title: Unrelated cord blood transplant in children with high-risk acute lymphoblastic leukemia: a long-term follow-up.
abstract: OBJECTIVE
  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.
  
  
  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.
  
  
  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.
authors.affiliation: Division of Hematology, Department of Cellular Biotechnologies and Hematology, University La Sapienza, Rome, Italy. iori@bce.uniroma1.it
authors.forename: Anna Paola
authors.initials: AP
authors.lastname: Iori
authors.forename: William
authors.initials: W
authors.lastname: Arcese
authors.forename: Filippo
authors.initials: F
authors.lastname: Milano
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Calabrese
authors.forename: Giovanni Fernando
authors.initials: GF
authors.lastname: Torelli
authors.forename: Walter
authors.initials: W
authors.lastname: Barberi
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Mascolo
authors.forename: Lidia
authors.initials: L
authors.lastname: De Felice
authors.forename: Maria
authors.initials: M
authors.lastname: Screnci
authors.forename: Barbarella
authors.initials: B
authors.lastname: Lucarelli
authors.forename: Luigi
authors.initials: L
authors.lastname: Malandruccolo
authors.forename: Maria Paola
authors.initials: MP
authors.lastname: Perrone
authors.forename: Simona
authors.initials: S
authors.lastname: Salvatori
authors.forename: Luca
authors.initials: L
authors.lastname: Laurenti
authors.forename: Emilia
authors.initials: E
authors.lastname: Iannella
authors.forename: Roberto
authors.initials: R
authors.lastname: Ricci
authors.forename: Maria Luisa
authors.initials: ML
authors.lastname: Moleti
authors.forename: Robin
authors.initials: R
authors.lastname: Foà
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D001772:Blood Cell Count; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D036101:Cord Blood Stem Cell Transplantation / Q000401:mortality / Q000706:statistics & numerical data*; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D011183:Postoperative Complications / Q000401:mortality; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000188:drug therapy / Q000401:mortality / Q000601:surgery*; D011379:Prognosis; D012008:Recurrence; D012306:Risk; D016879:Salvage Therapy; D016019:Survival Analysis; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents