Treatment options for fanconi anemia are decided based on how well or how poorly the patient's bone marrow is producing new cells and his/her age.
Doctors decide how to treat Fanconi anemia (FA) based on a person's age and how well or how poorly the person's bone marrow is making new blood cells.
Goals of Treatment
Long-term treatments for FA can:
- Cure the anemia. Damaged bone marrow cells are replaced with healthy ones that can make enough of all three types of blood cells on their own.
- Treat the symptoms without curing the cause. This is done using medicines and other substances that can help your body make more blood cells for a limited time.
Observation and Short-Term Treatment
If you or your child has FA, but your bone marrow is still able to make enough new blood cells, your doctor may do frequent blood count checks.
During this time of observation, your doctor will probably want you to have bone marrow tests once a year. Your doctor also will screen you closely for any signs of cancer or tumors.
If your blood counts begin to drop sharply and stay low, your doctor may assume your bone marrow is failing. He or she may give you antibiotics to help your body fight infections.
In the short term, your doctor also may want to give you blood transfusions to increase your blood cell counts to normal levels. However, long-term use of blood transfusions can reduce the chances that other treatments, which can help your body make enough blood cells on its own, will work.
The four main types of long-term treatment for FA are:
- Blood and marrow stem cell transplant
- Androgen therapy
- Synthetic growth factors
- Gene therapy
Blood and Marrow Stem Cell Transplant
A blood and marrow stem cell transplant is the current standard treatment for patients who have FA that's causing major bone marrow failure. Healthy stem cells from another person, called a donor, are used to replace the faulty cells in your bone marrow.
If you're going to receive stem cells from another person, your doctor will want to find a donor whose stem cells match yours as closely as possible.
Stem cell transplant is most successful in younger people who:
- Have few or no serious health problems
- Receive stem cells from a brother or sister who is a good donor match
- Have had few or no previous blood transfusions
During the transplant, you'll get donated stem cells in a procedure that's like a blood transfusion. Once the new stem cells are in your body, they travel to your bone marrow and begin making new blood cells.
A successful stem cell transplant will allow your body to make enough of all three types of blood cells to work normally. However, even if you've had a stem cell transplant to treat FA, you'll still be at risk for some types of blood cancer and cancerous solid tumors. Your doctor will check your health regularly and often after the procedure.
Before improvements made stem cell transplants more effective, androgen therapy was the standard ...
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