Urgent Advise on 2nd BMT

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Urgent Advise on 2nd BMT
« on: March 09, 2017, 07:36:05 AM »
Dear All,

I am new member to this group & understand that you guys are doing brilliant job interms of awareness & helping community to fight against this deadly decease.

Let me give brief about my case.

My elder son is having Thal-Major, We have done Bone Marrow transplant in 2010 at CMC-Vellore at the age of 7 yrs. & donor was my younger son (Thal-Minor). After lots struggle, fight  & facing difficult days procedure was completed successfully & he was maintaining HB level between 9-10.

Suddenly in mid 2014 during the routine check-up we had observe that his HB is going done to 8 & after further investigation we came to know that its a case of mix chimerism with donor 30% & recipient 70%. but still he was able to maintain HB between 6.5-7 and doctors were into wait & watch situation.

Now suddenly in 2017 his HB is start falling below 5 & new chimerism was at donor 20% & recipient 80%. we have done the BT twice in this year.

Now we are totally in confusion stage what to do next & doctors have given DLI treatment in this week, where they have transfused 20 ML of Donor blood to Patient & asked us to wait for one month to see the improvement (if any)

My Sons current age is 14 yrs, we are not sure what to do next either we should go ahead with 2nd BMT & where we should do either CMC Vellore or any other hospital within India or outside.

During last three years I have meet with all the doctors across India, everyone is suggesting that we should go ahead with 2nd BMT but it would be risky & life threatening also.

One more point we have met with endocrinologist in Sep-16 & they have started the injections of Growth hormone &  testosterone hormone. Not sure sudden dip in chimerism is due to these injection or now.

Kindly advise what is best option with us on following:-

1. Shall we go ahead with 2nd transplant & take the risk or any other alternative option is available.

2. Where we should go, CMC or any other hospital within India or outside India.

3. Shall we carry on with hormone therapy.

4. Not sure how DLI is going to help us, does anyone have any good/bad experience on the same.

Regards
Sanjay

Re: Urgent Advise on 2nd BMT
« Reply #1 on: March 14, 2017, 09:49:43 AM »
Dear Andy & Senior Members of Forum,

Request you to kindly let us know your feedback on the same.

Regards
Sanjay

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Offline Andy Battaglia

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Re: Urgent Advise on 2nd BMT
« Reply #2 on: March 18, 2017, 12:05:34 AM »
My opinion is that it shouldn't be done, as the risk is too high. The one case which I am very familiar with, resulted in success in curing thalassemia, but only after the boy almost died several times and spent long periods in comas, which did result in some negative effects on the brain. And this took place at the most experienced of all bone marrow transplant for thalassemia centers on earth. Today's treatment for thalassemia is very good and patients in good treatment programs who follow their programs as they should have excellent qualities of life and are healthy people. I believe the risk is too high to justify when compared to the quality of life that can be found in a good treatment program. Keep in mind that treatment just continues to improve.
Andy

All we are saying is give thals a chance.

Re: Urgent Advise on 2nd BMT
« Reply #3 on: March 23, 2017, 10:52:47 AM »
Dear Andy,

Thanks a lot for your reply, request you to kindly further advise on good treatment programs for Thalessemia.

Please advise on doctors or center based in India or outside India, where we can do the treatment without BMT.

Regards
Sanjay

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Offline Andy Battaglia

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Re: Urgent Advise on 2nd BMT
« Reply #4 on: March 26, 2017, 10:43:05 PM »
Good treatment is available in India and it continues to improve. A patient following a good transfusion and chelation program can live a fairly normal life. This will only improve as more new drugs are developed and go through trials. Luspatercept in currently in phase 3 trials and is very promising for raising Hb and reducing transfusion requirements, while also reducing iron load. Other drugs are also in various states of development.
Gene therapy is also quite a ways towards being a reality. Comparing the risks of a second BMT to advancements in treatment, brings me down on the side of treatment. This could change as new methods of BMT are put into use, which should greatly reduce many of the risks, but that is currently experimental only.
Andy

All we are saying is give thals a chance.

Re: Urgent Advise on 2nd BMT
« Reply #5 on: March 27, 2017, 02:20:42 PM »
Dear Andy,

Thanks for your reply, its really very helpful.

On alternative medicines, one of doctors in pune have suggested us to take Thalidomide to maintain HB level, we had take the same for 6 months, surprisely HB level was increased to 9 from 6.5.

But after discussion with senior doctors at Kolkata we have discontinue Thalidomide. as they were not sure on side effect of thalidomide & no trail & research has been done for Thalessemia patients.

Once we had discontinue the same again HB level came to 6.5.

Does anyone have any feedback on Thalidomide & its side effects.

Regards
Sanjay

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Offline Andy Battaglia

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Re: Urgent Advise on 2nd BMT
« Reply #6 on: March 28, 2017, 03:29:27 AM »
As you saw, the efficacy of thalidomide for raising the hemoglobin of thalassemics is proven. Dr Ramanan has implemented research done by others in his therapy, and has had much success using thalidomide with patients. Dr Ramanan made a judgment that took into account the cost of treatment, problems with compliance and blood safety in India and decided to try what had already been proven. It would be accurate to say that his patients are the test group, even though it is not a trial, but with no one willing to run trials, mostly due to the previous stigma associated with the use of higher doses of thalidomide in pregnant women in the 1960's, which caused birth defects in children, the doctor felt he had no choice. With the doses being used in thalassemia, there have not been any alarming side effects noted. Some patients have stopped transfusing now for several years. I think when a doctor like Dr Ramanan sees so many patients dying on a regular basis, utilizing an alternative approach to treatment would have to seem attractive, and so far it is working out.

I can see this as a viable alternative in countries where costs and blood safety are problems. India still has ongoing problems with blood safety. Since there is real hope for a new drug that will raise Hb significantly, I see thalidomide as a short term answer for some patients. Hopefully, we will see Luspatercept fulfill its promise and drugs like hydroxyurea and thalidomide will probably become outdated for thalassemia.

*Pregnant women should never take thalidomide.
Andy

All we are saying is give thals a chance.

Re: Urgent Advise on 2nd BMT
« Reply #7 on: April 03, 2017, 11:11:31 AM »
Dear Andy,

Thanks a lot for your detailed reply, we are going to meet next week to doctors at CMC vellore & will keep you posted on the same.

On more doubt, there are one french company called sanofi, was also developing medicine for Thal, any update on the same.

Regards
Sanjay

 

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