HLA-matched related-donor HSCT in Fanconi anemia patients conditioned with cyclophosphamide and fludarabine

Twenty years of the Italian Fanconi Anemia Registry: where we stand and what remains to be learned

“…Since the RIAF was not designed to study HSCT in the context of FA, the actual impact of HSCT on the natural history of FA needs to be investigated in more specific studies that also deal with all the transplant-specific factors affecting the outcome of HSCT. Indeed, the question is whether more recent HSCT, performed according to 34 have improved the outcome of FA, as compared with natural history. One may anticipate that combining earlier therapeutic intervention with improved HSCT protocols may lead in the near future to improved long-term outcomes for FA patients, 35,36 especially if a lack of an increased risk of malignancies is confirmed.…”

How I treat MDS and AML in Fanconi anemia

“…Regarding GVHD prophylaxis, the most commonly used combination is cyclosporine plus mycophenolate mofetil. 57,62,66 The role of pretransplant cytoreduction FA patients who develop MDS and/or leukemia are not easy to manage because sensitivity to DNA-damaging agents limits the therapy they can tolerate. 47 Chemotherapy in such patients is regularly associated with significant toxicity and a possible prolonged period of aplasia, with certain complications that may eventually contraindicate HSCT.…”

Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations

“…In order to reduce the risk of CY-related toxicities, Benajiba et al [38] explored the effectiveness of lower dose CY (40 mg/kg) in combination with FLU 90 mg/m 2 (with 6 also receiving ATG) in FA patients with BMF and a MSD. Neutrophil engraftment was achieved in 20 of 20 patients.…”