Other diseases treatable by bone marrow transplant

BMT, also known as a bone marrow transplant or blood stem cell transplant, can cure or treat more than 75 diseases. It replaces the unhealthy blood-forming cells (stem cells) with healthy ones.

A transplant may be a treatment option for:

• Diamond-Blackfan anemia
• Essential thrombocytosis
• Ewing sarcoma
• Fanconi anemia
• Germ cell ovarian cancer
• Hemophagocytic lymphohistiocytosis (HLH)
• Juvenile myelomonocytic leukemia (JMML)
• Medulloblastoma
• Myelofibrosis
• Neuroblastoma
• Polycythemia vera
• Testicular cancer
• Thalassemia

Other diseases not listed here may also be treated with transplant.

There are two types of BMT:

• Autologous transplant, which uses the patient’s own blood-forming cells. The cells are collected, stored, and given back after chemotherapy (chemo) or radiation.
• Allogeneic transplant, which uses healthy blood-forming cells from a family member, unrelated donor or umbilical cord blood.

For both types of transplant, you get chemo, with or without radiation, to kill the unhealthy cells. Then, the replacement cells are given to you through an intravenous (IV) line. The cells travel to the inside of your bones to make healthy blood cells.

The entire transplant process—from the start of chemo or radiation until hospital discharge—can last weeks to months. This is followed by many months of recovery near the transplant center and at home. The transplant team will closely care for you to prevent and treat any complications.

If you can’t find the disease you or your child has listed here, ask your doctor if transplant might be an option. You can also contact our Patient Support Center for help finding out if transplant might be an option.

At your first appointment, the transplant doctor will:

• Review your or your child’s medical history
• Talk with you about treatment options
• Discuss the risks and benefits of transplant
• Recommend the best time for you or your child to get a transplant and prepare for treatment
• Start a donor search

Ask questions so you understand your treatment options and can make decisions that are best for you or your child. Questions you may want to ask include:

• What are my chances of a cure or long-term remission with a transplant? Without a transplant?
• What are the risks of waiting or trying other treatments before a transplant?
• Do I or my child have any risk factors that might affect transplant outcomes?
• What are the possible side effects of transplant? How can they be reduced?
• How might my or my child’s quality of life change over time, with or without transplant?

Learn more about planning for transplant