Unrelated cord blood transplant in children with high-risk acute lymphoblastic leukemia: a long-term follow-up

Background and Objectives The aim of this single center study was to assess the impact of pre-transplant factors on long-term follow-up In young patients affected by high-risk acute lymphoblastic leukemia (ALL) who underwent an unrelated cord blood transplant (CBT). The conditioning regimens, graft-versus-host disease (GVHD) prophylaxis and supportive policies were uniform for all patients. Design and Methods We analyzed the results of CBT performed In 30 patients, aged <18 years, affected by high risk ALL. As conditioning regimen, all patients received 12 Gy fractionated total body irradiation, etoposide, cyclophosphamide and horse anti-lymphocyte globulin. GVHD prophylaxis consisted of 6-methylprednlsolone and cyclosporine A. Results The cumulative incidence of engraftment was 93% (95% CI:0.85-0.93). The cumulative incidence of grade lll-IV acute and chronic GVHD was 7% (95% 01:0.01-0.19) and 33% (95% CI: 0.17-0.64), respectively. The 9-year cumulative incidence of transplant-related mortality and relapse was 34% (95% 01:0.13-0.45) and 31% (95% 01:0.16-0.61), respectively. The 9-year overall survival, leukemia-free survival and event-free survival were 42% (95% 01:0.52-0.93), 47% (95% 01:0.25-0.61) and 46% (95% 01:0.33-0.61), respectively. A number of CFU-GM <1x10(4)/Kg of recipient body weight was the only factor that negatively affected all outcome parameters both In univariate and multivariate analyses. Interpretation and Conclusions The Infused cell dose expressed as in vitro progenitor cell growth represents the most Important pre-transplant factor affecting the long-term outcome after an unrelated CBT in young patients with high risk ALL. The number of CFU-GM should thus be considered in the selection process of cord blood units for transplant.