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Bone marrow transplantation from matched related donors for patients with Fanconi anemia using low‐dose busulfan and cyclophosphamide as conditioning
Author(s) -
Torjemane L.,
Ladeb S.,
Ben Othman T.,
Abdelkefi A.,
Lakhal A.,
Ben Abdeladhim A.
Publication year - 2006
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.20286
Subject(s) - medicine , busulfan , cyclophosphamide , transplantation , surgery , regimen , gastroenterology , methotrexate , total body irradiation , fanconi anemia , chemotherapy , biochemistry , chemistry , dna repair , gene
Seventeen patients with Fanconi anemia (FA) underwent allogeneic bone marrow transplantation (BMT) from matched related donors (MRD) between January 1999 and June 2003. Median age at BMT was 11 years. Conditioning regimen consisted of low‐dose cyclophosphamide (CY; 40 mg/kg) and busulfan (BU; 6 mg/kg) with the addition of lymphoglobulin (20 mg/kg) in two patients. Graft‐versus‐host disease (GVHD) prophylaxis included cyclosporine A (CsA) and methotrexate (MTX; 5 mg/m 2 at day 1, 3, 6). All patients engrafted (for an absolute neutrophil count >0.5 × 10 9 /L) after a median time of 12 days (range 10–16 days). Fourteen patients (82%) had sustained grafts, whereas three others (18%) rejected grafts between day +39 and +80 after transplantation. Two of them are still alive after successful second PBSC transplantation and one died. Acute and chronic GVHD occurred in 23% and 13% of patients, respectively. With a median follow‐up of 16 months (range 3–53 months), survival rate was 72% and Karnofsky score was at least 90%. The low‐dose BU/CY regimen, in FA patients allografted from an HLA‐matched related donor, allowed engraftment with relative low toxicity. Early graft failure (GF) remains a problem and may require modification of this regimen. Pediatr Blood Cancer 2006, 46:496–500. © 2005 Wiley‐Liss, Inc.