Aplastic Anemia

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What is aplastic anemia?

In aplastic anemia, the bone marrow shuts down and stops making new blood cells. 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 blood cells, children are at risk for serious health problems. Severe cases can be life-threatening. Aplastic anemia is a type of bone marrow failure disorder.

  • What causes aplastic anemia?

    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 parent passes down a condition that causes bone marrow failure.

 

Cancer and Blood Disorder Care at Seattle Children's

Our team treats children, teens and young adults, including those with the hardest-to-treat cancers and blood disorders. We provide treatment options that many centers do not offer.

 

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.
  • Be tired, dizzy or short of breath. Without enough red blood cells (anemia), your child may have low energy or these other symptoms.
  • Bruise or bleed easily because of low platelet levels.

Some people with aplastic anemia do not 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 is called bone marrow aspiration or biopsy. This test helps us understand the reason for your child’s marrow failure and rule out other reasons for low blood counts.
  • Check for other causes of low blood counts besides bone marrow failure. These include lack of nutrients, use of some medicines and infection from a virus.
  • Screen for conditions passed down from parent to child (inherited) that can cause bone marrow failure. Your child’s doctor will ask about other family members and may perform tests. Although inherited conditions are rarely the cause, it is important to check because they may need 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. Our treatment goals are to achieve the best possible outcome for your child and lessen the effects of this illness on their life.

At Seattle Children’s we offer these treatment options:

  • Blood transfusions

    Giving your child red blood cells and platelets from a healthy donor can help with anemia and bleeding problems. This is called a blood transfusion.

    How often children need a transfusion varies.

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

  • Antibiotics to fight infections

    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. Your child may receive antibiotics through a vein (IV) or take them by mouth.

  • Immunosuppression medicines

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

  • Growth factors

    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 granulocyte colony–stimulating factor (G-CSF). It is usually 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.

  • Stem cell transplant

    Some children may be cured of aplastic anemia by a transplant of blood-forming stem cells from a healthy donor. This is called a stem cell transplant or bone marrow transplant or hematopoietic (him-at-oh-poy-EH-tik) cell transplant.

    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 bone marrow failure are too sick to withstand the powerful drugs or radiation (called conditioning) that is usually used to prepare their bodies for the transplant. Our team has developed better ways to prepare them, called reduced-intensity conditioning. We keep improving our conditioning treatments to increase survival and reduce complications. We perform the transplants here at Seattle Children’s, working closely with our partner, Fred Hutchinson Cancer Center.

  • Prevention of infection and bleeding

    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.

  • Long-term monitoring

    Children with aplastic anemia need regular check-ups with a doctor who specializes in bone 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.

Aplastic Anemia at Seattle Children’s

At Seattle Children’s your child will get care from a blood specialist (hematologist) experienced in treating children with marrow failure. Our Bone Marrow Failure Program brings together many different types of healthcare providers to care for your child.

Our experience helps us know what to watch for and how to prevent and treat problems early. Early treatment 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. If you would like an appointment, ask your child’s primary care provider to refer you. If you have a referral or would like a second opinion, contact the center at 206-987-2106.

Providers, see how to refer a patient.

  • The experts you need for complete care

    Our physician-scientists are nationally known for diagnosing and 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, blood transfusions or a stem cell transplant.

    Our genetic counselor can talk with you about the pros and cons of genetic testing and explain test results. Although rare, it is important to check for inherited conditions that cause bone marrow failure.

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

  • Among the nation’s best children’s hospitals

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

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

    The doctors who will 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. We plan your child’s treatment based on years of experience plus the newest research on what works best — and most safely — for children.

  • Better outcomes from stem cell transplants

    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 perform the transplants here at Seattle Children’s, working closely with our partner, Fred Hutchinson Cancer Center. Fred Hutch pioneered stem cell transplants and is one of the largest stem cell transplant centers in the world.

  • National leaders in research to improve care and outcomes

    We are active in 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.

    As research leaders, we can offer our patients promising new therapies being studied in clinical trials. Your child’s doctor will talk with you about any new treatment options that might help your child. Then you can decide whether you want to take part.

    For children who might need stem cell transplants, our studies include:

    • Using 2 chemotherapy drugs to get the body ready to accept stem cells.
    • For children who do not have a sibling donor, studying if children who receive stem cells from an unrelated donor do better than children who get medicine to suppress their immune system. Siblings are more likely to have cells that are a close match, but good matches also can be found with nonfamily members who donate stem cells. The transplant has a better chance of success if there is a close match between the cells of the donor and the child getting the transplant.
    • Helping avoid graft-versus-host disease (GVHD) after transplant by controlling T cells from donors so they do not attack the cells of transplant patients.
  • Support for your whole family

    Having a child with aplastic anemia can be stressful for the whole family. We help take positive steps right away by offering appointments within 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. They can 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.

Contact Us 

If you would like an appointment, ask your child’s primary care provider to refer you.

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

Providers, see how to refer a patient.