Aplastic anemia

We understand that you may have a lot of questions if your child has been diagnosed with aplastic anemia.

• What exactly is it?
• What are potential complications in my child’s case?
• What are the treatments?
• What are possible side effects from treatment?
• How will it affect my child long term?

We’ve tried to provide some answers to those questions here, and when you meet with our experts, we can explain your child’s condition and treatment options fully.

What is aplastic anemia? 

Aplastic anemia is a blood disorder that occurs when the bone marrow produces too few of all types of blood cells: red cells, white and platelets.

• A low number of red blood cells reduces the blood’s ability to carry oxygen.
• A reduced number of white blood cells makes the child more susceptible to infection.
• A low number of platelets reduces the blood’s ability to clot.

Causes

What causes aplastic anemia?

Aplastic anemia in children has multiple causes, some of which are “idiopathic,” or occurring with no known reason. The disorder can be the result of a previous illness or existing problem, or be caused by an inherited genetic disorder.

Acquired causes of aplastic anemia in children make up about 80 to 85 percent of cases and may include a history of:

• specific infectious diseases, such as hepatitis, Epstein-Barr virus, or cytomegalovirus
• taking certain medications including some antibiotics and arthritis drugs
• exposure to certain toxins, such as benzene, pesticides and insecticides
• exposure to radiation or chemotherapy

In much fewer—about 15 to 20 percent of the time—children inherit a disorder that predisposes them to developing aplastic anemia, such as: 

• Fanconi anemia
• Dyskeratosis congenita
• Shwachman-Diamond syndrome
• Amegakaryocytic thrombocytopenia
• Myelodysplastic syndrome

Symptoms

What are the symptoms of aplastic anemia?

The most common symptoms of aplastic anemia are:

• lack of energy or tiring easily 
• pale skin, lips, and hands, or paleness under the eyelids
• shortness of breath
• fevers or infections
• bleeding, such as bruising, bleeding gums, nosebleeds or blood in the stool
• irregular heartbeat
• dizziness or headache

It is important to understand that the symptoms of aplastic anemia may resemble other blood disorders or medical problems, some of which are very common and easy to treat, while others are more serious. The symptoms listed above are common presentations of the disease, but do not include all possible symptoms.

Your child may experience symptoms differently. Therefore, it is important to be evaluated by a physician to obtain an accurate diagnosis. Always consult your child's physician if you have concerns.

Questions to ask your child’s doctor

You and your family are key players in your child’s medical care. It’s important that you share your observations and ideas with your child’s health care provider and that you understand your doctor’s recommendations.

If your child is suffering from aplastic anemia, you probably have a lot on your mind. So it’s often helpful to write questions down. Some of the questions you may want to ask include:

• What does a diagnosis of aplastic anemia mean for my child?
• How will my child’s symptoms be managed?
• Can my child receive a stem cell transplant?
• How long will my child need to be in the hospital?
• What are the possible short and long-term complications of treatment? How will they be addressed?
• What is the likelihood of cure?
• What services are available to help my child and my family cope?

FAQ

Q: What is the expected outcome after treatment for aplastic anemia?
A: Aplastic anemia was once fatal in many cases. Today, treatment has improved significantly due to innovative new therapies including hematopoietic stem cell transplantation and effective supportive care. However, aplastic anemia is still a very serious condition requiring extensive ongoing care. Your child’s physician is available to answer questions about treatment options and outcomes.

Q: Is there a cure for aplastic anemia?
A: Hematopoietic stem cell transplant can cure aplastic anemia when blood stem cells come from a matched sibling donor. However, a matched sibling donor may not be available in all cases. Stem cells from a matched unrelated donor, cord blood or family member may also be considered under certain circumstances, such as when drug therapy is ineffective.

Patients with aplastic anemia also have a higher risk of developing complications from stem cell transplantation (including graft-versus-host disease). Your child’s physician can tell you whether a stem cell transplant might be appropriate for your child.

Q: Where will my child be treated?
A: Children treated through the Fanconi Anemia and Bone Marrow Failure Multidisciplinary Clinic will receive outpatient care at Children’s Hospital Boston. If your child receives a stem cell transplant, he or she will see doctors at the Jimmy Fund Clinic at the Dana Farber Cancer Institute and will stay in  a specialized stem cell transplant unit at Children’s Hospital.

Q: What services are available to help my child and my family cope?
A: We offer a many of services to help you, your child and your family get through this difficult time. Read more about our support services.