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Conditioning Regimens

Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience

Bone Marrow Transplantation volume 48, pages 491–501 (2013)Cite this article

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Abstract

We report the long-term follow-up of children transplanted with Treosulfan (TREO)-based conditioning in Germany and Austria. Nine centres reported a total of 109 transplantations. Patients were stratified according to the paediatric TRM risk score derived from the paediatric BMT registry (PRST) and compared with the historical transplant population of this registry. Underlying diseases were malignancies, immunodeficiencies, and haematologic and metabolic disorders. TREO total dose ranged from 21–42 g/m2. Additional conditioning drugs included fludarabine, thiotepa, melphalan, CY and/or TBI. EFS at 3 years for non-malignant and malignant diseases was 88% and 49%, respectively. Leukaemia patients in remission had a survival of 51% at 3 years; nonremission patients relapsed and died within 18 months. TRM and OS in the low-risk groups 0 and 1 were similar to PRST controls. TRM in the high-risk groups 2 and 3 was markedly lower (9% vs 28% and 13% vs 53%, respectively) than in the PRST group, but OS was similar. In conclusion, TREO-based conditioning regimens in children resulted in excellent engraftment and long-term survival in nonmalignant disease. In high-risk malignancy, low acute toxicity was followed by low TRM but it did not translate into increased survival.

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Acknowledgements

This study was supported by the Integrated Research and Treatment Centre Transplantation (BMBF grant number 01E00802 IFB-TX, Hannover). We would like to give our special thanks to all the patients and their parents who took part in this study. Moreover, we want to thank all the physicians and nurses who have been involved in the transplantation and the follow-up care of the patients. We thank K Mischke and J Wolf for their diligent data management and S Blöss for patient management.

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Author notes

  1. I Müller

    Present address: Interdisciplinary Clinic for Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.,

Authors and Affiliations

  1. Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany

    R Beier, M Sauer, M Zimmermann & K-W Sykora

  2. Department of Pediatrics and Adolescent Medicine, Section of Stem Cell Transplantation, Ulm University, Ulm, Germany

    A Schulz & M Hönig

  3. Pediatric Stem Cell Transplantation Program, University Würzburg, Children’s Hospital, Würzburg, Germany

    M Eyrich & P-G Schlegel

  4. St Anna Kinderspital, Vienna, Austria

    W Holter

  5. Section of Oncology, Haematology and Cell Therapy, Children’s and Adolescent’s Hospital, University Hospital Erlangen, Erlangen, Germany

    K D Stachel

  6. Department of Paediatric Haematology-Oncology, University Children’s Hospital Münster, Münster, Germany

    K Ehlert

  7. Department of Oncology, Haematology, Immunology and Pneumonology, Centre for Child and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany

    J Greil

  8. Department of Paediatric Haematology-Oncology, Children’s Hospital Giessen, University Hospital Giessen and Marburg, Giessen, Germany

    W Nürnberger & W Wößmann

  9. Section of Stem Cell Transplantation, Center for Child and Adolescent Medicine –III, Goethe-University Frankfurt/Main, Frankurt/Main, Germany

    P Bader

  10. Clinical Department of Pediatric Haematology-Oncology, Graz Medical University, Graz, Austria

    C Urban

  11. Department of General Pediatrics, Hematology and Oncology, University Children’s Hospital Tübingen, Tübingen, Germany

    I Müller

  12. Department of Children’s and Adolescent Medicine, Division of Haematology and Oncology, University Hospital Carl Gustav Carus, Dresden, Germany

    M Suttorp

  13. Department of Pediatrics, Jena University Hospital, Friedrich Schiller University Jena, Jena, Germany

    B Gruhn

  14. Department of Pediatric Oncology, Hematology and Immunology, Center for Child and Adolescent Health, Heinrich-Heine-University, Duesseldorf, Germany

    R Meisel

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  1. R Beier
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  2. A Schulz
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  7. K D Stachel
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Corresponding author

Correspondence to K-W Sykora.

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Competing interests

The following authors received financial support from Medac AG, Germany: Rita Beier, Ingo Müller and Karl-Walter Sykora research support. Karoline Ehlert, Ingo Müller and Karl-W Sykora travel grants. Meinolf Suttorp an educational grant. The other authors have no conflicts of interests in relation to this work to declare.

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Beier, R., Schulz, A., Hönig, M. et al. Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience. Bone Marrow Transplant 48, 491–501 (2013). https://doi.org/10.1038/bmt.2012.188

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