Severe aplastic anemia (SAA)

Severe aplastic anemia (SAA) is a bone marrow disease. Bone marrow is the soft, spongy tissue inside bones. SAA causes the bone marrow to not make enough blood cells for the body, including:

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

Aplastic anemia can range from mild to severe. For mild cases, you may experience symptoms like fatigue, dizziness, shortness of breath and headache, but may not require hospitalization. When symptoms become more serious and a severe case of aplastic anemia is developed, transplant is the only cure. 

No, aplastic anemia is not a type of cancer. It’s a rare blood disorder where the bone marrow fails to produce enough blood cells. However, having aplastic anemia can increase a person’s risk of developing certain cancers—most notably, leukemia—especially if the condition is severe or long-standing.

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

For SAA, doctors use an allogeneic transplant, meaning the donated cells come from someone else. These healthy blood-forming cells can come from:

• A family member
• An unrelated donor
• Umbilical cord blood

Although each person’s journey is unique, bone marrow transplantation for severe aplastic anemia typically follows a series of distinct phases. From initial therapy to the infusion of donor cells and ongoing recovery, here’s how the transplant process generally works.

• Preparation: You’ll begin with chemotherapy (and sometimes radiation) to destroy the unhealthy marrow. This step, called conditioning therapy, makes room for new, healthy blood-forming cells.
• Transplant: Next, healthy donor cells are delivered through an intravenous (IV) catheter, often referred to as a stem cell infusion. These cells travel to the inside of your bones and begin producing new blood cells.
• Recovery: Full recovery can take several months to a year. From the start of conditioning to leaving the hospital can take weeks to months. You’ll then spend additional time recovering near the transplant center and at home. During this period, your transplant team carefully monitors you, helping prevent and manage any side effects or complications.

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 immunosuppressive medications, which help manage the body’s immune response.

Even if a transplant isn’t needed right away, scheduling an early appointment with a transplant doctor is still important. Many SAA patients depend on blood transfusions, but repeated transfusions can reduce the success rate of future transplants. For this reason, most doctors recommend meeting with a transplant specialist as soon as SAA is diagnosed.

Scheduling an appointment with a doctor to discuss your SAA diagnosis is the first step to finding a cure. At your first appointment, the transplant doctor will:

• Review your medical history.
• Talk with you about SAA treatment options.
• Discuss the risks and benefits of BMT 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.

Patients that come from ethnically diverse backgrounds will often have a harder time finding a matching bone marrow donor. This is because of genes called human leukocyte antigens (HLAs), which are the codes inside your body doctors use to help find a match. HLAs are inherited, which means it will be easier to find a match using a patient and donor that share the same ancestry.

Unfortunately, not all ethnicities are equally represented on the registry.

Ask questions so you can best understand the treatment options available for your severe aplastic anemia. Questions you may want to ask your doctor include:

• What are the chances of curing SAA with a transplant? Without a transplant?
• What are the risks of waiting or trying other SAA 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 a bone marrow or blood stem cell transplant? How can they be reduced?
• How might my (or my child’s) quality of life change over time due to SAA, with or without transplant?

By asking these questions, you can better understand your options and plan the next steps in your treatment journey.