This therapy is very effective, and although there are associated toxicities, there has been improvement in the management of toxicities for Fanconi patients over the past few years. It is typically given with low dose prednisone. However, patients rapidly relapse when the drug is stopped and nearly all eventually become refractory to therapy. Preclinical and very early clinical studies suggest that there could be a role for gene therapy in the future. If a neutropenic fever occurs, hospitalization with intravenous antibiotics is likely.
Most patients visit a hematologist, an endocrinologist and an ophthalmologist regularly.
An orthopedist, gynecologist or nephrologist may be seen as needed. It was previously thought that the median survival of patients with Fanconi's anemia was 25 years, although a wide range was observed. Survival is likely significantly improved with the improvement of current therapies in recent years. Bone marrow transplant can completely cure these patients of the primary disease. A physician should closely follow women with Fanconi's anemia who become pregnant.