Bone marrow

Bone Marrow Failure

Bone marrow is the spongy red tissue found inside bones; it produces red blood cells (RBC), white blood cells (WBC) and platelets. Red blood cells carry oxygen to the organs and tissues of the body, white blood cells help fight infection by attacking and killing germs, and platelets stop bleeding. Bone marrow failure occurs when the bone marrow does not produce these essential blood cells.

These rare disorders can be inherited (something your child is born with) or acquired (something that happened after birth). Inherited bone marrow failure is most commonly seen in young children. Acquired bone marrow is most commonly seen in young adults. Both types may lead to anemia (low RBC count).

No known cause

The cause of most bone marrow failure is unknown. However, inherited bone marrow failure is caused by a defect in the child's genes. Genes are the little pieces of information that tell the body what to do or what to make, from the colour of our eyes to how tall we are. Those children with inherited bone marrow failure still face the challenge of locating their defective gene before they can begin to search for a cure.

The research group at the Department of Haematology in the Hanson Institute have been studying blood formation for many years and are internationally recognized for their work in this field. With help from other research groups studying these rare bone marrow failures and with our support the researchers in this group will investigate the nature of the deficiency in blood cell production in bone marrow failure, focusing on why critical blood-producing cells that reside in bone marrow are unable to make the large numbers of blood cells required for a healthy blood and immune system.  This group will also use recombinant DNA technology to search for genes and proteins that can restore the blood producing capacity of disease cells, thus providing new clues into how blood cell production is controlled and possibly opening up new avenues for therapy.

Treatment

Treatment for bone marrow failure is different from case to case and depends on the cause and severity of the disease. Diagnosing and treating bone marrow failure as soon as possible may decrease and relieve symptoms, and prevent further medical problems.

For ongoing treatment a child may need any of the following:

Blood transfusions:

A blood transfusion is when donated blood is fed into the body through an intravenous line. This is donated whole blood, or parts of blood. Blood banks test all donated blood for AIDS, hepatitis, and other potential infectious agents. If you refuse a blood transfusion for your child, their condition may deteriorate and in extreme cases may lead to death. Multiple transfusions lead to iron poisoning and therefore your child will need to take a iron-chelator medication to prevent the accumulation of iron.

Medicines:

Hematopoietic growth factors
These medicines are used to help a child's bone marrow produce more blood cells. They may include androgens, erythropoietin, G-CSF, and GM-CSF.

Immunosuppressives
Immunosuppressive medicines help prevent the body from attacking its own bone marrow. This may allow the marrow to make more blood cells.

Steroids
Steroids may be given to help prevent the body from attacking its own bone marrow. This may allow the marrow to make more blood cells.

Stem Cell Transplants
Stem cell transplants (“SCT”) are also known as bone marrow or cord blood or peripheral blood stem cell transplantation (depending on the donor source). A SCT involves replacing a child’s unhealthy bone marrow with healthy cells from a suitable donor. The donor’s stem cells can be obtained from bone marrow, peripheral blood, or cord blood. This is a complicated medical procedure that requires several months in the hospital and is not without risk. The reward for a successful bone marrow transplant is that the child’s bone marrow will function normally and the child will not need repeated blood transfusion therapy or corticosteroid medication.