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.
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.
Ribera
Jose-Maria
JM
Servicio de Hematología Clínica, Institut Català d'Oncologia-Hospital Universitari Germans Trias i Pujol, Badalona, Spain. jribera@iconcologia.net
Ortega
Juan-José
JJ
Oriol
Albert
A
Bastida
Pilar
P
Calvo
Carlota
C
Pérez-Hurtado
José-María
JM
González-Valentín
María-Elvira
ME
Martín-Reina
Victoria
V
Molinés
Antonio
A
Ortega-Rivas
Fernando
F
Moreno
Maria-José
MJ
Rivas
Concepción
C
Egurbide
Izaskun
I
Heras
Inmaculada
I
Poderós
Concepción
C
Martínez-Revuelta
Eva
E
Guinea
José-Maria
JM
del Potro
Eloy
E
Deben
Guillermo
G
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
D000970:Antineoplastic Agents