Cooley's Anemia

Mild Versus Severe Forms of Cooley's Anemia

People with thalassemia intermedia, also known as mild Cooley's anemia, may need occasional transfusions when they are experiencing stress due to an infection. Thalassemia intermedia becomes thalassemia major when the disease worsens and the patient needs regular transfusions.
 

What Is the Prognosis?

For a person living with Cooley's anemia, prognosis will depend on:
 
  • How severe the condition is
  • How soon treatment begins
  • How successful the treatment is.
 
People living with Cooley's anemia who are able to continue therapy successfully may live into their 30s or 40s.
 

Is There a Cure for Cooley's Anemia?

Cooley's anemia can be cured using bone marrow transplants. However, this treatment is only possible for a small minority of patients who have a suitable bone marrow donor. The transplant procedure itself is still risky and can result in death.
 

Cooley's Anemia and Gene Therapy

Thalassemia research scientists are working to develop a gene therapy that may offer a cure for Cooley's anemia. They are currently looking at two gene therapy treatments, which include:
 
  • Beta-globin gene and stem cells
  • Medications and fetal hemoglobin.
 
Beta-Globin Gene and Stem Cells
This treatment option, once fully developed, would involve inserting a normal beta-globin gene (the gene that is abnormal in this disease) into the patient's stem cells, which are the immature bone marrow cells that are the precursors of all other cells in the blood.

Thalassemia Anemia

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