Treatment:

Patients with mild to moderate blood cell changes who do not need a transfusion may only need regular check-ups, frequent blood count checks, and yearly bone marrow exams. The health care provider will closely monitor the person for other cancers, usually leukemia or lymphoma.

Medicines called growth factors (such as erythropoietin, G-CSF, and GM-CSF) can improve blood counts for a short while.

A bone marrow transplant can cure the blood count problems of Fanconi's anemia. (The best donor is a brother or sister whose tissue type matches the patient.)

Persons who have had a successful bone marrow transplant still need regular check-ups because of the risk for additional cancers.

Hormone therapy combined with low doses of steroids (such as hydrocortisone or prednisone) is prescribed to those who do not have a bone marrow donor. Most patients respond to hormone therapy. But everyone with the disorder will quickly get worse when the drugs are stopped. In most cases, these drugs eventually stop working.

Additional treatments may include:

• Antibiotics (possibly given through a vein) to treat low white blood cell counts and infections
• Blood transfusions to treat symptoms due to low blood counts

Most people with this condition visit a blood disorder specialist (hematologist), a doctor who treats diseases related to glands (endocrinologist), and an eye doctor (ophthalmologist) regularly. They also may see a bone doctor (orthopedist), gynecologist, or kidney disease specialist (nephrologist).

Expectations (prognosis):

The survival rates vary from person to person. The outlook is poor in those with low blood counts. New and improved treatments, such as bone marrow transplants, have likely improved survival.

Persons with Fanconi's anemia are more likely to develop several types of cancers, including leukemia, myelodysplastic syndrome, and liver cancer.

Women with Fanconi's anemia who become pregnant should be watched carefully by a doctor. Such women often need transfusions throughout pregnancy.

Men with Fanconi's anemia have decreased fertility.