A bone marrow transplant (BMT) bone marrow transplant (BMT): A procedure where bone marrow stem cells are collected from marrow inside the donor's hipbone and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells. is also called a stem cell transplant (SCT) stem cell transplant (SCT): A procedure where blood-forming (hemapoietic) stem cells are taken from a healthy donor. They enter the patient's bloodstream through an intravenous (IV) needle and make their way into the bone marrow. The donor cells are called a graft. There are three sources of stem cells - bone marrow, cord… or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… 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 bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the 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 bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… 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 HLA: See human leukocyte antigen. 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) graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… , 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 aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… , 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 immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… or those patients with low platelet platelet: The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte. counts while on eculizumab eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … 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 Cytogenetics: (sie-toe-juh-NEH-tiks) The study of chromosomes (DNA), the part of the cell that contains genetic information. Some cytogenetic abnormalities are linked to different forms of myelodysplastic syndromes (MDS). , or the study of abnormal chromosomes chromosomes: The part of the cell that contains our DNA or genetic code. , can also affect SCT outcomes. People with secondary MDS secondary MDS: A type of MDS that is caused by a previous treatment foranother another disorder or disease. Treatments typically associated with secondary MDS include radiation therapy and chemotherapy used to treat cancer. Also called therapy-related MDS, T-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.