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Allogeneic haematopoietic cell transplantation from alternative donors with a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia

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A pilot study was undertaken using a fludarabine-based conditioning regimen to improve haematopoietic cell transplantation (HCT) from alternative donors in 27 Fanconi anaemia (FA) patients. Patients were conditioned with 150–180 mg/m2 of fludarabine, 40 mg/kg of cyclophosphamide, 5–10 mg/kg of antithymocyte globulin, and 300–450 cGy of thoracoabdominal/total body irradiation. One patient who received unrelated cord blood transplantation failed to engraft, another patient died of sepsis. The 1-year overall survival was 96ยท3% (95% CI, 89–100). This conditioning regimen exerted an immunosuppressive effect that enabled durable engraftment in alternative donor HCT without severe toxicity.

Keywords: Fanconi anaemia; alternative donors; fludarabine; haematopoietic cell transplantation; tacrolimus

Document Type: Research Article


Affiliations: 1: Specialized Clinical Science, Pediatrics, Tokai University School of Medicine, Isehara 2: Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara 3: Department of Pediatrics, Nagoya University, Nagoya 4: Department of Pediatrics, Ibaragi Children's Hospital, Mito 5: Department of Pediatrics, Keio University, Tokyo 6: Department of Pediatrics, Kyoto University, Kyoto 7: Division of Cellular Therapy, Advanced Clinical Research Center, Institute of Medical Science, University of Tokyo, Tokyo 8: Department of Pediatrics, Shinshu University, Matsumoto 9: Department of Pediatrics, Kyoto Prefectural Medical University, Kyoto 10: Department of Pediatrics, Osaka City General Hospital, Osaka, Japan

Publication date: July 1, 2006


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