Allogeneic CD56+ cell-based immunotherapy in a patient with Fanconi anemia developing acute myeloid leukemia

Fanconi anemia (FA) is a rare inherited disorder characterized by genomic instability, bone marrow failure, and a markedly increased risk of developing acute myeloid leukemia (AML). The intrinsic hypersensitivity of FA cells to DNA-damaging agents renders conventional chemotherapy particularly toxic and often ineffective. A 31-year-old woman with FA who progressed to AML and failed to achieve remission with standard induction therapy. Bone marrow blasts were initially 10%. As part of a clinical trial, she received CD56+ cell-based immunotherapy after FLAG chemotherapy. She subsequently underwent allogeneic hematopoietic stem cell transplantation (HSCT) from a matched sibling donor using a CD3+/CD19-depleted graft. Post-transplant, she received additional infusions of CD56+ cells. CD56+ immunotherapy reduced bone marrow blasts from 10 to 3%, enabling successful HSCT. Post-transplant, she remained in complete remission with no detectable minimal residual disease (MRD) at both day +30 and day +90. CD56+ cell-based immunotherapy can effectively decrease the leukemic burden in FA patients with AML, facilitating successful HSCT. This innovative approach may enhance outcomes for high-risk patients and merits further research.