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When would you consider transplant for a patient with a bone marrow failure syndrome (FA, DC, etc.)?

2 Answers
Mednet Member
Mednet MemberInvited Expert
Pediatric Hematology/Oncology · University of Florida
Answered on

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 disorder...

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Mednet Member
Mednet MemberInvited Expert
Pediatric Hematology/Oncology · St Jude Children's Research Hospital
Answered on

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).

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