Bone Marrow Transplantation

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  Treating graft-vs-host disease
  Collecting stem cells for future BMT
  Cord blood transplantation
  Peripheral blood stem cell transplant vs. regular BMT
  Statistics on "mini-transplants"
  Platelet counts after BMT
  ANC response after transplant

Treating graft-vs-host disease

How is graft-vs-host disease treated in people who have had BMT for aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria? 

Patients with these disorders receive the same graft-vs-host prophylaxis (prevention) as do patients with other diagnoses.  This means immune suppression and/or T-cell depletion of the marrow.  The strength of the suppression depends upon the type of donor and the degree of match. There is no one regimen that is considered as a "standard." 

Graft-vs-host disease (GVHD) is treated with powerful immune suppressive medicines including steroids, methotrexate, mycophenolate, rapamycin, cyclosporine, and tacrolimus (Prograf), to name some of the more commonly used agents.  Also used are ATG (antithymocyte globulin) and pentostatin (Nipent).  There are different approaches to acute and chronic GVHD.

Contact the BMT InofNet for general information on graft-vs-host disease and other issues associate with bone marrow transplants.  BMT InfoNet can be reached at 888.597.7674 or www.bmtinfonet.org.

Collecting stem cells for future BMT

Can aplastic anemia patients treated successfully with ATG and currently in remission then have their functioning stem cells collected and stored for future use in BMT with their own stem cells?

That is an interesting question. Technically this is possible. However, patients who are "in remission" from aplastic anemia have fewer

stem cells than normal. In addition, the cells that they do have are not normal in their function. Finally it is not clear that using a person's own stem cells would be wise since the body turned against them in the first place.

Cord blood transplantation

Is cord blood used for bone marrow transplantation in aplastic anemia and MDS patients – children or adults? How does it compare to other sources of stem cells – success rates, risk of graft-versus-host disease, other risks?

Cord blood has been used for these diseases with good success. There is some question as to whether graft rejection rates are higher than expected in patients with aplastic anemia who receive cord blood transplants. This is being tested prospectively.

Peripheral blood stem cell transplant vs. regular BMT

What are the advantages and disadvantages for both donor and patient between peripheral stem cell and a regular bone marrow transplant?

In both procedures, doctors are collecting blood

stem cells from the donor and transplanting them into the patient. The difference is the source of stem cells.

If a patient is having a bone marrow transplant, the donor is put under general anesthesia and a large needle is inserted in the hip bone.

Soft tissue occupying the inner cavities of bones responsible for blood cell production.

Bone marrow, which contains blood stem cells, is withdrawn with a syringe. The donor experiences soreness in the hip area, and may experience discomfort up to 2 weeks.

If a person is donating peripheral blood stem cells, general anesthesia and overnight hospital stay are avoided. The donor must first take injections of a drug called Filgrastim (

Filgrastim (G-CSF) and Sargramostim (GM-CSF) promote white cell growth in the bone marrow and are known as hematopoietic (blood forming) growth factors or colony stimulating factors. They are a man-made (synthetic) version of the colony stimulating factors produced naturally in your body. These drugs are prescribed "off label" for patients with MDS and other patients with low white cell counts (neutropenia).

G-CSF) over a four to five day period. Filgrastim causes the stem cells in the bone marrow to move into the blood stream where they can be collected. Filgrastim is usually tolerated well, though some donors experience side effects. Side effects usually resolve a few days after injections of filgrastim are stopped. Stem cell collection is a relatively painless procedure. Stem cells are collected from the donor's blood stream by inserting a needle into one arm vein.

In general, patients transplanted with stem cells collected from the bloodstream engraft slightly more rapidly than those transplanted with bone marrow. Some studies have found a slight increase in the risk of developing graft-versus-host disease for patients transplanted with peripheral blood stem cells, but it is not absolutely clear that this is the case.

Statistics on "mini-transplants"

Are there any statistics on the success rate of mini-transplants on MDS patients?

Mini-transplants, also called non-myeloablative transplants, use a less toxic pre-treatment regimen (chemotherapy) than is used for standard bone marrow transplants.  This method of transplantation is considered investigational; there are not sufficient numbers of reported cases to give a firm answer to this question. The conservative approach is to reserve mini-transplants for patients who are not eligible for full transplants (because of older age, the lack of an appropriately matched donor, or other reasons).

Platelet counts after BMT

Is it unusual for platelet levels to vary from 100,000 to 150,000 range six months after transplant for aplastic anemia?

That

platelet level is below the normal level (150-450,000). In some patients there is slow improvement while in others the low level persists. Regardless, you should be able to participate in all normal activities with platelet counts in the 100,000-150,000 range.

ANC response after transplantation

How long does it take for a 14 year old aplastic anemia patient, who received marrow from a younger matched sibling donor, to show high (over 500) ANC counts?

In most cases this average is 14-21 days, but there is a wide range.