Bone marrow transplant from non HLA identical relatives

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Offline zahra

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Bone marrow transplant from non HLA identical relatives
« on: May 31, 2011, 03:07:51 PM »
Hi ,
I came across this

http://bloodjournal.hematologylibrary.org/content/115/6/1296.full.html

From what little I understand there has been success in transplants from haploidentical (Does that mean one HLA match? and other not exactly same?) close relatives. These are mostly mothers but some brothers

Manal has posted about it before . Anyone know how its progressing? I have heard a Pakistani doctor is going to Japan to learn this technique. Does this mean it is improved now?

Zahra

« Last Edit: May 31, 2011, 03:20:12 PM by zahra »

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Offline Manal

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #1 on: May 31, 2011, 11:55:37 PM »
Dear Zahra,
Haploidentical transplant refers to the transplant from the mother to the child regardless she is a match or not. The idea of this procedure is based on the idea that a pregnant woman does not reject her baby though the baby is different than her body in terms of everything (blood, tissues, blood group,....)

A certain engraftment happened between the blood of both the mother and the child which will not let the child fully reject the mother blood though they may not match.

This special engraftment disappears as the child grows.

I remember that in 2007, Prof Lucarelli sis this technique on 15 patients. Nine succeed,4 were rejected and back to thal again one or two died, I am not sure, but I am sure that the success rate at this time was 67%
And by all means the total number of this type of transplants does not count as big number where you give accurate statistics.

What I knew later that Prof Lucarelli stopped this kind of transplant.

By coincidence, I got to know that Prof Lucatly (another known Italian professor too from Calgary) perform these kind of transplants but he is seen as a high risk taker and these types of transplants are not highly appreciated from the European medical community (this what I have known from talking to a transplant doctor in the last Egyptian conference)

Hope this helps
Manal

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Offline Andy Battaglia

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #2 on: June 01, 2011, 12:47:26 AM »
I remember a comment Dr Sadelain, the gene therapy pioneer made at the Dubai conference in 2006 about the lack of science behind mother to child BMT. I have to agree. I will be much more comfortable with it when they understand why it works for some people. The mother is only a half match and theoretically, this shouldn't be enough. When they can explain how and why, you can expect to see much higher success rates with this method.
Andy

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Offline zahra

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #3 on: June 02, 2011, 06:39:34 AM »
Thank you Manal and Andy.

I asked my sons doctor yesterday about the differences between the various kinds of transplants. He explained that the difference is in the chances of rejection and GVHD. The safest ( with little chance of GVHDand if there is GVHD then it is transient) is HLA identical sibling cord blood. In fact if you list them according to increasing GVHD it would be

HLA matched sibling cord blood.
HLA matched sibling bone marrow
HLA matched unrelated cord blood
haploidentical mother bone marrow
HLA matched unrelated bone marrow

He said he thought the risk was too great in HLA identical unrelated bone marrow and that he wouldnt reccomend it.

Zahra




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Offline surendra

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #4 on: June 02, 2011, 06:47:12 PM »
Hello Andy                                                      my daughter is 15 months old and we are thinking for BMT. Is it safe for her and which one is better for her? In which BMT side effects are very low :

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Offline Andy Battaglia

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #5 on: June 02, 2011, 08:34:49 PM »
I have to agree with what Zahra was told. Sibling cord blood is the method I feel is doing the best job with the least amount of side effects.
Andy

All we are saying is give thals a chance.

Re: Bone marrow transplant from non HLA identical relatives
« Reply #6 on: September 03, 2011, 12:28:42 PM »
Half-Matched Transplants Widen Pool of Donors for Leukemia and Lymphoma
ScienceDaily (July 8, 2011) — Identifying a suitable donor for leukemia and lymphoma patients who need bone marrow transplants may be far easier now that results of two clinical trials show transplant results with half-matched bone marrow or umbilical cord blood are comparable to fully matched tissue, thanks in large part to the availability of effective antirejection drugs and special post-transplant chemotherapy. The finding means that nearly all patients in need of a transplant can find donors, according to Johns Hopkins scientists who participated in the trials.

Plans are under way for a four-year randomized trial for so-called haploidentical marrow or cord blood transplants in 380 patients to begin late this year or early next year. Many large medical centers, including Johns Hopkins, are expected to participate.

The results of the two studies are good news, Hopkins researchers say, because they address the problem faced by patients when no family members are a complete match for the patient's tissue type. Although patients and physicians may then seek donors through national registries, as many as half or more of patients looking for matches in these registries don't find one, and the search can take weeks to months.

During this time, a patient's disease can progress, notes bone marrow transplant expert Ephraim Fuchs, M.D., who adds that it is especially difficult for minorities to find matches because of their underrepresentation in national registries.

"People are dying waiting for matched donors from a registry," says Fuchs, associate professor of oncology at the Johns Hopkins Kimmel Cancer Center.

In the clinical trials, investigators from the Blood and Marrow Transplant Clinical Trials Network at 27 medical centers tested two types of transplants, those from half-matched (or haploidentical) bone marrow or cord blood, and published their results in the July 14 issue of Blood. Some 50 adult leukemia or lymphoma patients with advanced disease or at high risk for relapse were included in each of the Phase II trials. Six of the participating centers conducted both types of transplants. Johns Hopkins conducted the haploidentical transplant trial only.

Transplants with cord blood, collected from the umbilical cord and placenta after a baby is born, are generally done in children because of the small number of cells in a single unit of cord blood. Adults, on the other hand, need two units.

Bone marrow tissue is extracted by needle from the hip bone of donors. Marrow that is half-identical to a patient's tissue type can be obtained from parents, children and most siblings.

Results of the two trials show one-year survival rates of 54 percent for cord blood transplant and 62 percent for haploidentical marrow. Survival without disease progression at one year was 46 percent for cord blood and 48 percent for haploidentical marrow. The investigators say this is comparable to survival achieved by similar patients undergoing transplants from fully matched siblings or unrelated adult donors.

Relapse rate after one year was 45 percent for haploidentical marrow transplants and 31 percent for cord blood. No patients had severe graft versus host disease (GVHD) -- an attack on the patient's normal tissues by immune cells of the donor -- after bone marrow transplant.

At one year, deaths not caused by relapse occurred in seven percent of haploidentical patients and 24 percent of cord blood transplants.

Fuchs says that in the past, haploidentical transplants failed more often because the transplanted cells caused severe GVHD, especially in older patients. Half of the patients enrolled in the current trials were 50 and older.

"Ten years ago, it was unthinkable to do a haploidentical transplant," says Fuchs, who led the haploidentical transplant clinical trial.

To overcome the GVHD problems, Fuchs and colleagues at the Johns Hopkins Bone Marrow Transplant Program pioneered the use of a chemotherapy drug called cyclophosphamide after the transplant to minimize the effects of GVHD. The drug wipes out the patient's immune system, leaving the transplanted blood stem cells intact. The stem cells create new disease-free blood cells in the patient.

The investigators also believe that a lower-intensity "conditioning" regimen of chemotherapy and radiation before the haploidentical marrow transplant may help prevent severe GVHD. The outpatient conditioning treatment lasts six days. Then, the donor's bone marrow is harvested and, that same day, infused intravenously into the patient. This is followed by two days of high-dose cyclophosphamide, and then, other immune-suppressing drugs.

Within 16 to 24 days, patients begin forming new blood cells including white blood cells and platelets, important for fighting infection and clotting blood. Thirty to 40 percent of patients are able to receive their transplants on an outpatient basis, but some patients are admitted for fevers or infections. Patients are monitored carefully and remain in proximity to the hospital for 60 days after the transplant.

Patients receiving cord blood transplants follow a similar regimen, but have seven days of pre-transplant chemo and radiation therapy and receive cord blood from two separate donors.

Fuchs anticipates that haploidentical bone marrow transplants may be tested more widely in nonmalignant diseases, such as aplastic anemia, lupus, and sickle cell anemia. Haploidentical transplants using post-transplant cyclophosphamide are being performed in medical centers outside of the U.S., including Italy, Thailand, Singapore, Israel, Australia, Belgium and England.

Funding for the clinical trials was provided by the National Heart, Lung and Blood Institute and the National Cancer Institute.

Claudio Brunstein, M.D., Ph.D., at the University of Minnesota, led the cord blood transplant clinical trial. Other investigators involved in the trials were Shelly Carter and Juan Wu from the Emmes Corporation, a data collection center; Chatchada Karanes from the City of Hope; Luciano Costa from the Medical Unversity of South Carolina; Steven Devine from the Ohio State University; John Wingard from the University of Florida; Omar Aljitawi from the University of Kansas; Corey Cutler from the Dana Farber Cancer Institute; Madan Jagasia from Vanderbilt University; Karen Ballen from Massachusetts General Hospital; Mary Eapen from the Medical College of Wisconsin and Paul O' Donnell from the Fred Hutchinson Cancer Center.

http://www.sciencedaily.com/releases/2011/07/110707101956.htm

Re: Bone marrow transplant from non HLA identical relatives
« Reply #7 on: March 06, 2012, 01:34:02 AM »
Hi Andy,

I am planning to do the bone marrow transplant to my daughter. We have appointment on 27/3/2012 to test whether Fatin's blood is matched to her younger sister (4yrs) or not. But I'm very worried either the procedure is safe or not to my both daughters. Could you please give some opinion about this.

Thanks in advance

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Offline Andy Battaglia

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #8 on: March 06, 2012, 02:54:28 AM »
The matching is done by simple blood tests that only involve taking some blood from each child by needle. There is no danger at all.
Andy

All we are saying is give thals a chance.

Re: Bone marrow transplant from non HLA identical relatives
« Reply #9 on: March 07, 2012, 01:16:11 AM »
Owh I see... but what if their blood are 100% matched and the BMT has to be committed, then what is the chances to cure my daughter? how successful is the BMT so far especially to young children? what is the effect to the other daughter? I am so worried...  :scared

thanks for your attention

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Offline Andy Battaglia

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Re: Bone marrow transplant from non HLA identical relatives
« Reply #10 on: March 07, 2012, 06:47:21 AM »
There is no commitment by getting the test. If they match, then you think about whether or not to go ahead with the BMT. The success rates vary from clinic to clinic. Not all BMTs work and there is also a risk of death, but success rates have risen in recent years. With stem cell transplants, stem cells are collected from the blood of the donor, so there is no risk to the donor.

Deciding on a BMT is a decision that requires much thought and familiarization with the entire process. I suggest that you continue to learn about BMT. I am very happy that we have a new ally in www.curethalassemia.org and that they are planning on adding a section on their website that will give testimonials from parents and patients who have gone through BMTs. Even now, you can send questions to Dr Pietro Sodani through their website. This is a wonderful resource and I recommend looking at the FAQ page and then sending any questions you have to Dr Sodani.
Andy

All we are saying is give thals a chance.

Re: Bone marrow transplant from non HLA identical relatives
« Reply #11 on: March 07, 2012, 11:33:17 AM »
Thanks Andy for the useful information...

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Offline EugenioLaMesa

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Re: Bone marrow transplant success rate from 100% matched sibling
« Reply #12 on: March 17, 2012, 01:24:49 PM »
Owh I see... but what if their blood are 100% matched and the BMT has to be committed, then what is the chances to cure my daughter? how successful is the BMT so far especially to young children? what is the effect to the other daughter? I am so worried...  :scared

thanks for your attention

I paste you the appropriate FAQ of the Cure Thalassemia site:
http://www.curethalassemia.org/faq/
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%.
Eugenio La Mesa
Co-founder and Chairman
Cure Thalassemia
Live without Thalassemia major - No more transfusions
Become our fan on Facebook: http://fb.com/CureThalassemia

 

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