Treatment of Fanconi Anemia with Advanced Myelodysplastic Syndrome or Acute Myeloid Leukemia

Fanconi anemia (FA) patients who develop advanced myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) present a distinct and challenging clinical scenario requiring careful treatment planning before transplantation.

This protocol specifically addresses Fanconi anemia with advanced myelodysplastic syndrome or acute myeloid leukemia — a high-risk progression in which the approach to pretransplant cytoreduction remains controversial and the choice of strategy is critical.

Treatment Approach (partial overview)

Management involves pretransplant cytoreduction before hematopoietic stem cell transplantation, with more than one chemotherapy-based strategy in use or under active evaluation — the complete regimen, sequencing, and decision criteria are available in the full protocol.

Instant Access to Structured Evidence-Based Regimens

References

Pretransplant cytoreduction in patients with FA and advanced MDS/AML remains controversial.

Sequential strategies of fludarabine/cytarabine-based chemotherapy followed by HSCT several weeks later regardless of aplasia status may improve relapse-free survival.

Combination azacytidine/venetoclax as pretransplant cytoreduction is currently being tested in a combined safety/efficacy basket trial that includes patients with FA and MDS/AML (NCT05292664).

DOI: 10.1182/hematology.2023000489

View source ↗