Fanconi anemia, or FA, is a rare, inherited blood disorder that leads to bone marrow failure. A variety of supportive treatments is available but stem cell transplantation is the only curative option (gene transfer therapies are still experimental). The two year survival rate is around 70%. Alternative conditioning regimens using fludarabine and avoiding irradiation have shown promising results so far. For those patients without matched sibling stem cell source, the prognosis for unrelated donors is around 20–40% survival at two years and there is a higher risk of graft rejection in somatic mosaics.
Stem cell transplantation is generally accepted as medically appropriate.
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