PURPOSE: 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). PATIENTS AND 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 toallogeneic 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. CONCLUSION: 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.
RCT Entities:
PURPOSE: 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). PATIENTS AND 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. CONCLUSION: 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: Izaskun Elorza; Carlos Palacio; Jose Luis Dapena; Laura Gallur; José Sánchez de Toledo; Cristina Díaz de Heredia Journal: Haematologica Date: 2010-02-23 Impact factor: 9.941
Authors: Jan J Melenhorst; Paul Castillo; Patrick J Hanley; Michael D Keller; Robert A Krance; Judith Margolin; Ann M Leen; Helen E Heslop; A John Barrett; Cliona M Rooney; Catherine M Bollard Journal: Mol Ther Date: 2014-09-30 Impact factor: 11.454
Authors: Raquel Malumbres; Vicente Fresquet; Jose Roman-Gomez; Miriam Bobadilla; Eloy F Robles; Giovanna G Altobelli; M José Calasanz; Erlend B Smeland; Maria Angela Aznar; Xabier Agirre; Vanesa Martin-Palanco; Felipe Prosper; Izidore S Lossos; Jose A Martinez-Climent Journal: Haematologica Date: 2011-04-01 Impact factor: 9.941
Authors: Elen Oliveira; Elaine S Costa; Juana Ciudad; Giuseppe Gaipa; Łukasz Sedek; Susana Barrena; Tomasz Szczepanski; Chiara Buracchi; Daniela Silvestri; Patrícia F R Siqueira; Fabiana V Mello; Rafael C Torres; Leonardo M R Oliveira; Isabelle V C Fay-Neves; Edwin Sonneveld; Vincent H J van der Velden; Esther Mejstrikova; Josep-Maria Ribera; Valentino Conter; Martin Schrappe; Jacques J M van Dongen; Marcelo G P Land; Alberto Orfao Journal: Cancers (Basel) Date: 2022-06-23 Impact factor: 6.575