Allogeneic Stem-Cell Transplantation in Patients With NPM1-Mutated Acute Myeloid Leukemia: Results From a Prospective Donor Versus No-Donor Analysis of Patients After Upfront HLA Typing Within the SAL-AML 2003 Trial

Author:

Röllig Christoph1,Bornhäuser Martin1,Kramer Michael1,Thiede Christian1,Ho Anthony D.1,Krämer Alwin1,Schäfer-Eckart Kerstin1,Wandt Hannes1,Hänel Mathias1,Einsele Hermann1,Aulitzky Walter E.1,Schmitz Norbert1,Berdel Wolfgang E.1,Stelljes Matthias1,Müller-Tidow Carsten1,Krug Utz1,Platzbecker Uwe1,Wermke Martin1,Baldus Claudia D.1,Krause Stefan W.1,Stölzel Friedrich1,von Bonin Malte1,Schaich Markus1,Serve Hubert1,Schetelig Johannes1,Ehninger Gerhard1

Affiliation:

1. Christoph Röllig, Martin Bornhäuser, Michael Kramer, Christian Thiede, Uwe Platzbecker, Martin Wermke, Friedrich Stölzel, Malte von Bonin, Markus Schaich, Johannes Schetelig, and Gerhard Ehninger, Medizinische Klinik und Poliklinik I, Universitätsklinikum der Technischen Universität Dresden; Johannes Schetelig, DKMS, German Bone Marrow Donor Center, Dresden; Anthony D. Ho and Alwin Krämer, Medizinische Universitätsklinik, Abteilung Innere Medizin V, Heidelberg; Kerstin Schäfer-Eckart and Hannes Wandt, 5....

Abstract

Purpose The presence of a mutated nucleophosmin-1 gene (NPM1mut) in acute myeloid leukemia (AML) is associated with a favorable prognosis. To assess the predictive value with regard to allogeneic stem-cell transplantation (SCT), we compared the clinical course of patients with NPM1mut AML eligible for allogeneic SCT in a donor versus no-donor analysis. Patients and Methods Of 1,179 patients with AML (age 18 to 60 years) treated in the Study Alliance Leukemia AML 2003 trial, we identified all NPM1mut patients with an intermediate-risk karyotype. According to the trial protocol, patients were intended to receive an allogeneic SCT if an HLA-identical sibling donor was available. Patients with no available donor received consolidation or autologous SCT. We compared relapse-free survival (RFS) and overall survival (OS) depending on the availability of a suitable donor. Results Of 304 eligible patients, 77 patients had a sibling donor and 227 had no available matched family donor. The 3-year RFS rates in the donor and no-donor groups were 71% and 47%, respectively (P = .005); OS rates were 70% and 60%, respectively (P = .114). In patients with normal karyotype and no FLT3 internal tandem duplication (n = 148), the 3-year RFS rates in the donor and no-donor groups were 83% and 53%, respectively (P = .004); and the 3-year OS rates were 81% and 75%, respectively (P = .300). Conclusion Allogeneic SCT led to a significantly prolonged RFS in patients with NPM1mut AML. The absence of a statistically significant difference in OS is most likely a result of the fact that NPM1mut patients who experienced relapse responded well to salvage treatment. Allogeneic SCT in first remission has potent antileukemic efficacy and is a valuable treatment option in patients with NPM1mut AML with a sibling donor.

Publisher

American Society of Clinical Oncology (ASCO)

Subject

Cancer Research,Oncology

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