Venetoclax and Azacitidine in the Treatment of NPM1-Mutated Donor Cell–Derived Leukemia in a Patient With Fanconi Anemia: Case Report and Literature Review

Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) constitutes curative therapy for BM failure,MDS, andAML inFA, but importantly, thenonhematopoietic cells remain unable to repair DNAdamage post-transplant. Typical intensive AMLchemotherapy regimens carry an unacceptably high risk of toxicity in patients with FA, including prolonged myelosuppression and mucositis. FA-associated MDS/AML is treated with reduced intensity conditioning (RIC) HSCT preceded by no or limited chemotherapy because of the extreme sensitivity of FA cells to alkylating agents and radiation, higher rates of graft-versus-host disease, and predisposition to solid malignancies. Of note, given the presence of the FA DDR deficiency in the MDS/AML cells, FA-associated MDS/AML is sensitive to low-intensity chemotherapy.

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