Waldenstrom Macroglobulinemia

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Treating Waldenstrom Macroglobulinemia TOPICS

Stem cell transplant for Waldenstrom macroglobulinemia

The doses of chemotherapy drugs (and radiation) doctors can give are limited by the side effects they can cause. Higher doses can’t be used, even if they might kill more cancer cells, because they would severely damage the bone marrow, where new blood cells are made. This could lead to life-threatening infections, bleeding, and other problems due to low blood cell counts. Doctors can try to get around this problem by giving an infusion of blood-forming stem cells after treatment. These stem cells settle in the bone marrow, where they can create new blood cells.

A stem cell transplant (SCT) is not a common treatment for Waldenstrom macroglobulinemia (WM), but it might be an option in younger patients for whom other treatments are no longer working.

Blood-forming stem cells used for a transplant come either from blood (for a peripheral blood stem cell transplant, or PBSCT) or from the bone marrow (for a bone marrow transplant, or BMT). Peripheral blood stem cells are obtained using a procedure similar to a blood donation, while bone marrow donation is usually done in an operating room (while the donor is asleep under general anesthesia). Bone marrow transplants were more common in the past, but they have largely been replaced by PBSCTs.

There are 2 main methods of SCT, based on whether the blood-forming stem cells that are used come from the patient or from a donor.

Autologous stem cell transplant

This is the type of transplant used most often in WM. In an autologous SCT, a patient’s own blood-forming stem cells are removed from his bloodstream and stored to use later. Then high doses of chemotherapy are given to kill the WM cells. The high doses of chemotherapy kill the normal bone marrow cells as well as the cancer cells. After chemotherapy, the frozen stem cells are thawed and returned to the body (like a blood transfusion).

Autologous transplants can help some people with WM, but doctors are still trying to figure out which patients will benefit the most.

Allogeneic stem cell transplant

This is a treatment that is still being studied for WM, and experts recommend it be done only as part of a clinical trial.

In an allogeneic SCT, the stem cells for the transplant come from someone else (a donor). The donor’s tissue type (also known as the HLA type) needs to match the patient’s tissue type as closely as possible to help prevent the risk of major problems with the transplant. Usually this donor is a brother or sister if they have the same tissue type as the patient. If there are no siblings with a good match, the cells may come from an HLA-matched, unrelated donor – a stranger who has volunteered to donate their cells.

The stem cells for an allogeneic SCT are usually collected from a donor’s bone marrow or peripheral (circulating) blood on several occasions. Regardless of the source, the stem cells are then frozen and stored until they are needed for the transplant.

Allogeneic transplants have more risks and side effects than autologous transplants, so patients typically need to be younger and relatively healthy to be good candidates. Another challenge is that it can sometimes be difficult to find a matched donor.

One of the most serious complications of allogeneic SCTs is known as graft-versus-host disease (GVHD). It happens when the patient’s immune system is taken over by that of the donor. When this happens, the donor immune system may consider the patient’s own body tissues to be foreign and reacts against them.

Symptoms can include severe skin rashes, itching, mouth sores (which can affect eating), nausea, and severe diarrhea. Liver damage may cause yellowing of the skin and eyes (jaundice). The lungs may also be damaged. The patient may also become easily fatigued and develop muscle aches. Sometimes GVHD can become disabling, and if it is severe enough, it can be life-threatening.

Non-myeloablative transplant: In this newer approach to allogeneic SCT (also called a mini-transplant), lower doses of chemotherapy or radiation therapy are used than in traditional allogeneic SCT. Patients are given drugs to suppress their immune system. This allows the donor cells to grow and partly take over the patient’s immune system. The donor cells then begin reacting against the lymphoma cells and killing them (known as a graft-versus-lymphoma effect).

This type of transplant may be an option for some patients who couldn’t tolerate a regular allogeneic transplant because it would be too toxic. But this type of transplant can still cause graft-versus-host disease (GVHD), which can make patients very sick.

Doctors are trying to refine this treatment to work against the lymphoma cells without affecting the normal cells.

Practical points

A stem cell transplant is a complex treatment that can cause life-threatening side effects because of the high doses of chemotherapy used. Be sure you understand the possible benefits and risks. If the doctors think you might benefit from a transplant, it should be done at a hospital where the staff has experience with the procedure and with managing the recovery phase. Some stem cell transplant programs might not have experience in certain types of transplants, especially transplants from unrelated donors.

SCTs often require a long hospital stay and can be very expensive (costing well over $100,000). Because some insurance companies might view it as an experimental treatment, they might not pay for it. Even if the transplant is covered by your insurance, your co-pays or other costs could easily amount to tens of thousands of dollars. Find out what your insurer will cover before deciding on a transplant so you will have an idea of what you might have to pay.

For more information about stem cell transplants, please see our document Stem Cell Transplant (Peripheral Blood, Bone Marrow, and Cord Blood Transplants).


Last Medical Review: 06/19/2013
Last Revised: 06/19/2013