title: Comparison of intensive chemotherapy, allogeneic, or autologous stem-cell transplantation as postremission treatment for children with very high risk acute lymphoblastic leukemia: PETHEMA ALL-93 Trial.
abstract: OBJECTIVE
  The optimal postremission therapy for children with very high-risk (VHR) acute lymphoblastic leukemia (ALL) is not well established. This randomized trial compared three options of postremission therapy: chemotherapy and allogeneic or autologous stem-cell transplantation (SCT).
  
  
  METHODS
  All 106 VHR-ALL patients received induction with five drugs followed by intensification with three cycles of chemotherapy. Patients in complete remission (CR) with an HLA-identical family donor were assigned to allogeneic SCT (n = 24) and the remaining were randomly assigned to autologous SCT (n = 38) or to delayed intensification followed by maintenance chemotherapy up to 2 years in CR (n = 38).
  
  
  RESULTS
  Overall, 100 patients achieved CR (94%). With a median follow-up of 6.5 years, 5-year disease-free survival (DFS) and overall survival (OS) probabilities were 45% (95% CI, 37% to 54%) and 48% (95% CI, 40% to 57%), respectively. The three groups were comparable in the main pretreatment ALL characteristics. Intention-to-treat analysis showed no differences for donor versus no donor in DFS (45%; 95% CI, 27% to 65% v 45%; 95% CI, 37% to 55%) and OS (48%; 95% CI, 30% to 67% v 51%; 95% CI, 43% to 61%), as well as for autologous SCT versus chemotherapy comparisons (DFS: 44%; 95% CI, 29% to 60% v 46%; 95% CI, 32% to 62%; OS: 45%; 95% CI, 31% to 62% v 57%; 95% CI, 43% to 73%). No differences were found within the different subgroups of ALL and neither were differences observed when the analysis was made by treatment actually performed.
  
  
  CONCLUSIONS
  This study failed to prove that, when a family donor is available, allogeneic SCT produces a better outcome than autologous SCT or chemotherapy in children with VHR-ALL.
authors.affiliation: Servicio de Hematología Clínica, Institut Català d'Oncologia-Hospital Universitari Germans Trias i Pujol, Badalona, Spain. jribera@iconcologia.net
authors.forename: Jose-Maria
authors.initials: JM
authors.lastname: Ribera
authors.forename: Juan-José
authors.initials: JJ
authors.lastname: Ortega
authors.forename: Albert
authors.initials: A
authors.lastname: Oriol
authors.forename: Pilar
authors.initials: P
authors.lastname: Bastida
authors.forename: Carlota
authors.initials: C
authors.lastname: Calvo
authors.forename: José-María
authors.initials: JM
authors.lastname: Pérez-Hurtado
authors.forename: María-Elvira
authors.initials: ME
authors.lastname: González-Valentín
authors.forename: Victoria
authors.initials: V
authors.lastname: Martín-Reina
authors.forename: Antonio
authors.initials: A
authors.lastname: Molinés
authors.forename: Fernando
authors.initials: F
authors.lastname: Ortega-Rivas
authors.forename: Maria-José
authors.initials: MJ
authors.lastname: Moreno
authors.forename: Concepción
authors.initials: C
authors.lastname: Rivas
authors.forename: Izaskun
authors.initials: I
authors.lastname: Egurbide
authors.forename: Inmaculada
authors.initials: I
authors.lastname: Heras
authors.forename: Concepción
authors.initials: C
authors.lastname: Poderós
authors.forename: Eva
authors.initials: E
authors.lastname: Martínez-Revuelta
authors.forename: José-Maria
authors.initials: JM
authors.lastname: Guinea
authors.forename: Eloy
authors.initials: E
authors.lastname: del Potro
authors.forename: Guillermo
authors.initials: G
authors.lastname: Deben
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000628:therapy*; D012074:Remission Induction; D012306:Risk; D033581:Stem Cell Transplantation / Q000379:methods*; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents