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-methylprednisolone and cyclosporine A. RESULTS The cumulative incidence of engraftment was 93% (95% CI:0.85-0.93). The cumulative incidence of grade III-IV acute and chronic GVHD was 7% (95% CI: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% CI:0.13-0.45) and 31% (95% CI:0.16-0.61), respectively. The 9-year overall survival, leukemia-free survival and event-free survival were 42% (95% CI:0.52-0.93), 47% (95% CI:0.25-0.61) and 46% (95% CI:0.33-0.61), respectively. A number of CFU-GM <1 x 10(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.