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Bone marrow failure

Each year, approximately 25-40 children in the Netherlands are diagnosed with a severe form of bone marrow failure, such as myelodysplastic syndrome, juvenile myelomonocytic leukemia, Fanconi anemia and aplastic anemia. Congenital bone marrow failure is diagnosed in some instances immediately after birth, but is often not determined until some time later.  Acquired bone marrow failure can develop at any age.

The brochure Bone marrow failure and myelodysplastic syndrome contains comprehensive information about this disease. Please note this brochure is currently only available in Dutch. The most important information is provided below.

Cause

Bone marrow failure is often caused by an error in one of the immature blood cells. Something in the DNA or the chromosomes has been damaged in the nucleus of the cell and that damage cannot always be identified. The cause of the damage is often unknown. Sometimes the bone marrow failure is already present at birth (congenital), sometimes it develops later in life (acquired). Sometimes bone marrow failure is hereditary.

Symptoms

The symptoms of bone marrow failure depend on the cell line (the type of cells) that fails. If your child has a shortage of red blood cells (anaemia), your child is pale and tired, and can also suffer from dizziness and shortness of breath. A lack of healthy white blood cells can cause recurring or severe infections and fever. Nosebleeds, easy bruising, pinpoint-sized purple-red spots, and sores that keep bleeding are due to a shortage of platelets. Your child may also have a fever, swollen lymph nodes, bone or abdominal pain, or an enlarged spleen. A mix of the aforementioned symptoms occurs when all cell lines fail.

How is bone marrow failure diagnosed?

Blood tests are done and bone marrow is extracted under sedation. A chromosome breakage test is performed as well in some instances.


Treatment

Children with bone marrow failure are treated according to a protocol drawn up by national and international experts. Treatment depends on the cause and severity of the disease and consists of medication, blood transfusions and/or an allogeneic stem cell transplant. The latter is only possible if a suitable donor is found.


Chance of recovery

The chance of recovery depends greatly on any complications during treatment.


Questions

Children with bone marrow failure are treated in the hematology oncology ward.