The Bone Marrow Failure Program
The Bone Marrow Failure Program at the Children’s Minnesota provides comprehensive multidisciplinary care for children with bone marrow failure syndromes. Our program is part of a large hematology program and offers evaluation, diagnosis and treatment for both inherited and acquired bone marrow failure, including these conditions:
- Aplastic anemia
- Fanconi anemia
- Shwachman-Diamond syndrome
- Diamond-Blackfan anemia
- Dyskeratosis congenital
- Severe congenital neutropenia (Kostmann’s syndrome)
- Cyclic neutropenia
- Congenital amegakaryocytic thrombocytopenia
- Thrombocytopenia with absent radii
- Sideroblastic anemia
- Paroxysmal nocturnal hemoglobinuria
Why choose Children’s?
- Experts work together for your child. Children’s Minnesota offers a full range of specialists to diagnose and treat these disorders. Our doctors have worked with many children with bone marrow failure syndromes and their families. Our comprehensive team will help you understand the disease, provide various treatment options and help you get support throughout the process.
- Access to the latest treatment options. Our Bone Marrow Failure Program participates in several national registries, such as the Severe Chronic Neutropenia International Registry (SCNIR) and the Diamond-Blackfan Anemia Registry, which collect data to help experts learn more about these rare syndromes and their treatment.
- Second opinions for children not seen in our program. At Children’s, we provide consultation services for children who are not seen in our program but would like input from our expert team. If you or your child’s doctor would like us to review your child’s case, you may arrange to have health records and test results for your child sent to us. The child does not need to live in the area or be seen in our clinic.
What is Bone Marrow Failure?
Bone marrow failure occurs when the bone marrow in the center of the bone does not make enough healthy blood cells for the body to function normally. Bone marrow failure can result from an inherited bone marrow failure syndrome or as a result of exposure to environmental toxins. Complications from bone marrow failure may be complex and life threatening requiring immediate care.
There are two main types of bone marrow failure, inherited, meaning there is a genetic component to the disease, or acquired, meaning the bone marrow failure developed without a genetic component. Bone marrow failure can affect one or more types of blood cells in the body, including white blood cells, red blood cells and platelets.
The symptoms a child develops depends on which cells of the bone marrow are affected by the failure of the bone marrow.
- Low levels of white blood cells result in increased chance for infections. The child may get sick more often or may take longer to get better than other children.
- Low levels of red blood cell affect how much oxygen the blood cells carry to the body and can lead to feeling tired, weak, dizzy, or pale. This can be a gradual change and may not lead to a visit to the doctor for weeks or months. Illnesses with low levels of red blood cells are called “anemias”.
- Low levels of platelets affect how the blood clots. The child may have increased bruising or bleeding in the skin, nose and mouth. Bleeding issues may be the reason for the first visit to the doctor.
A thorough physical exam, family history and lab tests are completed. Often a bone marrow test to look at how the blood cells are growing is required. Sometimes imaging studies to assess other organs may also be needed.
Treatment options are based on the symptoms of the child, physical exam, history and lab test results. Options may include blood transfusion, antibiotics, and other medications to increase blood cell production in the bone marrow. A bone marrow transplant may be required.