​Bone Marrow Failure (Pediatric)

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What is bone marrow failure?

Bone marrow, the spongy substance within the larger bones of the body, is where blood cells are made. Bone marrow failure occurs when the bone marrow does not create enough red blood cells, white blood cells, and platelets for blood to perform its normal functions to keep the body working properly.

In normal body function, red blood cells carry oxygen throughout the body’s tissue, white blood cells help fight infections, and platelets assist with blood clotting and wound healing. Blood marrow failure can affect one or all three of these important functions.

The signs and symptoms of bone marrow failure include excessive bleeding or bleeding that takes a long time to stop, excessive tiredness and shortness of breath, and frequent infections and fever.

When bone marrow failure happens, there are multiple mechanisms involved at the cellular level. This includes problems with the stem cells that create new blood cells and defects in the mechanisms that tell those cells how to grow and develop.

Bone marrow failure can either be passed down through families or can be brought on by illness or exposure to toxins. There are a number of inherited conditions that can cause bone marrow failure from birth. When bone marrow failure is acquired later, the most common cause is aplastic anemia, which may be associated with exposure to chemicals such as benzene and insecticides. Other factors that can cause bone marrow failure include radiation or chemotherapy treatments, immune system problems, infections, and viral illnesses such as hepatitis and Epstein Barr Virus.

Our approach to bone marrow failure

When bone marrow failure is suspected, your medical team will likely conduct a blood test with a complete blood count, to find out whether there are abnormalities in the number of blood cells. Your health care provider might also perform specialized tests to look at abnormalities in the blood cells themselves or in DNA.

In order to confirm the diagnosis, the health care provider must examine the bone marrow by performing a bone marrow aspiration and biopsy. In a bone marrow aspiration, a small amount of bone marrow is removed through a needle that goes into the bone. In a biopsy, a small part of bone is removed with the bone marrow inside to be looked at under a microscope.

Treatment of bone marrow failure is managed by a supportive care team and includes transfusions of red blood cells and platelets, management of underlying conditions that are causing the problem, chemotherapy (the use of medicines to attack the abnormal cells), immunotherapy (which uses medicines to stimulate the immune system to go after the abnormal cells), hormonal therapy, and, if needed, ultimately a stem cell transplant.