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Stem cell transplantation

Hematopoietic stem cell transplantation for children with acute myeloid leukemia—results of the AML SCT-BFM 2007 trial

Leukemia volume 34, pages 613–624 (2020)Cite this article

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A Correction to this article was published on 25 November 2019

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Abstract

AML SCT-BFM 2007 was the first hematopoietic stem cell transplantation (HCT) trial in Germany to comply with the European Clinical Trials Directive, and aimed to standardize pediatric HCT for acute myeloid leukemia (AML) across centers in Germany, Austria, and the Czech Republic. Children with high-risk features and a good early response achieving a complete first remission (CR-1) and those in CR-2 after a first relapse were stratified to receive HCT from a matched donor after myeloablative conditioning consisting of busulfan, cyclophosphamide, and melphalan. Four-year EFS and OS were 61 and 70%. Cumulative incidence of relapse (CIR) was 22%. TRM was 15% and correlated with age reaching 9% (SE 3%) in children younger than 12 years and 31% (SE 9%) in older children and adolescents. Children with poorly responding primary disease or relapse were allocated to receive early HCT after a cytoreductive regimen with fludarabine, amsacrine, and cytarabine, followed by reduced intensity conditioning and prophylactic donor lymphocyte infusions. Four-year EFS and OS were 49 and 53%. CIR was 38% and TRM 11%. For patients with primary poor response disease, early use of RIC HCT followed by prophylactic DLI can induce long-term remissions in more than 50% (EFS 46% (SE 9%)).

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Acknowledgements

This work was supported by grants from the German Federal Ministry of Education and Research (IFB-TX) (MGS), the Deutsche Forschungsgemeinschaft (Cluster of Excellence REBIRTH) (HL), Verein für krebskranke Kinder E.V. Hannover (MGS), and Neovii Biotech (formerly Fresenius) (MGS).

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Authors and Affiliations

  1. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Medizinische Hochschule Hannover, Hanover, Germany

    Martin G. Sauer, Ursula Creutzig, Kirsten Mischke & Martin Zimmermann

  2. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Tuebingen, Tuebingen, Germany

    Peter J. Lang & Rupert Handgretinger

  3. Department of Pediatrics, Division of Pediatric Hematology and Oncology, Dr von Hauner Children’s Hospital, LMU, Munich, Germany

    Michael H. Albert

  4. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Frankfurt, Frankfurt, Germany

    Peter Bader & Thomas Klingebiel

  5. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Würzburg, Würzburg, Germany

    Matthias Eyrich & Paul G. Schlegel

  6. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Heidelberg, Heidelberg, Germany

    Johann Greil

  7. Department of Pediatrics, Jena University Hospital, Jena, Germany

    Bernd Gruhn

  8. Department of Pediatrics, Division of Pediatric Hematology and Oncology, St Anna Kinderspital Vienna, Vienna, Austria

    Wolfgang Holter & Christina Peters

  9. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Duisburg-Essen, Essen, Germany

    Bernhard Kremens & Dirk Reinhardt

  10. Department of Pediatrics, Division of Pediatric Hematology and Oncology, Hannover Clinical Trial Center, Hanover, Germany

    Heiko von der Leyen & Christine Pohler

  11. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Giessen, Giessen, Germany

    Christine Mauz-Körholz & Wilhelm Woessmann

  12. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Düsseldorf, Düsseldorf, Germany

    Roland Meisel & Arndt Borkhardt

  13. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Hamburg, Hamburg, Germany

    Ingo Müller & Johanna Schrum

  14. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Freiburg, Freiburg, Germany

    Charlotte M. Niemeyer & Brigitte Strahm

  15. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Muenster, Muenster, Germany

    Birgit Burkhardt

  16. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Ulm, Ulm, Germany

    Ansgar S. Schulz

  17. Department of Pediatrics, Division of Pediatric Hematology and Oncology, Hospital Motol, Charles University, Prague, Czechia

    Petr Sedlacek

  18. Department of Pediatrics, Division of Pediatric Hematology and Oncology, University of Duesseldorf, Duesseldorf, Germany

    Petr Sedlacek

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  1. Martin G. Sauer
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Contributions

MGS was principal investigator of the trial, designed the trial, collected and reviewed data, analyzed data, wrote and edited the manuscript; AB and RH were co-investigators of the trial, designed the trial, collected and reviewed data, wrote and edited the manuscript; HL and CPo legally sponsored the trial, verified and monitored data, wrote and edited the manuscript; KM collected data, analyzed data, and edited the manuscript; MZ designed the trial, did interim and final statistical analysis and created the figures; UC and DR coordinated the interface between upfront studies and the HCT trial, designed the trial, and edited the manuscript; PJL, MHA, PB, MKE, JG, BG, WH, TK, BK, CMK, RM, IM, CMN, CPe, CR, PGS, ASS, JS, PS, BS, WW, and RH, collected data and edited the manuscript.

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Correspondence to Martin G. Sauer.

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MGS received research support from Neovii Biotech. The remaining authors declare that they have no conflict of interest.

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Sauer, M.G., Lang, P.J., Albert, M.H. et al. Hematopoietic stem cell transplantation for children with acute myeloid leukemia—results of the AML SCT-BFM 2007 trial. Leukemia 34, 613–624 (2020). https://doi.org/10.1038/s41375-019-0584-8

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