:53:42 PM
There are quite a few treatment options that will be available in the near future that may offer cure or reduced transfusion with much lower risk. so in my opinion, not going for haploidentical transplant is the best approach now unless your child is not able to take regular transfusion and chelation for certain health reasons.
I would keep up with regular transfusion and chelation and be in the best possible health to avail the benefits of the new treatments that will be available soon.


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on March 09, 2016, 04:23:58 PM
For more information to help you make a choice, email info@curethalassemia.org


Title: Re: Haploidentical transplant
Post by: Baby_boy on March 09, 2016, 06:25:21 PM
Noted. Thank you for your kind words and advice.


Title: Re: Haploidentical transplant
Post by: zahra on December 25, 2016, 10:27:35 AM
Hi Everybody,
My son has started chemo for his haploidentical transplant from his brother at Johns Hopkins on the 29th, inshallah.  Keep us in your prayers.
Zahra


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on December 26, 2016, 05:44:44 PM
Zahra,

Best wishes with the transplant.


Title: Re: Haploidentical transplant
Post by: Dori on December 31, 2016, 04:24:22 AM
Best wishes with the transplant.


Title: Re: Haploidentical transplant
Post by: MC on January 03, 2017, 09:26:10 PM
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


Title: Re: Haploidentical transplant
Post by: MC on January 03, 2017, 09:28:38 PM
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.


Title: Re: Haploidentical transplant
Post by: zahra on March 08, 2017, 09:09:19 AM
Hi Everyone,
We are home from the hospital for a week now and approximately day 70 after the transplant. Should have been home alot earlier but my son got a fever that couldn't be diagnosed.  Thank God it finally went on its own.
Chimerism results are good so far showing 100%donor cells. Not final yet.
Johns Hopkins has now done 6 haplo transplants for thalassemia  ( much more for other diseases). 5 have been successful.
Keep us in your prayers.
Zahra


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on March 09, 2017, 07:15:41 PM
That's good news, Zahra. I'm happy he's back home.


Title: Re: Haploidentical transplant
Post by: BabyRiya on March 13, 2017, 09:23:21 PM
Friends
My daughter has had 6 transfusion already since last one year and she will be two in couple of weeks.
We started hydrauxia since last month and doctors are saying it could take 6 months to see the effect.

I do nt want to consider BMT  but reading scary posts of hearing loss and short lifespan liver issues, I am worried. I have older son it I have not considered a Hal match
Cause if it's a match, I may consider BMT as an option even if I am not for it.
I am so confused.  Would you guys advise doing a hla match irrespective of I consider or not?

Also where did zahara did the BMT. Is it John Hopkins in Baltimore?
Is there any easy way to contact dr Sodani?

Thank you friends.  Thank you Andy


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on March 17, 2017, 07:19:30 PM
Yes, I believe Zahra had it done at Johns Hopkins. I do not believe Dr Sodani currently has a place to practice. You can try emailing info@curethalassemia.org to see if you can contact Dr Sodani.

I would advise against rushing into a decision on BMT until you've talked to more people whose children have gone through it. I would also suggest keeping your eye on the Luspatercept trial to see if it looks like it will be a viable alternative to transfusion. A child who has only transfused 6 times by age 2 would benefit greatly from this drug, based on phase 2 trial results.


Title: Re: Haploidentical transplant
Post by: zahra on March 24, 2017, 06:08:22 PM
Hi Sharmin,
I was talking to my son's transplant doctor today and he mentioned that because of reduced intensity conditioning it is possible to do haploidentical transplants for older patients ( depending on how thalassemia has affected their health otherwise) . Your question about your son immediately popped into my head so I tried to get an outside limit. He said they handle patients upto 23-24 in the pediatric section. Adult hematology could handle older.
Regards,
Zahra Syyida


Title: Re: Haploidentical transplant
Post by: zahra on March 24, 2017, 06:15:31 PM
Hi Baby Riya,
Yes my son had his transplant at Johns Hopkins in Baltimore, Maryland.  His main doctor is Dr Kenneth Cooke.  You can email  him at Kcooke5@jhmi.edu for any further questions about the procedure. He said he would be happy to provide answers.
Regards,
Zahra


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on March 26, 2017, 05:49:13 PM
David Levy had a BMT (10/10 unrelated donor) at age 33 and reduced intensity conditioning was used. Perhaps this is opening the door for older patients. There's a lot going on that might make BMT for older patients much safer in the future.


Title: Re: Haploidentical transplant
Post by: zahra on August 04, 2017, 04:28:53 PM
Hi everyone,
We are home now with chimerism at 100% donor , no gvhd so far and no blood transfusion since end of February, alhamdulillah.
Have been advised to continue on immune supression till 1 year post BMT . Then will start vaccinations all over again.
Thank you all for your prayers. Please continue them.
So happy mashallah.
Zahra


Title: Re: Haploidentical transplant
Post by: Fahed_FN on August 08, 2017, 11:32:54 AM
So happy to hear the good news Zahra, Alhamdullilah...


Title: Re: Haploidentical transplant
Post by: Bostonian_04 on August 08, 2017, 02:54:56 PM
a really wonderful news Zahra! SO happy for you. Congratulations to your entire family.  Is this treatment done in John Hopkins in Maryland? who is the doctor/BMT specialist you worked with and did insurance pay for it? please reply if you are comfortable answering the questions.


Title: Re: Haploidentical transplant
Post by: Sharmin on August 08, 2017, 11:09:22 PM
Zahra,

Hearty congratulations to you and your family!  I am so happy to hear!

Was the haplo transplant low intensity conditioning?

Once again, so so happy for you!

Sharmin


Title: Re: Haploidentical transplant
Post by: zahra on August 09, 2017, 05:16:00 AM
Thanks everyone.
Zahra


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on August 11, 2017, 08:38:54 PM
I'm glad to know things are going well, Zahra. I've been a bit distracted by the birth of my granddaughter last week.



Title: Re: Haploidentical transplant
Post by: Fahed_FN on August 12, 2017, 02:42:57 AM
Congratulations Andy...I wish your granddaughter a long happy and healthy life ahead.


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on August 14, 2017, 07:39:30 PM
Thank you, Fahed.


Title: Re: Haploidentical transplant
Post by: zahra on September 19, 2017, 01:43:17 PM
Congratulations on the birth of your granddaughter Andy.
You're a granddad. Wow!
May she keep you distracted with her cuteness and love always.


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on September 23, 2017, 02:06:21 PM
Grandfather twice now. My daughter also has a two year old son, who I spent a ton of time playing with during our trip.

I haven't talked about this here, but during the pregnancy, my daughter was diagnosed with breast cancer. She went through 4 rounds of chemo during the pregnancy after the surgery, and next week, she will begin 4 treatments with taxol, the second chemo drug. This has been very difficult to deal with, especially very recently when a young local woman that my wife knew, died from breast cancer while we were in California visiting our daughter. This woman was 3 days older than my daughter and left three children behind. It's very sad and a stark reminder of the possibilities facing my daughter.


Title: Re: Haploidentical transplant
Post by: zahra on September 23, 2017, 02:23:59 PM
May God grant your daughter a complete recovery and give you and your family the strength to support her during this difficult time. My mother also went through this a few years ago and by the grace of God is fine now. You guys will make it through this.


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on September 23, 2017, 02:34:31 PM
Thank you, Zahra. Since my wife went through breast cancer shortly after Lisa's death in 2004, we have learned to appreciate every moment. Every year that passes is a major victory.


Title: Re: Haploidentical transplant
Post by: Baby_boy on February 16, 2018, 09:26:26 AM
Good day to each and everyone.

Happy new year 2018.

My son recently had his Haploidentical Transplant done at the Bambino Gesu Pediatric Hospital in Rome, Italy. He did the transplant in September 2017 and is transfusion free since with NO INCIDENCE OF GVHD and a stable Hb around 12. He had NO fever, vomiting or diarrhea during the transplant and even after the transplant - the doctors are so good with the level of drug administration and care over there. His appetite is amazing and he is full of energy now.
All his blood work results are within range and the doctors are extremely pleased with his recovery thus far.

I will like to take this opportunity to express my sincere gratitude for the words of advice from Andy and his esteemed team and you the caring parents/guardians who form part of this forum.
To each and every parent in this forum reading this message - DO NOT GIVE UP!!! I have seen children from all over the world cured from Thalassemia, Leukemia, Sickel cell and Diamond-Blackfan anemia. I saw them ill and now they are all cured. The oldest patient is 17 years old - wow! I even saw a sibling match and there was no difference in terms of engraftment and recovery.

The techniques used by the doctors are really God sent and everyone should take the opportunity to cure their child. I am of the opinion that it is unfair for a child to reach adulthood and still be transfusion dependent. In the past yes, but the present - NO. I will not go into much details, but, anyone who wishes to contact the hospital can do so by calling the International Assistance Team at +39 06 6859.3061/4497/3586.

God bless! and best regards.


Title: Re: Haploidentical transplant
Post by: Fahed_FN on February 17, 2018, 01:51:52 AM
Congrats Baby_boy for the good news, so happy for you.

Was it a reduced intensity chemo?


Title: Re: Haploidentical transplant
Post by: Baby_boy on February 17, 2018, 05:59:34 AM
Yes.


Title: Re: Haploidentical transplant
Post by: Fahed_FN on February 17, 2018, 09:49:30 AM
Amazing.

Are you from Italy?

For an international patient, how much would it cost and what is the best way of communication other than phone?


Title: Re: Haploidentical transplant
Post by: Baby_boy on February 17, 2018, 09:57:07 PM
I'm from the Caribbean.

Around  €200,000.00

When you call then they will advise you.


Title: Re: Haploidentical transplant
Post by: Fahed_FN on February 20, 2018, 10:00:27 PM
Is there any medical insurance that covers this?


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on February 25, 2018, 12:25:48 PM
That is great news!

Fahed, reduced intensity chemo is exactly what is sounds like. The chemotherapy regimen used before the transplant can be a reduced dose or involve different chemo agents. It causes lessened side effects to the patient.


Title: Re: Haploidentical transplant
Post by: Fahed_FN on March 01, 2018, 03:40:18 AM
Thanks Andy,

Is it advised to conduct Reduced Intensity Chemo BMT in Thailand, specifically Bumrungrad hospital?


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on March 02, 2018, 05:08:29 PM
I can't answer questions about specific hospitals. It is up to the parents to investigate the success rates of the BMT procedures at various hospitals.


Title: Re: Haploidentical transplant
Post by: zahra on November 06, 2019, 10:58:00 PM
Just an update for people thinking of haplo BMT. My son had his almost 3 years ago now with no GVHD.It was with reduced intensity conditioning and from his brother who was 50% match. He is 11.5 years old now and in the 7th grade at school. He was recently seen by an endocrinologist and all seems well
 Had started making testosterone so seems like he will go through puberty normally. Height and weight also in normal range.
May God bless all of our children with happy, healthy futures


Title: Re: Haploidentical transplant
Post by: Andy Battaglia on November 08, 2019, 03:34:32 PM
Zahra, I am so happy to know that your son is doing well.


Title: Re: Haploidentical transplant
Post by: zahra on November 09, 2019, 12:28:00 PM
Thank you Andy. Your guidance has assured that. Will forever be grateful for that


Title: Re: Haploidentical transplant
Post by: kaleem on February 13, 2020, 08:08:47 AM
Our son also received BMT from Aga Khan University Hospital Karachi, Pakistan and unfortunately it was unsuccessful. It is so pleasing to know that your kid is now free from Thalassemia. Can you kindly advise us the doctor who supervised the BMT for your kid. Was it conducted under direct supervision of Dr Sodani? Thanks and regards..


Title: Re: Haploidentical transplant
Post by: zahra on February 13, 2020, 01:29:54 PM
Dr Kenneth Cooke at Johns Hopkins in Baltimore.