Factors associated with outcome after cord blood transplantation in children with acute leukemia

We have analyzed factors influencing the outcome of 102 children with acute leukemia given a cord blood transplantation (CBT) and reported to the Euro cord Registry. Seventy patients with acute lymphoblastic and 32 with acute myeloid leukemia were given either a related (n = 42) or an unrelated (n = 60) CBT. Children given CBT during first or second complete remission were considered as belonging to the good-risk group (n = 66), whereas those who received a transplant in a more advanced stage of disease were assigned to the poor-risk group (n = 36). In the related group (RCBT), 12 of 42 patient s received transplantation from an HLA-disparate donor, whereas in the unre lated group (UCBT) 54 of 60 received an HLA mismatched CBT Kaplan-Meier est imates for neutrophil recovery at day 60 were 84% +/- 7% in RCBT and 79 +/- 6% in UCBT (P=.16). In multivariate analysis, the most important factor in fluencing neutrophil engraftment in UCBT was a nucleated cell dose infused greater than 3.7 x 10(7)/kg (P =.05, relative risk [RR] of 1.85, 95% confid ence interval [CII: 0.98-3.4). The incidence of grade II through IV acute g raft-versus-host disease was 41% +/- 8% in the RCBT group and 37% +/- 6% In the UCBT group (P =.59). Kaplan-Meier estimates of 2-year event-free survi val (EFS) after RCBT or UCBT were 39% +/- 8% and 30% +/- 7%, respectively ( P=.19). In multivariate analysis, the most important factor influencing EFS was disease status at time of transplantation: good-risk patients had a 2- year EFS of 49% +/- 7% as compared to 8% +/- 5% in patients with more advan ced disease (P =.0003, RR: 0.40, 95% CI: 0.24 to 0.65). This was a conseque nce of both an increased 1-year transplant related mortality and a higher 2 -year relapse rate in the poor-risk group (65% +/- 9% and 77% +/- 14%, resp ectively), as compared with good risk patients (34% +/- 6% and 31% +/- 9%, respectively). These data confirm that allogeneic CBT from either a related or an unrelated donor is a feasible procedure able to cure a significant p roportion of children with acute leukemia, especially if transplanted in a favorable phase of disease. (C) 1999 by The American Society of Hematology.