title: Autologous peripheral blood stem cell transplantation for acute myeloid leukemia.
abstract: We report the results of a prospective, randomized phase 3 trial evaluating autologous peripheral blood stem cell transplantation (ASCT) versus intensive consolidation chemotherapy in newly diagnosed AML patients in complete remission (CR1). Patients with AML (16-60 years) in CR1 after 2 cycles of intensive chemotherapy and not eligible for allogeneic SCT were randomized between intensive chemotherapy with etoposide and mitoxantrone or ASCT ater high-dose cyclophosphamide and busulfan. Of patients randomized (chemotherapy, n = 259; ASCT, n = 258), more than 90% received their assigned treatment. The 2 groups were comparable with regard to prognostic factors. The ASCT group showed a markedly reduced relapse rate (58% vs 70%, P = .02) and better relapse-free survival at 5 years (38% vs 29%, P = .065, hazard ratio = 0.82; 95% confidence interval, 0.66-1.1) with nonrelapse mortality of 4% versus 1% in the chemotherapy arm (P = .02). Overall survival was similar (44% vs 41% at 5 years, P = .86) because of more opportunities for salvage with second-line chemotherapy and stem cell transplantation in patients relapsing on the chemotherapy arm. This large study shows a relapse advantage for ASCT as postremission therapy but similar survival because more relapsing patients on the chemotherapy arm were salvaged with a late transplantation for relapse. This trial is registered at www.trialregister.nl as #NTR230 and #NTR291.
authors.affiliation: Department of Hematology, University of Groningen and University Medical Center Groningen, Groningen, The Netherlands. e.vellenga@umcg.nl
authors.forename: Edo
authors.initials: E
authors.lastname: Vellenga
authors.forename: Wim
authors.initials: W
authors.lastname: van Putten
authors.forename: Gert J
authors.initials: GJ
authors.lastname: Ossenkoppele
authors.forename: Leo F
authors.initials: LF
authors.lastname: Verdonck
authors.forename: Matthias
authors.initials: M
authors.lastname: Theobald
authors.forename: Jan J
authors.initials: JJ
authors.lastname: Cornelissen
authors.forename: Peter C
authors.initials: PC
authors.lastname: Huijgens
authors.forename: Johan
authors.initials: J
authors.lastname: Maertens
authors.forename: Alois
authors.initials: A
authors.lastname: Gratwohl
authors.forename: Ron
authors.initials: R
authors.lastname: Schaafsma
authors.forename: Urs
authors.initials: U
authors.lastname: Schanz
authors.forename: Carlos
authors.initials: C
authors.lastname: Graux
authors.forename: Harry C
authors.initials: HC
authors.lastname: Schouten
authors.forename: Augustin
authors.initials: A
authors.lastname: Ferrant
authors.forename: Mario
authors.initials: M
authors.lastname: Bargetzi
authors.forename: Martin F
authors.initials: MF
authors.lastname: Fey
authors.forename: Bob
authors.initials: B
authors.lastname: Löwenberg
mesh_terms: D000293:Adolescent; D000328:Adult; D000970:Antineoplastic Agents / Q000627:therapeutic use; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use; D000972:Antineoplastic Agents, Phytogenic / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002066:Busulfan / Q000627:therapeutic use; D003131:Combined Modality Therapy / Q000379:methods; D003520:Cyclophosphamide / Q000627:therapeutic use; D005047:Etoposide / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy*; D008875:Middle Aged; D008942:Mitoxantrone / Q000627:therapeutic use; D011379:Prognosis; D011446:Prospective Studies; D012074:Remission Induction; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents; D018906:Antineoplastic Agents, Alkylating; D000972:Antineoplastic Agents, Phytogenic; D005047:Etoposide; D003520:Cyclophosphamide; D008942:Mitoxantrone; D002066:Busulfan