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Multicenter Study Comparative Study Clinical Trial
Stem-cell transplantation in children with acute lymphoblastic leukemia: A prospective international multicenter trial comparing sibling donors with matched unrelated donors-The ALL-SCT-BFM-2003 trial.
- Christina Peters, Martin Schrappe, Arend von Stackelberg, André Schrauder, Peter Bader, Wolfram Ebell, Peter Lang, Karl-Walter Sykora, Johanna Schrum, Bernhard Kremens, Karoline Ehlert, Michael H Albert, Roland Meisel, Susanne Matthes-Martin, Tayfun Gungor, Wolfgang Holter, Brigitte Strahm, Bernd Gruhn, Ansgar Schulz, Wilhelm Woessmann, Ulrike Poetschger, Martin Zimmermann, and Thomas Klingebiel.
- Christina Peters, Susanne Matthes-Martin, and Ulrike Poetschger, St Anna Children's Hospital, Vienna, Austria; Martin Schrappe, University Medical Center Schleswig-Holstein and Christian-Albrechts-University Kiel; André Schrauder, Kinderarztpraxis am Aalborgring, Kiel; Arend von Stackelberg and Wolfram Ebell, Charité-Children's Hospital Berlin, Berlin; Peter Bader and Thomas Klingebiel, Johann Wolfgang Goethe University, Frankfurt; Peter Lang, University Hospital Tübingen, Tübingen; Karl-Walter Sykora and Martin Zimmerman, Hannover Medical School, Hannover; Johanna Schrum, University Medical Center Hamburg-Eppendorf, Hamburg; Bernhard Kremens, University Hospital Essen, Essen; Karoline Ehlert, University Clinic Greifswald, Greifswald; Michael H. Albert, Dr. von Hauner University Children's Hospital, München; Roland Meisel, University Hospital Düsseldorf, Düsseldorf; Wolfgang Holter, Children's University Hospital Erlangen, Erlangen; Brigitte Strahm, University Hospital Freiburg, Freiburg; Bernd Gruhn, University Hospital Jena, Jena; Ansgar Schulz, University Hospital Ulm, Ulm; Wilhelm Woessmann, University Clinic Giessen, Giessen, Germany; and Tayfun Gungor, University Children's Hospital Zürich, Zürich, Switzerland. christina.peters@stanna.at.
- J. Clin. Oncol. 2015 Apr 10; 33 (11): 1265-74.
PurposeAlthough hematopoietic stem-cell transplantation is widely performed in children with high-risk acute lymphoblastic leukemia (ALL), the influence of donor types is poorly understood. Thus, transplantation outcomes were compared in the prospective multinational Berlin-Frankfurt-Muenster (BFM) study group trial: ALL-SCT-BFM 2003 (Allogeneic Stem Cell Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia).Patients And MethodsAfter conditioning with total-body irradiation and etoposide, 411 children with high-risk ALL received highly standardized stem-cell transplantations during the first or later remissions. Depending on donor availability, grafts originated from HLA-genoidentical siblings or from HLA-matched unrelated donors who were identified and matched by high-resolution allelic typing and were compatible in at least 9 of 10 HLA loci.ResultsFour-year event-free survival (± standard deviation [SD]) did not differ between patients with transplantations from unrelated or sibling donors (0.67 ± 0.03 v 0.71 ± 0.05; P = .405), with cumulative incidences of nonrelapse mortality (± SD) of 0.10 ± 0.02 and 0.03 ± 0.02 (P = .017) and relapse rates (± SD) of 0.22 ± 0.02 and 0.24 ± 0.04 (P = .732), respectively. Among recipients of transplantations from unrelated donors, no significant differences in event-free survival, overall survival, or nonrelapse mortality were observed between 9/10 and 10/10 matched grafts or between peripheral blood stem cells and bone marrow. The absence of chronic graft-versus-host disease had no effect on event-free survival. Engraftment was faster after bone marrow transplantation from siblings and was associated with fewer severe infections and pulmonary complications.ConclusionOutcome among high-risk pediatric patients with ALL after hematopoietic stem-cell transplantation was not affected by donor type. Standardized myeloablative conditioning produced a low incidence of treatment-related mortality and effective control of leukemia.© 2015 by American Society of Clinical Oncology.
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