To assess the outcome of unrelated umbilical cord blood transplantation (UCBT), 141 children with AML who underwent UCBT (39 in first CR (CR1), 33 in CR2, 4 in CR3 and 65 at more advanced stages (not in CR)) were analyzed in a retrospective multicenter study in Japan. Short-term MTX was used for prophylaxis of acute GVHD in 80 cases (57%). The cumulative incidences of neutrophil recovery, platelet recovery and acute GVHD (grades 2-4) were 78.7, 62.4 and 40.1%, respectively, and the 100-day transplantation-related mortality (TRM) was 10.8%. Multivariate analysis showed that an infused CD34+ cell dose of 1.35 × 105 cells per kg or more was associated with favorable neutrophil and platelet recovery, and that short-term MTX was associated with a lower 100-day TRM. The 6-year relapse rate was 38.8% and was associated with disease status. Six-year overall survival was 45.8% (70.4 ± 8.3% in CR1, 59.3 ± 11.3% in CR2, 75.5 ± 21% in CR3 and 20.6 ± 6.2% for children with non-CR). We conclude that the results of UCBT are particularly promising for children with a karyotype suggesting a poor prognosis, and for those who receive transplants in CR2 and CR3 after an early relapse.
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