Summary:We report results of unrelated cord blood transplants (UCBT) in 29 pediatric recipients in one center and the risk factors associated with survival. Median age: 9 years (0.5-20); diagnosis: ALL (9), AML (4), CML (1), HD (3), HLH (1), NHL (3), NBL (2); B-thal (1), FA (1), FEL (1), Krabbe (1), WAS (1), SAA (1); median follow-up: 11 months; conditioning: total body irradiation (TBI)-ablative (14), chemotherapy-ablative (6) and reduced intensity chemotherapy (9); GVHD prophylaxis: MMF/FK506 (18), cyclosporin A (CsA) þ steroids7MMF (7) or CsA þ methotrexate (MTX) (4); median total nucleated cells (TNC): 3.8 Â 10 7 /kg (1.1-11); median CD34 þ : 2.3 Â 10 5 /kg (0.2-9.9); and HLA match: 2 (6/6), 5 (5/ 6), 22 (4/6). Neutrophil engraftment by cumulative incidence curves 63% (median 28 (95% confidence interval (CI) 18-32)). Probability of Xgrade II acute graft-versus-host disease (aGVHD) by day þ 60 27%, Xgrade III aGVHD 20% and chronic graft-versus-host disease 3%. Estimated 1-year overall survival (OS) 46% (95% CI 30-71) and standard risk 60% (95% CI 29-100%). Variables associated with improved survival by multivariate analysis include non-TBI-ablative conditioning (P ¼ 0.024), CD34 þ /kg (P ¼ 0.038) and gender (P ¼ 0.048). These results suggest that CD34/kg cell dose and non-TBI-ablative conditioning may be important variables influencing OS following UCBT in pediatric recipients. Given the small number of patients, these results should be viewed cautiously.

Summary:Reduced intensity (RI) allogeneic stem cell transplantation (AlloSCT) was initially demonstrated in adults following HLA-matched family and unrelated adult donor AlloSCT. There is little information about RI AlloSCT in children. We report results of a pilot study of RI AlloSCT in 21 recipients (p21 years). Age: median 13 (0.5-21) years, 8F:13M, 14 unrelated cord blood units (UCB) (10 4/6, 4 5/6), two related BM (6/6, 5/6), four related PBSC (2 6/6, 2 5/6), and one related BM þ PBSC (6/6). RI: fludarabine, busulfan (n ¼ 14); fludarabine, cyclophosphamide (n ¼ 4); fludarabine, melphalan (n ¼ 1); total body irradiation, fludarabine, cyclophosphamide (n ¼ 1); or fludarabine, cyclophosphamide, and etoposide (n ¼ 1). Graft-versus-host disease prophylaxis: FK506 0.03 mg/ kg/day and mycophenolate mofetil 15 mg/kg/q 12 h. UCB median nuc/kg and CD34/kg was 4.3 Â 10 7 /kg (0.9-10.8) and 1.9 Â 10 5 /kg (0.3-6.9), and related BM/PBSC median nuc/kg and CD34/kg was 8.3 Â 10 8 (4.7-18.9) and 5.0 Â 10 6 /kg (4.6-6.4). Maximal chimerism following unrelated cord blood transplantation, 100% Â 7, 98% Â 1, 95% Â 2, 55% Â 1, and 0% Â 3; related PBSC/BM, 100% Â 5, 65% Â 1, and 55% Â 1. Graft failure occurred in 5/21 (24%). In summary, RI AlloSCT in children is feasible and tolerable (p25% GF) and results in X85% of recipients initially achieving X50% donor chimerism.

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