When would you consider transplant for a patient with a bone marrow failure syndrome (FA, DC, etc.)?
Answer from: at Academic Institution
At the time of early bone marrow failure - before they become transfusion-dependent or develop a significant risk for infection and/or bleeding.
With Fanconi's and BRCA2 mutation - would follow bone marrow carefully (2x a year) as they have a risk of AML.
Because both FA and DK are systemic disord...
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Answer from: at Academic Institution
I agree with Dr. Horn.
The field is moving towards earlier transplants for FA (less toxic transplant regimens, better outcomes, anabolic steroids are not good), and no transplants at all for DC (vascular problems after HSCT, higher risk of tumors, mucosal fragility with GIT bleeding).