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What is severe aplastic anemia (SAA)?

SAA is a bone marrow disease. The bone marrow is the soft, spongy tissue inside bones. SAA causes the bone marrow to not make enough blood cells for the body. These blood cells are:

  • Red blood cells to carry oxygen
  • White blood cells to fight infection
  • Platelets to control bleeding

Aplastic anemia can range from mild to severe. Transplant is used for severe cases.

How does blood or marrow transplant (BMT) work for SAA?

BMT, also known as a bone marrow transplant or blood stem cell transplant, replaces the unhealthy bone marrow with healthy blood-forming cells (stem cells) from a donor. Transplant is the only potential cure for SAA.

An allogeneic transplant is used for SAA. This type of transplant uses healthy blood-forming cells donated by someone else. These healthy cells can come from a family member, unrelated donor or umbilical cord blood. First, you get chemotherapy (chemo), with or without radiation, to kill the unhealthy marrow. Then, the healthy cells are given to you through an intravenous (IV) catheter. The new cells travel to the inside of your bones and begin to make healthy blood cells.

The entire transplant process, from when you 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. Your transplant team closely watch you to prevent and treat any side effects or complications.

When to see a transplant doctor

If you’re diagnosed with SAA, you should see a transplant doctor right away. Young patients who have a closely matched donor and get a transplant soon after diagnosis may have a better chance for a cure. Older patients without a closely matched donor may do better with medicines that hold back the immune system (immunosuppressant medicines).

Even if you don’t need a transplant right away, it’s important to see a transplant doctor early. Patients with SAA typically get blood transfusions, but that may also lower the chance that a later transplant will work. For this reason, doctors recommend that patients make an appointment with a transplant doctor as soon as they’re diagnosed.

Your first appointment with a transplant doctor

At your first appointment, the transplant doctor will:

  • Review your medical history.
  • Talk with you about your treatment options.
  • Discuss the risks and benefits of transplant.
  • Recommend the best time for you to get a transplant and prepare for treatment
  • Start a donor search even if you don’t need a transplant right away. This can help you get a transplant faster if it’s needed later.

Questions to ask your doctor

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

  • What are the chances of cure 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?

Most recent medical review completed March 2017.