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If the hematological changes are mild to moderate and the patient does not require transfusions, a period of observation is currently recommended with frequent blood count checks and yearly bone marrow examinations. Observations for the development of secondary malignancies are also performed. In the short term, growth factors (such as erythropoetin, G-CSF, and GM-CSF) can be used to improve blood counts. Other growth factors for platelet stimulation are currently under investigation.
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Bone marrow transplantation can cure the blood count problems associated with Fanconi's anemia. A HLA matched sibling is the best donor source, although umbilical cord blood cells and unrelated bone marrow can also be used. This therapy is very effective, and although there are associated toxicities, there has been improvement in the care of Fanconi patients during the transplant. There is approximately a 70% success rate for those patients fortunate enough to have a donor.
Even though a successful bone marrow transplant can cure the bone marrow problems from Fanconi's anemia, patients are at risk for other cancers and must be regularly followed by a physician (see below).
Prior to bone marrow transplantation, androgen therapy (oxymetholone, nandrolone decanoate) combined with low doses of steroids (hydrocortisone, prednisone) was the standard treatment, and this approach is currently used if the patient does not have an appropriate bone marrow donor. Typically, 50-75% of patients initially respond to androgen therapy, however, all patients will rapidly relapse when the drug is stopped. In most cases, these drugs eventually become ineffective.
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