115 FAQ written by Dr. Pietro Sodani, Cure Thalassemia co-founder &  Scientific coordinator

Bone Marrow Transplantation (BMT) for Thalassemia

General questions

Is there a cure for thalassemia major?
Yes,it is Bone Marrow Transplantation (BMT),the only definitive cure for Thalassemia,with no more need of blood transfusions (thalassemia free).

My child has beta thalassemia major,can he be cured?
He can be cured (no more transfusions) with Bone Marrow Transplant(BMT). The best potential donors for a BMT are brothers and sisters;we need to know if he has brothers or sisters and how old is him.

My baby doesn’t have a matching sibling. What can we do to cure him from thalassemia?
These are other options that you have:
a) you can find a donor in a bone marrow donor bank. It usually takes time (3-6 months) and money;we can help you to search for the donor
b) sometimes the mother is a compatible donor
c) even if the mother is not compatible,she can be used as donor:

For options b) and c),we suggest to do for both parents (sometimes the father can be used as donor) a simple blood test called HLA typing.

I’ve given you all the information about my son,who has thalassemia major. Do you suggest to do the BMT?
From an ethical point of view we can’t tell you what to do. As doctors,our role is to tell you the facts and the risks,but the final decision must be taken by you.

Why should I choose Cure Thalassemia to do a BMT?
There are at least 18 good reasons. Read them here.

How can I talk to a patient who did a Bone Marrow Transplantation (BMT) for a thalassemia?
You can use our BMT testimonials program.

How can I have the free advice of Dr. Pietro Sodani on Bone Marrow Transplantation (BMT)?
Dr. Pietro Sodani (our co-founder and scientific coordinator) gives his free advice via email to patients from all over the world. Read more information in this page.

What book do you suggest to read to know more about BMT?
Bone Marrow and Blood Stem Cell Transplants:A Guide for Patients and family members”,written by Susan K. Stewart,Executive Director of BMT InfoNet and a transplant survivor herself.

What are some the most useful scientific papers on Bone Marrow Transplantation (BMT)?
May 2012
Cold Spring Harb Perspect Med.:Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia –Lucarelli G,Isgrò A,Sodani P,Gaziev J.

August 2011
Blood Journal:How I treat thalassemia – Eliezer A. Rachmilewitz1 and Patricia J. Giardina

June 2011
Pediatric Reports:T cell-depleted hla-haploidentical stem cell transplantation in thalassemia young patients –Sodani P,Isgrò A,Gaziev J,Paciaroni K,Marziali M,Simone MD,Roveda A,De Angelis G,Gallucci C,Torelli F,Isacchi G,Zinno F,Landi F,Adorno G,Lanti A,Testi M,Andreani M,Lucarelli G.

November 2010
Blood Journal:HLA-matched sibling bone marrow transplantation for β-thalassemia major – Mitchell Sabloff,Mammen Chandy,Zhiwei Wang,Brent R. Logan,Ardeshir Ghavamzadeh,Chi-Kong Li,Syed Mohammad Irfan,Christopher N. Bredeson,Morton J. Cowan,Robert Peter Gale,Gregory A. Hale,John Horan,Suradej Hongeng,Mary Eapen,and Mark C. Walters

August 2010
NY Academy of science:Progress in hematopoietic stem cell transplantationas allogeneic cellular gene therapy in thalassemia – Antonella Isgrò,Javid Gaziev,Pietro Sodani,Guido Lucarelli.

November 2009
Blood Journal:Purified T-depleted,CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia – Pietro Sodani,Antonella Isgrò,Javid Gaziev,Paola Polchi,Katia Paciaroni,Marco Marziali,Maria Domenica Simone,Andrea Roveda,Aldo Montuoro,Cecilia Alfieri,Gioia De Angelis,Cristiano Gallucci,Buket Erer,Giancarlo Isacchi,Francesco Zinno,Gaspare Adorno,Alessandro Lanti,Lawrence Faulkner,Manuela Testi,Marco Andreani,and Guido Lucarelli.

December 2008
Haematologica:Allogeneic stem cell transplantation for thalassemia major -Emanuele Angelucci and Donatella Baronciani

November 2007
Blood reviews: Advances in the allogeneic transplantation for thalassemia –Guido Lucarelli,Javid Gaziev

November 2005
NY Academy of science:Unrelated bone marrow transplantation for beta-thalassemia patients:The experience of the Italian Bone Marrow Transplant Group –La Nasa G,Argiolu F,Giardini C,Pession A,Fagioli F,Caocci G,Vacca A,De Stefano P,Piras E,Ledda A,Piroddi A,Littera R,Nesci S,Locatelli F.

March 2004
Blood Journal:New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years –Pietro Sodani,David Gaziev,Paola Polchi,Buket Erer,Claudio Giardini,Emanuele Angelucci,Donatella Baronciani,Marco Andreani,Marisa Manna,Sonia Nesci,Barbarella Lucarelli,Reginald A. Clift,and Guido Lucarelli

February 1999
Blood Journal:Bone Marrow Transplantation in Adult Thalassemic Patients –G. Lucarelli,R.A. Clift,M. Galimberti,E. Angelucci,C. Giardini,D. Baronciani,P. Polchi,M. Andreani,D. Gaziev,B. Erer,A. Ciaroni,F. D’Adamo,F. Albertini,and P. Muretto

What is the HLA typing test?
The human leukocyte antigen (HLA) test,also known as HLA typing or tissue typing,is blood test and it identifies antigens on the white blood cells (WBCs) that determine tissue compatibility for organ transplantation.

There are different kinds of HLA typying test,which one do you recommend?
We recommend a high resolution HLA typing,with the locus detailed in this page.

HLA can be done in Bangalore at Narayana Health hospital? If yes,how much is the cost?
Yes it can be done and the cost is about 12000 rupees for high resolution HLA. If you can’t do it,then as first step start with the low resolution HLA.

I’ve done the low resolution HLA typing test for all family members and there isn’t any matched donor. Do I have to do anyway the high resolution HLA?
Yes,becacuse the high resolution is necessary to start searching a matched unrelated donor in a bone marrow donor bank.

How long does it take to have the results of the high resolution HLA typing test?
About 2 weeks.

As the results of HLA typing are variables among laboratories,is it worth to repeat the test in another laboratory?
If you can,yes. In any case,before the BMT,the hospital doing the BMT will do again the HLA typing.

If I do an HLA match test in one place,do I have to repeat it in the hospital where the BMT is done?
Yes,it is a best practice that the hospital doing the BMT repeats again the HLA match test,to be absolutely sure that the donor matches.

May I know more about the Thalassemia major BMT process?
A patient admitted to the bone marrow transplant unit will first undergo several days of chemotherapy and/or radiation which destroys bone marrow and cancerous cells and makes room for the new bone marrow. This is called the conditioning or preparative regimen
Prior to conditioning,a small flexible tube called a catheter (sometimes called a “Hickman®”or central venous line) will be inserted into a large vein in the patient’s chest just above the heart.
A day or two following the chemotherapy and/or radiation treatment,the transplant will occur. The bone marrow is infused into the patient intravenously in much the same way that any blood product is given.
The transplant is not a surgical procedure. It takes place in the patient’s room,not an operating room. Multiple antibiotics and blood transfusions will be administered to the patient to help prevent and fight infection.
Transfusions of platelets will be given to prevent bleeding. There also medication medications to prevent and control graft-versus-host disease.

How old a thalassemia major patient can be to be successfully cured with a BMT?
Thalassemic patients with a HLA identical donor has be transplanted as soon as possible,but the results in adult patients( age >17 years old) are not so brilliant.
Adult thalassemia patients are higher risk patients for transplant-related toxicity due to an advanced phase of disease and have a cure rate of 62% with current treatment protocol.
BMT is not the best therapeutic option for adult patients.

If I’ll have another baby he might have thalassemia major?
The probability that the new born will have thalassemia major is always 25%.

What is a BMT rejection?
The patient’s immune system may destroy the new bone marrow. This is called graft rejection.

What is the difference between a related/familiar and an unrelated donor?
A related donor is a family member of the patient (mother,father,brothers and sisters);an unrelated is anybody else,and it usually can be found in bone marrow donor banks.

I understand the risk of graft-versus-host disease (GVHD) is there in 5/6 HLA matched BMT. Actually the child is less than two year old and have 5/6 HLA matched sibling,do you suggest BMT?
5/6 is a risk of GVHD regardless of the age of the child.

My sister has baby boy in her womb and he has thalassemia major. Is there any cure before birth of baby? Because it is in initial stage.
There is little experience regarding bone marrow transplant in utero but wit very bad results
This kind of transplant it is possible in some case of immunodeficiency disease but no in thalassemia major.

If the patient doesn’t have an HLA matching sibling,can we do a search of an unrelated bone marrow donor from a bank?
You can try,but the probability to find matched unrelated donor is low,especially if the bone marrow bank has few donors with the same ethnic background of the patient.

How much does it cost to do a research of an unrelated bone marrow donor from a bank?
From 10,000 USD up to 400,000 USD,depending on how many searches are done,how many donors have a good match,and some other variables.

How long does it take to do a research of an unrelated bone marrow donor from a bank?
On average 6 months.

If I find an unrelated identical donor in a bank,will the donor certainly donate?
Usually yes;but in some rare cases it could happen that donor doesn’t want to donate anymore,or he is not in good health conditions.

If HLA match is 7 of 8 for adult donor from a bank is there any possibility for transplant?
We don’t suggest transplant from non HLA identical from the bank.

It is better to use as donor an HLA-matched sibling (brother or sister),or an unrelated donor from a bank?
For class 1 and class 2 patients,if the HLA match is 6/6 both for the matched sibling and for the unrelated donor,the results of the BMT are the same;for class 3 we use the sibling.
If it is 5/6 for both,we use the sibling.
If the sibling is 5/6 and the unrelated donor is 6/6,we use the unrelated donor.

During our daughter’s birth I had preserved her stems cells of the umbilical cord. Can they be used for a BMT?
No,because the patient can’t be a donor (autologous BMT),nor with his umbilical cord and nor with his bone marrow.

My son is thalassemia major,and his brother umbilical cord blood is stored at the time of the birth. Is it possible to use it?
You have to tell us where the umbilical cord is stored and you have to inform the hospital doctor team that you want to perform the HLA of umbilical blood to test the grade of antigen disparity. In other words we have to know if the cord blood is HLA identical or not.

My child is thalassemia major and he doesn’t have a matching sibling,can we use the mismatched unrelated cord blood?
The use of mismatched,unrelated cord blood is still experimental.

How easy it is to find a mismatched unrelated donor for cord blood transplant?
It is impossible to predict the probability to find a cord blood identical from the bank. Many factors are involved,so there isn’t an easy answer,but,from our experience,the possibility is low.

If I don’t have an HLA-matched compatible donor,is it possible to do the BMT using the patient himself as the donor?
No,autologous Bone Marrow Transplant it is not an option to cure thalassemia.

My daughter has Haemoglobin E-Beta thalassaemia. Can she do a BMT?
Yes,but only if she is transfusion-dependent and she is having regular blood transfusions.

My son has thalassaemia lepore. Can he be cured with BMT?
Yes,but only if he is transfusion-dependent and he is having regular blood transfusions.

I would like to get your opinion on reduced-intensity BMT. Between the traditional and the reduced,which procedure do you feel would be best for my son?
We don’t believe in reduced-intensity transplant,especially for very young children.

Is there a replacement for blood transfusions? I have not been feeling well lately after my blood transfusion. Is there anything I can do?
Unfortunately,you must keep doing do iron chelation therapy and transfusions. The only way to stop doing transfusions is a Bone Marrow Transplantation (BMT).

I have a child with alpha thalassemia and I want to know if he could receive BMT?
There is no experience in BMT in alpha thalassemia.

A thalassemia minor can do BMT to feel less exhausted?
No,only who is transfusion dependent can do a a BMT.

My son’s jaundice is high like 38-40.Is the BMT possible?
Yes it is possible,high it is not a problem.

As my son’s spleen has enlarged to 17.5 cm;doctors in our place have suggessted us to undergo splenectomy. Is there any problem for undergoing transplant in the future if the speen is removed?
If the spleen is too large it is better to remove it before the transplant. Spleen does not play a crucial role in term of the transplant. Before splenectomy a vaccination is needed.
After the transplant the child has to receive antibiotic therapy prophylaxis against bacterial infections,which,in his experience and the Lucarelli group,hasn’t caused any increase in mortality (with pre-splenectomy vaccination and post transplant antibiotic therapy prophylaxis)
If the enlarged spleen is removed before the BMT,there are these important benefits:

  • faster engraftment
  • reduced transfusion support

Furthermore,if it is not removed,its dimension could cause obstruction.

I have recently adopted a child who has been found to have a certain form of thalassemia. We have been called today to start transfusions and would like to understand if we can have a chance to receive BMT considering that our child is adopted and we do not know who and where they parents and/or relatives live.
First of all we need to know exactly what kind of thalassemia is. If there is no way to get in touch with the parents,brothers and sisters,you can try to search an unrelated matching donor through a donor registry bank. The search can only be done through an hospital.

BMT from haploidentical mother to child with thalassemia using protocol 30

If I don’t have an HLA-matched compatible donor,can the BMT be done using the mother (haploidentical BMT)?
There is another recent cure called BMT from haploidentical mother (mismatch donor),in which usually the donor is the mother or in some cases the father. It has been invented by Dr. Pietro Sodani and published on Blood journal on November 2009:
Purified T-depleted,CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia.
There is also a more recent update on Pediatric Reports in June 2011:
T cell-depleted hla-haploidentical stem cell transplantation in thalassemia young patients.

The mother is at least 50% identical,and the fetus inherited half from mother,half from father.
Haploidentical BMT has been explored as an option for treating patients with leukemia who lack an HLA-identical sibling or parent donor. Feto-maternal microchimerism suggests immunological tolerance between mother and fetus. Thus,we performed a BMT from mismatched mother to thalassemic patient without an HLA-identical donor.
If the patient doesn’t have an HLA identical donor from family and also from the bank,the mismatched transplant from mother could be a therapeutic strategy.

What are the results of the Haploidentical Mother BMT?
The best results are with very young children. Read more details in the results section of this page.

When the first Haploidentical Mother BMT was done?
In 2002,by Prof. Lucarelli and Dr. Sodani.

How many Haploidentical Mother BMT have been done?
53 since 2002 and you can read more details in this page.

What is the protocol for the Haploidentical Mother BMT?
It is protocol 30,developed by Dr. Pietro Sodani.

What is the minimum age for an Haploidentical Mother BMT?
1.5 years.

What is the payment mode? All money should be paid in one payment or in installments?
Payments are not to be done all together. Outpatient (for the first 60 days) they can do day to day billing. And inpatient week or two weeks at a time.

Can we do BMT with parent as donor even in case of mismatch?
Yes,because the mother and the father are often mismatch with the patient,and they can be used as donor in the Haploidentical BMT.

Can the mother be a donor if she is also thalassemic carrier/trait?
Yes,the mother of thalassemic child is by definition a carrier/trait,and she could be used as donor in the haploidentical BMT,since she is a normal person with an haemoglobin level around 10-12.

After a successful BMT,the child will be thalassemia minor/trait carrier?
Yes,the child will be a thalassemia minor carrier,with an haemoglobin level around 10-12.

My daughter has Thal Major.I want to know whether either of my husband or myself could be successful bone marrow donors for our daughter considering we’re second cousins (our dads are first cousins)?
Yes,you can be donors even if you are “second cousins”.

My daughter is beta thalassemia major,we want to go for Haploidentical BMT. We also did MLC test for that Lymphocyte crossmatch for T cell and B cell is negative. Can we proceed for Haploidentical BMT?
This test is not very important,and you can go for the Haploidentical BMT,but you first must do the pre transplant investigations and evaluation for the recipient(child) and the donor.

How long the child doing the Bone Marrow Transplantation(BMT) has to stay in the hospital?
Usually 35 days.

Are there risks for the mother who is the bone marrow donor?
In the BMT from haploidentical mother the procedure used for the donor is called Peripheral Blood Stem Cells (PBSC).Fewer than 1% of PBSC donors experience a serious side effect from the donation process.

PBSC donation may require placement of a central line if the donor doesn’t have suitable arm veins. A central venous line is a sterile tube that is inserted into one of the larger veins –the femoral vein,internal jugular vein or subclavian vein.
The risk of serious complications from use of a central line is small.
A central line will be placed only with the consent of the donor after he has received information about the possible risks.

Another potential risk is associated with Grow Factor injections,which is required for about 30% of the donors. Therefore,there are some data available about the long-term safety. We began using grow to aid in transplants in the 1990s. Since then,no donors (also from from the international registry) have reported any long-term complications from grow factor injections.

Will you give any medicine to the mother before taking out her bone marrow?
There are no medicines for the mother. Only growth factor 4-5 days before the BMT.

After haplo BMT for how much time immunosuppression is required?
Usually we are using cyclosporin and steroids at least for one year,depending from complication like GVHD.

After haplo BMT,when the vaccination can be done?
The child will be observed by doctors for 1 year after the BMT,and within 1 year the vaccination can start.

Our doctor says that we can do half matching with either father or mother,so haploidentical matching. it is better to use the mother or the father?
The first choice between father and mother is the mother;the reason is the intent to minimize the risk of severe GVHD,an immunological reaction,and it is based on the hypothesis of the immunological tolerance established during pregnancy between fetus and mother.

What is the percentage of required match of the mother?
It is not important if the mother is 50% match or 80% match.

My nephew has b+ blood group and his mother has o-blood group. Can BMT be possible?
It is OK,the different blood group is not a problem.

During the procedure of the mother do you require test to check how much bone marrow is matching? Or how you evaluate that the mother bone marrow is sufficiently matched to treat to the child?
No,we don’t. We know in advance all we need.

When the chemotherapy is given to the patient to remove his own existing bone marrow is this very painful? if it is painful any other medicine given to the patient to reduce the pain?
No,it is not very painful. We can control the side effects of chemotherapy using drugs.

How is the new bone marrow injected to the child?
Using a central line with a peripheral venous access,like a blood transfusion.

Once the new bone marrow is given to the child,how long it takes to engraft and be able to make new blood cells?
Approximately two weeks.

How long we need to take care after bone marrow transplant.i.e. after how many months the child can be able to go to school?
It depends on the outcome of the transplant,if everything will be fine,after 1 year from transplant the child can go at school leading a normal life.

After the transplant once the child body start to make blood,is there any chance again the body needs blood from outside or again body can reject?
Yes there is the probability of rejection,in this case we are going to infuse back his bone marrow which has been previosly cryopreserved. Rejection is one of the complications of bone marrow from HLA not identical donor.

Can a mother who is diabetic NIDDM (noninsulin-dependent diabetes mellitus) be the donor?
No,the mother in case of diabete is not able to be the donor.

Once the haploidentical BMT is done,is thalessemia cured permanently or is there any chance to diagnose after some years? Did you find such case where thalassemia diagnose after haploidentical done successfully?
In our experience after persistent engraftment there is not any rejection case. It could happen in the future,but so far it has never happened.

Is haploidentical BMT possible if HLA match 4 out of 6?
Yes,it is possible. Haploidentical means 50% identical.

In BMT from Haploidentical mother,have you observed any difference in results among class 1,class 2 and class 3 patients? Is the success rate different among the three classes of patients?
In class 1 and class2 the difference in term of survival is not so relevant.
In class 3 it is full of risks,it is not the right therapeutic option.

If the mother has a history of breast cancer surgery some years ago,can she be a donor?

If the BMT is done on children under the age of 2,are there short/term effects?
In very young patients,the possible side effects are the same of older patients.

Did Dr. Sodani perform BMTs on very young children i.e. between the age of 13 months –24 months?
Yes,and the younger the patient,the better are the results.

Once the BMT is done,how long does it take to stop doing the blood transfusions?
In most cases it happens within a month from the transplant. We anyway suggests to do a regular checkup for the first year,because there might be some complications related to the BMT,and also late rejections.

Once the BMT is done,do I still have to remove the excess iron,even if I don’t need anymore the blood transfusions?
Most patients keep doing iron chelation for 1 year and a half after the transplant,with a procedure called phlebotomy (salasso-therapy).

My son is thalassemia major and I have done his HLA Typing and there is a 4/6 match with the father and unfortunately we are unable to get 6/6 match
He could do the haploidentical BMT from the mother,or alternatively from the father.

My son has thalassemia major,we have a 4/6 match from the father but unfortunately we didn’t find any 6/6 match. What we should do?
In this case it is better to use the mother (haploidentical BMT) as the donor;if for any reason the mother can’t be used,then the father is the second best option.

My son doesn’t have an HLA-matched sibling,and I’ve found maximum a 5/6 match in the unrelated donors bank. We want to go for another baby only as last option,what are the risks and the chances?
If you’ll have another baby,there is a 25% probability that he will have thalassemia major as well. Having said that,there is a 25% probability that he will be a 6/6 HLA match. You can also consider to have haploidentical BMT from the mother.

My daughter is 16 months old,would it be worth waiting to do the BMT when she’s 2 years old so that her brain has developed fully?
We suggest to do the BMT as soon as possible.

Is it necessary for the patient to keep particular food diet if he plans to do a BMT?
Food is not an issue,the patient can eat every kind of food.

What is a “non identical” donor?
It means that result of the HLA typing is less than 6/6 (which is the identical matched donor),so 5/6 or less. It is also called mismatched.

If I don’t have an HLA-matched sibling,it is better to use an unrelated donor (from a bank),or the mother?
It is better the unrelated donor only if the patient is class1 or class2,and if the HLA match of the donor is 6/6.
In all other cases (HLA match of 5/6 or lower;every class3 patient),it is better to use the mother (haploidentical BMT);anyway,if the donor has the same HLA typing of the mother,he can be used,especially if the mother is not available.

BMT from matching sibling

What is Bone Marrow Transplantation (BMT) from a matching sibling (allogeneic BMT)?
Bone marrow transplantation (BMT) is used to treat diseases once thought incurable. Since its first successful use in 1968,BMTs have been used to treat patients diagnosed with leukemia,aplastic anemia,thalassemia major,lymphomas such as Hodgkin’s disease,multiple myeloma,immune deficiency disorders and some solid tumors such as breast and ovarian cancer.

In a bone marrow transplant,the patient’s diseased bone marrow is destroyed and healthy marrow is infused into the patient’s blood-stream. In a successful transplant,the new bone marrow migrates to the cavities of the large bones,engrafts and begins producing normal blood cells.

If bone marrow from a donor is used,the transplant is called an “allogeneic”BMT;the new bone marrow infused into the patient must match the genetic makeup of the patient’s own marrow as much as possible. A blood test,called “HLA typing”,is needed to know if donor’s bone marrow matches the patient’s.

Who has invented the Bone Marrow Transplantation (BMT) for thalassemia??
Prof. Lucarelli,in the 1980’s in Italy;he is Cure Thalassemia scientific advisor.

Who has the greatest experience in the world in Bone Marrow Transplantation (BMT) for thalassemia?
BMT for Thalassemia has been successfully performed since 1980’s on more than 3,000 patients worldwide. Prof. Lucarelli (Cure Thalassemia scientific advisor) and his team did about 1.500 BMT’s (about 50% of all the BMTs done in the world) in the last 30 years;Dr. Pietro Sodani (Cure Thalassemia scientific coordinator) did about 400 of them.

On PubMed,a free database managed by the US National Library of Medicine and the National Institutes of Health accessing primarily the MEDLINE database of references and abstracts on life sciences and biomedical topics,there are 292 scientific papers co-authored by Prof. Lucarelli and 27 by Dr. Sodani.

What are the results of Bone Marrow Transplantation (BMT)?
The results have substantially improved over the last two decades,due in large part to improvements in preventive strategies,the effective control of transplant-related complications,and the development of new preparative regimens.
The youngest the patients,the better are the results. In low risk (class 1 and class 2) young children in good shape,the thalassemia free is 89%,rejection is 8% and the mortality is 3%.
In class 3 patients,the thalassemia-free is 85%,the rejection is 8% and the mortality is 6%.

Are there risks,complications and infections after a Bone Marrow Transplantation(BMT)?
First of all,there is a mortality risk,which is significantly lower than some decades ago;it goes from 3% to 6%,depending on the class of risk of the patient,his health conditions and the age.

BMT may not be successful causing a rejection,because of disease recurrence or regimen-related toxicity.

Having said that,in about 30% of the BMTs there might be complications,side effects or infections,and they are managed successfully;they are less frequent than some years ago thanks to a more accurate diagnosis and a higher number of antibacterial. The probability depends on some variables,like the class of risk of the patient and the outcome of the transplant.

Regimen-related toxicity refers to side effects that patients may experience during the course of transplantation. These side effects include graft-vs-host disease (GvHD),bacterial infections,fungal infections,viral infections,gastrointestinal and hepatic complications,neurologic complications,pulmonary complications,and late effects after stem cell transplant.

Infection complications are one of the most serious side effects;in same rare cases there is the fungal infection aspergillus,and if it is not treated well,it could be dangerous.
Conditioning regimens that usually consist of high-dose chemotherapy,radiation therapy,or both,can cause mucositis. Late effects after BMT includes endocrine (gland) problems like hypothyroidism,adrenal insufficiency or growth hormone insufficiency.

Although improvements in conservative treatment have considerably improved the prognosis of thalassemia,disease- and treatment-related complications in these patients progress over time,causing severe morbidity and shortened life expectancy,especially when the patient is not young.

Do patient’s appearances completely change after transplant (excessive weight gain,hair growth due to steroids)? Are these changes permanent?
All the complications related to weight gain and changed appearance are not permanent,and they are exclusively visible during the time of the therapy with cortisone and immunosuppressive drugs.
The length of these complications is variable,and it could happen that a patient during a cortisone therapy could have permanent problems to bones,joints and head of femur,but it is very rare.

Are there risks for the bone marrow donor?
We want to assure donor safety,but no medical procedure is risk-free. A small percentage(1.3%) of donors experience a serious complication due to anesthesia or damage to bone,nerve or muscle in their hip region.

The risk of side effects of anesthesia during marrow donation is similar to that during other surgical procedures. Serious side effects of anesthesia are rare. Common side effects of general anesthesia include sore throat(caused by the breathing tube) or mild nausea and vomiting. Common side effects of regional anesthesia are a decrease in blood pressure and a headache after the procedure.

What are the different classifications of patients of the Lucarelli protocol for BMT?
Prof. Lucarelli classifies the patients in 3 classes on the basis of presence of the risk factors hepatomegaly (enlarged liver),liver fibrosis and iron chelation history. Each class has different probability of cure.
Lucarelli Staging System for BMT:
Class 1
Absence of hepatomegaly (enlargeed liver),regular iron chelation before transplant,absence of fibrosis in pretransplant liver biopsy result.
Class 2
Hepatomegaly,a history of irregular iron chelation before transplant,histological evidence of liver fibrosis,or various combinations of the above.
Class 3
All of the following:large liver,poor compliance with chelation therapy,liver damage
Class 2 or 3,irregular iron chelation,with a range of clinical symptoms and other diagnoses.

How do I understand the class of a patient?
We need
1) to know when the patient has started the blood transfusions and the iron chelation
2) a liver biopsy or the more recently applied noninvasive T2* magnetic resonance imaging (MRI)
3) a liver ultrasound.

How many different Lucarelli protocols exist,and for which class of patients are they used?
In the last 30 years,Prof. Lucarelli has developed 3 different protocols,each one with a different drug regimen:
a) 6 for class 1 and 2
b) 6.1 for class 1 and 2 if the patient is less than 3 years old
c) 26 for class 3.

What is the minimum age for a BMT?
Thalassemic patients with an HLA identical donor should be transplanted as soon as possible.

How do I know if I have a compatible donor?
You need to perform an HLA typing testing.

What is the probability to find an HLA-matched sibling donor?
Approximately 25% of thalassemic patients could have a sibling donor whose bone marrow is a perfect match.

What is the minimum age for bone marrow donor?
After 1 year of age.

What has to be done to know if a thalassemia patient can have a Bone Marrow Transplantation (BMT) from an HLA matching identical donor?
1) We need to know:

  • the age of the patient
  • if he has brothers and sisters
  • when he started the blood transfusions
  • the history of the iron chelation
  • the most recent checked iron concentration
  • if he is Hepatitis C Virus (HCV) positive
  • the class of risk.

2) We need to know if there is an HLA matching identical donor in the family (father,mother,brothers and sisters). If not,we have to explore the possibility to open the bank research,but should be very expensive.

3) If there isn’t an HLA identical donor in the family and also from the bank,the mismatched haploidentical BMT from mother could be done.

If the donor has thalassemia intermedia,he can be used to do the Bone Marrow Transplantation(BMT)?

How do you collect the bone marrow from the donor?
It is collected under general anesthesia from the upper part of hip bone. It is done in day hospital.

When is there a perfect HLA match (identical donor)?
It is called 6/6 match,both from a related/familiar donor or from an unrelated.

Can the mother be an identical donor?
Yes,the mother can be 100% phenotypically identical if she shares one haplotype with her husband. In this case the patient can have a BMT using the mother ad the identical donor,and there is a higher risk of graft-vs-host disease (GvHD).

If the result of the HLA typing is 5/6,can the donor be used for the BMT?
It could be used,but we have the study the results of the HLA typing,because there might the risk of graft-versus-host disease (GVHD) and can be life-threatening.
This applies both for a related/familiar donor and for the unrelated.

Which is the minimum result of the HLA typing to know if the match is good enough so that the donor be used for the BMT?
The minimum is 5/6,with 4/6 or less it can’t be done.

What happens if the donor’s bone marrow is not a perfect (6/6) genetic match?
It could perceive the patient’s body as foreign material to be attacked and destroyed. This condition is known as graft-versus-host disease (GVHD) and can be life-threatening.

If everything goes smooth and the BMT is successful,the patient will lead a normal life?
Yes,especially if the BMT is done as soon as possible.

What are the tests required to be done for a BMT,once that the donor has been found?
Abdominal Ultrasound,Chest X-rays and other ones that the doctor will ask,after having evaluated the the situation of the patient.

My daughter has thalassemia major and she has an HLA identical sibling. Should we do the BMT as soon as her ferritin level is reaching the threshold where she would need to start the iron chelation therapy?
The BMT should be done as soon as possible,without waiting the need to start the iron chelation therapy.

What is better,a BMT from a HLA-matched sibling at a later age or Haploidentical Mother BMT at the earliest age possible?
It is always better to do a BMT from an HLA-matched sibling,there are higher results and less risks.

Can I plan to have 1 more baby and use the umbilical cord blood for the BMT?
In case of HLA compatibility of a matching sibling,we prefer to use the bone marrow and not the cord blood,because with the cord blood there is a higher risk both of rejection and to have a graft-versus-host disease (GVHD).

If a thalassemia patient is Hepatitis C Virus (HCV) positive,can he receive Bone Marrow Transplantation(BMT)?
If there is an HLA-matched identical donor we can do the BMT for a patient who is HCV positive.
HCV is not an issue,we just have to be more careful to prevent infections.

If the donor is Hepatitis C Virus (HCV) positive,can we do anyway the Bone Marrow Transplantation(BMT)?
No,if the donor is HCV positive,his bone marrow can’t be used.

Cure Thalassemia-Narayana Health/Mazumdar Shaw Cancer Centre

Where can I read more?
We have a page dedicated to this topic.

Do you accept patients from all over the world?
Yes,we are glad to have thalassemia patients from all over the world.

Social business

What is a social business?
A Social Business (as defined by by the 7 principles of the Nobel Prize Yunus) is a non-loss,non-dividend company designed to address a social problem,and not to look for profit maximization. The investors cannot take any dividend;it is financially self-sustainable,without relying on donors’ money. People working in it are paid at market prices.

Who is Prof. Yunus?
He is the inventor of micro-credit,Grameen Bank and the social business concept. Nobel Prize winner in 2006. Read more in his website.

Why Cure Thalassemia is a social business and not a charity or a “for profit” company?
Charity is hard to be financially self-sustainable you need a constant flow of donors money,and it is though to do Bone Marrow Transplantation (with 3 doctors,8 nurses and 2-3 other staff people) just rely on donors.
At Cure Thalassemia we believe that money is not the most important thing in like,and that’s why we have decide to follow the Social Business of Prof. Yunus,where investors don’t get any profit,and the goal of the company is to solve a social problem,in this case to cure thalassemia patients.