Autologous peripheral blood stem cell transplantation for acute myeloid leukemia

Edo Vellenga*, Wim van Putten, Gert Jan Ossenkoppele, Leo F. Verdonck, Matthias Theobald, Jan J. Cornelissen, Peter C. Huijgens, Johan Maertens, Alois Gratwohl, M Ron Schaafsma, Urs Schanz, Carlos Graux, Harry C. Schouten, Augustin Ferrant, Mario Bargetzi, Martin F. Fey, Bob Lowenberg

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


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 as # NTR230 and #NTR291. (Blood. 2011;118(23):6037-6042)
Original languageEnglish
Pages (from-to)6037-6042
Issue number23
Publication statusPublished - 1 Dec 2011

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