Successful bone marrow transplantation for children with aplastic anemia based on a best-available evidence strategy.

A best-available evidence strategy, i.e., the best-available donors, conditioning regimens and GVHD prophylaxis were chosen at the time of BMT for AA, was analyzed retrospectively. The outcomes for 18 children with AA who underwent allogeneic BMT were analyzed. The median age was 11 yr (range 4-16), and nine were men. As conditioning regimens, seven had low-dose irradiation + CY, six had ATG + CY + Flu, and five had ATG + CY. Donors were HLA-matched siblings in 10, HLA-mismatched family in one, HLA-matched unrelated in three, and HLA-mismatched unrelated in four. As GVHD prophylaxis, three received CsA alone, nine received CsA + MTX, and six received tacrolimus + MTX. All 18 patients showed engraftment. The median number of days until the neutrophil count exceeded 500/μL was 16 (range 11-21) post-transplant. Five developed more than grade 2 acute GVHD, and three developed extensive cGVHD. One patient died because of interstitial pneumonia complicated with cGVHD. Five-yr OS was 94% (95% CI: 83-105). These results suggest that a strategy of treating patients based on the best-available evidence is acceptable.