guidebook. - Fanconi Anemia Research Fund

Chapter 3: Treatment of Hematologic Abnormalities in FA
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• Hematopoietic stem cell transplant with or without
prior induction chemotherapy.
• Phase I/II trials for MDS or AML.
Published reports of chemotherapy regimens for AML
in FA patients are sparse and limited by the lack of
longitudinal follow-up. It remains unclear whether
pre-transplant chemotherapy improves or worsens outcomes.
These recommendations are summarized in Table 4.
Table 4: Marrow Failure Algorithm
Normal marrow/
Mild marrow failure
Monitor blood counts and
marrow as described in
Table 2
Moderate marrow
failure*
Matched sibling HSCT
Unrelated donor transplant
Androgens
Severe marrow
failure*
Unrelated donor transplant
Androgens, cytokines (G-CSF)
MDS or AML
HSCT +/- chemotherapy
Investigational trials for
MDS or AML
*These represent nodal points at which discussion of the risks and
benefits of the therapeutic options should be initiated. Refer to
text for full discussion.