Aplastic Anemia

What is aplastic anemia?

In aplastic anemia, the bone marrow shuts down and stops making new blood cells. It is a type of marrow failure disorder.

Bone marrow is found in the soft, spongy center of most bones. Healthy bone marrow makes young cells called stem cells. The stem cells, in turn, make the 3 major types of blood cells: 

  • White blood cells: fight infection
  • Red blood cells: carry oxygen
  • Platelets: make the blood clot and stop bleeding 

Without enough of these blood cells, children are at risk for serious health problems. Severe cases can be life threatening.

Children with aplastic anemia need care from a blood specialist (hematologist) with experience in marrow failure conditions. Our Bone Marrow Failure Program brings together many different types of healthcare providers to care for your child.

  • In most children with aplastic anemia, the condition develops after birth. Doctors think it happens when a child’s immune system stops their bone marrow from making new blood cells. Doctors do not understand what causes this.

    Rarely, a child inherits a condition that causes bone marrow failure.

Aplastic Anemia at Seattle Children’s

Experts in our Bone Marrow Failure Program have lots of experience caring for children and teens with aplastic anemia.

Our experience helps us know what to watch for and how to prevent and treat problems early. This increases the chances of successful treatment and helps your child feel better.

The Bone Marrow Failure Program is part of our Cancer and Blood Disorders Center. Please contact the center at 206-987-2106 for more information, a second opinion or to make an appointment.

  • For the 27th year in a row, Seattle Children’s ranks among the nation's best children's hospitals, according to U.S. News & World Report. Our physician-scientists are nationally known for treating children with marrow failure. They help set national standards for care of young people with blood disorders.

    We offer a full range of treatments for children with aplastic anemia. Your child may need medicines, growth factor, blood transfusions or a stem cell transplant.

    We care for your whole child. We don’t just treat their disease. Your family has a full team behind you, including specialists in nutrition, pain management, social work, physical therapy, psychology and emotional health. Read more about the supportive care we offer.

  • Aplastic anemia can sometimes be cured by a stem cell transplant, using young blood-forming cells from a healthy donor. Our Non-Malignant Transplant Program specializes in stem cell transplants for children with noncancer conditions.

    Our transplant team is very experienced in preparing children for transplant and helping them recover. For details on the number of stem cell transplants we do each year and survival rates for children who receive them, see statistics and outcomes.

    We work closely with Fred Hutch, our partner in the Seattle Cancer Care Alliance, to perform the transplants. More than 30 years ago, Fred Hutch pioneered stem cell transplants to treat blood disorders.

  • Our patients have access to promising new therapies offered only in research studies, called clinical trials. Examples include: 

    • A new conditioning preparation using 2 chemotherapy drugs (treosulfan and fludarabine phosphate) to get the body ready to accept stem cells. Dr. Lauri Burroughs leads the study. Read how results so far show much better survival.
    • Approaches that help children avoid graft-versus-host disease (GVHD) after transplant. Dr. Marie Bleakley work on ways to control T cells from donors so they don’t attack the cells of transplant patients. 

    We belong to national and international research groups that work to understand causes, improve care and find cures for blood disorders. One such group is the North American Pediatric Aplastic Anemia Consortium (NAPAAC). It supports patients and families with bone marrow failure.

    Learn more about clinical trials at Seattle Children’s 

  • Our specialty is treating children’s conditions while helping them grow up to be healthy and productive.

    Children do not react to illness, injury, pain and medicine in the same way as adults. They need – and deserve – care designed just for them.

    Our doctors have special training in how to diagnose and care for children with blood conditions. The doctors who guide your child’s care are board certified in pediatric hematology. This means they are approved to give the special care your child needs, and they constantly expand their knowledge about blood disorders.

    Our experts base their treatment plans on years of experience and the newest research on what works best – and most safely – for children.

  • A diagnosis of aplastic anemia can be stressful. We help take positive steps right away by offering appointments within 1 to 3 days to new patients with urgent needs. If needs are not urgent, new patients can be seen within weeks.

    During visits, we take time to explain your child’s condition. We help you fully understand your treatment options and make the choices that are right for your family.

    Our child life specialists and social workers help your child and your family through the challenges of this condition. We connect you to community resources and support groups.

    At Seattle Children’s, we work with many children and families from around the Northwest and beyond. Whether you live nearby or far away, we can help with financial counseling, schooling, housing, transportation, interpreter services and spiritual care. Read about our services for patients and families.

Symptoms of Aplastic Anemia

Aplastic anemia can cause low levels of white blood cells, red blood cells or platelets. Most children with this disease have low levels of all 3 types of cells.

Your child’s symptoms will depend on which cells are low and how low they are. Your child may: 

  • Have more infections and not get over them as quickly as other children. Low levels of white blood cells increase the risk of infection. Fever is a likely sign of infection.
  • Be tired, have low energy, be dizzy or be short of breath. Not having enough red blood cells (anemia) can cause these symptoms. Red blood cells carry oxygen to all the organs and tissues.
  • Bruise or bleed easily because of low platelet levels. Platelets help blood clot and stop bleeding. 

Some people with aplastic anemia don’t notice any symptoms. They may learn about a problem after a routine blood test is done for another reason.

Diagnosing Aplastic Anemia

To diagnose aplastic anemia, doctors: 

  • Take a blood sample to check the levels of each type of blood cell. This test is called a complete blood count, or CBC.
  • Get a sample of bone marrow. This helps us understand the reason for your child’s marrow failure and rules out other reasons for low blood counts.
    • Your child will get medicine to make them sleep so they do not feel pain (anesthesia) during the procedure.
    • Your child’s doctor will place a hollow needle into the hip bone and suck out (aspirate) a small sample of liquid bone marrow.
    • Then the doctor pushes a larger needle into the bone to remove a small amount of bone containing marrow.
    • These samples are checked under a microscope to look for anything that’s not normal. The doctor may order special studies to look for cell damage.
  • Check for other causes of low blood counts besides bone marrow failure. These include lack of nutrients, use of some medicines and infection with a virus.
  • Screen for inherited (passed down from parent to child) conditions that can cause bone marrow failure. Your doctor will ask questions about other family members and may perform tests. Although inherited conditions are rarely the cause, it is important to check because they may require different treatment.

Treating Aplastic Anemia

Your child’s care plan depends on their illness. We watch your child closely and recommend the right treatments at the right time. At Seattle Children’s we offer these treatment options:

  • Giving your child red blood cells and platelets from a healthy donor can help with anemia and bleeding problems. Your child receives the cells into their vein using an intravenous (IV) line. This is called a blood transfusion.

    How often children need a transfusion varies.

    Our outpatient infusion unit is staffed by expert nurses and has weekend hours. This helps your child get care without having to spend a night in the hospital.

  • If your child has a fever and a low level of white blood cells (neutropenia), they likely have trouble fighting off infections.

    They may need antibiotics to help fight infections caused by bacteria. Antibiotics may be put into your child’s vein using an IV line. Some children take them by mouth.

  • Medicines that make the immune system less active may help the bone marrow make blood cells again.

  • Children with low levels of blood cells may benefit from medicine that helps their bone marrow make more blood cells.

    The medicine is called hematopoietic growth factor, filgrastim or G-CSF. Usually it is given as a shot under the skin.

    There are benefits and risks to this type of medicine. Your child’s doctor will talk with you about whether this treatment is right for your child.

  • Some children may be cured of aplastic anemia by a transplant of blood-forming stem cells from a healthy donor (stem cell transplant). It is also called a bone marrow transplant or hematopoietic cell transplant. Hematopoietic (him-at-oh-poy-EH-tik) stem cells are immature cells that grow into blood cells.

    This treatment helps your child’s bone marrow make healthy blood cells. With normal levels of healthy blood cells, a child is no longer at risk for severe infections or bleeding.

    Our Non-Malignant Transplant Program specializes in stem cell transplants for children with noncancer conditions. Some children with marrow failure are too sick to tolerate the powerful drugs or radiation (called conditioning) that is usually used to prepare their bodies for the transplant. Our team – led by Dr. Lauri Burroughs – has developed better ways to prepare them, called reduced-intensity conditioning. We continue to fine-tune conditioning treatments to improve survival and reduce complications.

    We perform the transplants here at Seattle Children’s, working closely with Fred Hutch, our partner in the Seattle Cancer Care Alliance. Fred Hutch pioneered this lifesaving procedure and is one of the largest stem cell transplant centers in the world.

  • Your child’s doctor may suggest ways to help avoid infection or bleeding problems, such as: 

    • Limiting contact with other people, even healthy people
    • Avoiding activities with a high risk of injury, such as contact sports 

    Your doctor will let you know if your child needs to limit their activities.

  • Children with aplastic anemia need regular check-ups with a doctor who specializes in marrow failure syndromes.

    Checking blood cell counts and bone marrow regularly can help find problems early. That gives your child the best chance of successful treatment.

Contact Us 

If you would like an appointment, ask your child’s primary care provider for a referral.

If you have a referral or would like a second opinion, call the Cancer and Blood Disorders Center at 206-987-2106.

Providers, see how to refer a patient.