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Reduced dose cyclophosphamide, fludarabine and antithymocyte globulin for sibling and unrelated transplant of children with severe and very severe aplastic anemia
Author(s) -
Chung NackGyun,
Lee Jae Wook,
Jang PilSang,
Jeong DaeChul,
Cho Bin,
Kim HackKi
Publication year - 2013
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12073
Subject(s) - medicine , fludarabine , cyclophosphamide , cumulative incidence , regimen , gastroenterology , incidence (geometry) , surgery , cohort , chemotherapy , physics , optics
We evaluated the results of a novel conditioning regimen of reduced dose cyclophosphamide (Cy, 25 mg/kg for four days), fludarabine (Flu, 30 mg/m 2 for four days), and rabbit ATG (2.5 mg/kg for three days) for allogeneic transplant of children with SAA , implemented since January 2009. Overall, 23 patients were treated with this regimen (16 male, seven female), including 10 diagnosed with VSAA . Donors included eight‐ MSD and 15 UD (five‐matched UD , and 10 mismatched UD ). All patients showed neutrophil and platelet engraftment. Cumulative incidence of acute (grade 2 or above) and chronic GVHD was 26.1% and 8.7%, respectively. Estimated two‐yr FFS and OS for the entire cohort was 90.3 ± 6.5%. Rates of TRM and graft failure were 5.3% and 4.3%, respectively. No difference in OS was found according to disease severity ( SAA vs. VSAA , p = 0.184), or according to donor type ( MSD vs. UD , p = 0.699). Excellent outcomes of patients with VSAA underscore the efficacy of allogeneic transplant as a means of expediting hematopoietic recovery. Improved survival of UD transplant reaffirms its role as a valid therapeutic alternative in the absence of MSD .