Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes

•We assessed the generalizability of results of a phase III trial for cord blood transplantation•We observed superior survival with the trial-specified regimen compared with that of other regimens•The results underscore the relative importance of the conditioning regimen for umbilical cord blood transplantation

The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation with 1 or 2 cord blood units (UCB) between 2006 and 2012. While the trial concluded that survival was similar regardless of number of units infused, survival was better than previously reported. This prompted a comparison of survival of trial versus nontrial patients to determine the generalizability of trial results and whether survival was better because of the trial treatment regimen. During the trial period, 396 recipients of a single UCB unit met trial eligibility but were not enrolled. Trial patients (n = 100) received total body irradiation (TBI) 1320 cGy, cyclophosphamide 120 mg/kg, and fludarabine 75 mg/m2 (TCF). Nontrial patients either received the same regimen (n = 62; nontrial TCF) or alternative regimens (n = 334; nontrial regimens). Five-year survival between trial and nontrial patients conditioned with TCF was similar (70% versus 62%). However, 5-year survival was significantly lower with nontrial TBI-containing (47%; hazard ratio [HR], 1.97; P = .001) and chemotherapy-only regimens (49%; HR, 1.87; P = .007). The results of BMT CTN 0501 appear generalizable to the population of trial-eligible patients. The survival difference between the trial-specified regimen and other regimens indicate the importance of conditioning regimen for UCB transplantation.