Bone Marrow Transplant | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

Bone Marrow Transplant

Introductory image: Patient and Doctor Wearing Masks at Hospital

A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due to the risks and potential long-term side effects as an "imperfect cure".

What are the types of transplantation?

There are two basic types of bone marrow/stem cell transplants. A transplant may use cells taken from a donor or from the patient:

  • Autologous transplants use a patient's own blood-forming stem cells. These are collected from the patient's marrow or blood and frozen for later use. Autologous transplants are often not an option for patients with bone marrow failure diseases.
  • Allogeneic transplants use cells from a healthy donor. These might come from a sibling, or a matched unrelated donor (MUD). The donor's tissue type must be a suitable match with the patient's type. This is called HLA matching. A well-matched donor is important to the success of the transplant. To find a match, a patient can search the bone marrow donor registry managed by the National Marrow Donor Program.

How well do they work?

The chances the transplant will work are different for each patient. How well the transplant works depends on many things, such as:

  • The disease being treated
  • The stage of the disease
  • The patient's age and general health
  • How well the donor's tissue type matches the patient

What are the side effects?

Some patients suffer from life-threatening problems as a result of their transplant. Some people do die because of complications. These problems can include serious infections and graft-versus-host disease (GVHD), in which the transplanted cells attack the patient's body.

Is it right for you?

There are many risks and benefits of a transplant. You, your doctor and your family need to consider many things when making a decision about whether a BMT is right for you, including:

  • Your disease stage
  • Your age
  • Your overall health
  • Whether a matching donor is available
  • Other treatment options

IMPORTANT: If your doctor thinks you may be a candidate for a bone marrow or stem cell transplant, you should start looking for a donor now. First you will want to see if a sibling is a match. If not, you and your doctor can use the Be The Match Registry managed by the National Marrow Donor Program to search for a match. It can take a long time to find a matched donor.

How is Transplantation Used?

  • Aplastic anemia: Some doctors believe that patients with non-malignant disease (not cancer) such as aplastic anemia, should receive stem cells from bone marrow rather than stem cells that are harvested from peripheral blood. Research seems to show that using bone marrow stem cells can decrease the chance of graft-versus-host disease.
  • PNH: Due to current advances in the treatment of PNH, transplantation may not be recommended as an initial treatment. However, those PNH patients with severe bone marrow failure that does not respond to immunosuppressive therapy or those patients with low platelet counts while on eculizumab should talk to their doctor about bone marrow transplant options.
  • MDS: Whether patients with MDS should undergo hematopoietic stem cell transplantation (HSCT) as soon as their MDS is diagnosed or wait for months or even years depends on their IPSS score. Delaying SCT after diagnosis improves survival in patients with low-risk or intermediate-1 MDS. Survival in patients with intermediate-2 or high-risk MDS is best when they have the SCT right away. Also, survival after SCT is better when patients have a transplant before progression to AML.

Cytogenetics, or the study of abnormal chromosomes, can also affect SCT outcomes. People with secondary MDS, caused by a previous treatment for another disease or disorder, tend to have more abnormal chromosomes and worse outcomes after SCT than people with primary MDS.

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any drug, treatment or therapy listed on this website. Some therapies listed on our site are considered experimental for the treatment of bone marrow failure diseases. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding any therapy, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.