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​Bone Marrow Failure (Pediatric)

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What is bone marrow failure?

The bone marrow is the soft, spongy substance located within the central core of the body’s larger bones where blood cells are made. The bone marrow is the body's "blood factory," and when not functioning normally, bone marrow failure occurs and insufficient blood cells are created. This condition results in inadequate red blood cells, white blood cells, and platelets for blood to perform its respective normal functions such as carry oxygen, fight infections, and assist with blood clotting and wound healing. The failure may involve all three cell lines or be isolated to only one type of blood cell.

What causes bone marrow failure?

When bone marrow failure happens, there are multiple mechanisms involved at the cellular level, such as 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 syndromes may be either hereditary (passed down through families) or acquired (brought on by illness or exposure to toxins).

Inherited conditions that may cause bone marrow failure from birth are:

  • Blackfan Diamond Syndrome
  • Fanconi’s Syndrome
  • Schwachman Diamond Syndrome

Acquired conditions occur later; the most common cause is aplastic anemia. Conditions  associated with acquired bone marrow failure are:

  • Exposure to chemicals such as benzene and insecticides
  • Radiation and chemotherapy
  • Infections such as hepatitis
  • Epstein Barr Virus

What are the symptoms of bone marrow failure?

  • Bleeding 
  • Tiredness 
  • Shortness of breath
  • Frequent infections and fever

How is bone marrow failure diagnosed?

  • Complete blood count 
  • Bone marrow aspiration and biopsy 
  • Specialized genetic testing and evaluation of DNA

What is the treatment for bone marrow failure?

  • Transfusions of red blood cells and platelets
  • Management of underlying conditions causing the problem
  • Chemotherapy
  • Immunotherapy
  • Hormonal therapy
  • Stem cell transplant