Stem Cell Transplant for Myelodysplastic Syndrome (MDS)

A stem cell transplant (SCT), also known as a bone marrow transplant (BMT), might be an option to treat some people with myelodysplastic syndromes (MDS), especially people with higher-risk MDS.

In this treatment, a person first gets high-dose chemotherapy and/or total body irradiation to kill the cells in the bone marrow (including the abnormal bone marrow cells). Then they get new blood-forming stem cells to help rebuild the bone marrow.

Types of stem cell transplants

The main types of stem cell transplants differ in the source of the blood stem cells used (who they come from).

Allogeneic stem cell transplant

For an allogeneic (ALLO) SCT, after a person has received treatment to kill the  bone marrow cells, they get new blood-forming stem cells from another person (a donor). This is the type of transplant typically used for MDS.

Autologous stem cell transplant

In an autologous (AUTO) SCT, the person has some of their own stem cells removed before treating the bone marrow, and then gets them back after treatment. While this type of transplant tends to cause fewer side effects, it’s not as effective in treating MDS, so it’s rarely used.

When is a stem cell transplant used to treat MDS?

Allogeneic SCTs are most often done in people with higher-risk MDS, as they offer the best chance to put the MDS into long-term remission. They might also be an option in people for whom other treatments are no longer working.

Allogeneic SCTs can have serious, even life-threatening, side effects, so they are most often done in people who are younger and in relatively good health. There is no strict age cutoff, but in general, the younger and heathier a person is, the more like it is they can tolerate the intense treatment.

Non-myeloablative transplant (mini-transplant): Many people who are older or who have other health issues might not be able to tolerate a standard allogeneic SCT using high doses of chemo. Some people may still be able to get a non-myeloablative transplant (also known as a mini-transplant or reduced-intensity transplant).

For this treatment, a person gets lower doses of chemo and radiation that don’t destroy the cells in their bone marrow completely. They then get the allogeneic (donor) stem cells. These cells enter the body and establish a new immune system, which sees the MDS cells as foreign and attacks them.

A non-myeloablative transplant can still sometimes work, while having less severe side effects. In fact, a person can often get the transplant as an outpatient.

Side effects of stem cell transplants

The side effects from a SCT are similar to the side effects expected from chemotherapy and radiation, although they might be more severe because of the higher doses used.

One of the most serious side effects is low blood cell counts, which can lead to risks of serious infections and bleeding. Because of this, people getting a SCT typically need to stay in the hospital until their blood cell counts have recovered.

Another possible serious side effect from allogeneic transplants is graft-versus-host disease (GVHD). This occurs when the new immune cells (from the donor) see the patient’s tissues as foreign and attack them. GVHD can affect any part of the body and can be life-threatening.

While allogeneic SCT offers the best chance to cure some people with MDS, not everyone who gets a transplant is cured, and some people may die from complications of this treatment. Because of this, doctors often don’t recommend stem cell transplants for lower-risk MDS, as long as other treatments are still likely to be helpful.